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33 Bone Marrow Failure Disorders Trials Near You
Power is an online platform that helps thousands of Bone Marrow Failure Disorders patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
Learn More About PowerTreosulfan-Based Conditioning for Bone Marrow Failure
Columbus, Ohio
This trial tests if a combination of three drugs can reduce complications for patients with bone marrow failure diseases. The drugs work by killing harmful cells, stopping their growth, and reducing immune reactions.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:1 - 49
Key Eligibility Criteria
Disqualifiers:Aplastic Anemia, Fanconi Anemia, MDS, Others
40 Participants Needed
Geriatric Care Optimization for Blood Cancers
Columbus, Ohio
This clinical trial tests whether a geriatric optimization plan (GO!) works to improve survival in patients over 60 with a hematologic malignancy or bone marrow failure syndrome eligible for allogeneic hematopoietic cell transplant. GO! focuses on creating a tailored and specific plan for each patient to make changes in their daily lives. These may include changes to their diet, sleep, activity, medicines, or even referrals to other providers depending on the patient's needs. Studying survival and quality of life in patients over 60 receiving an allogeneic hematopoietic cell transplant may help identify the effects of treatment.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:60+
Key Eligibility Criteria
Disqualifiers:Dementia, No Transplant, Others
30 Participants Needed
EXG34217 for Bone Marrow Failure
Cincinnati, Ohio
This trial tests the safety and tolerability of EXG34217 in patients with bone marrow failure due to telomere biology disorders. The treatment involves collecting, processing, and reinfusing the patient's own blood cells to help improve their bone marrow function.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:12+
Key Eligibility Criteria
Disqualifiers:Cancer, Severe Bone Marrow Failure, Others
Must Not Be Taking:Danazol, Androgens
12 Participants Needed
Emapalumab for Aplastic Anemia
Cincinnati, Ohio
The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options.
Funding Source- FDA OOPD
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:0 - 25
Key Eligibility Criteria
Disqualifiers:Uncontrolled Infection, Previous SAA Treatment, Inherited Bone Marrow Failure, Others
35 Participants Needed
Bone Marrow Transplant for Dyskeratosis Congenita
Cincinnati, Ohio
This trial tests a new bone marrow transplant method for patients with Dyskeratosis congenita. It uses fludarabine and antibodies to avoid harmful side effects, aiming to improve survival and reduce complications. Fludarabine-based regimens have been shown to be effective and feasible in reducing transplant-related morbidity in patients with Dyskeratosis congenita.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:30 - 65
Key Eligibility Criteria
Disqualifiers:MDS, AML, HIV, Pregnancy, Others
Must Not Be Taking:Fludarabine, Alemtuzumab, Mycophenolate, Others
40 Participants Needed
EXG34217 for Bone Marrow Failure Syndrome
Cincinnati, Ohio
This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 1
12 Participants Needed
Reduced-Intensity Stem Cell Transplant for Non-Malignant Disorders
Pittsburgh, Pennsylvania
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:2 - 55
Key Eligibility Criteria
Disqualifiers:Active Malignancy, Severe Aplastic Anemia, Others
100 Participants Needed
RIC + BMT for Non-Malignant Disorders
Grand Rapids, Michigan
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:1 - 21
Key Eligibility Criteria
Disqualifiers:HLA-identical Sibling Donor, Cirrhosis, Uncontrolled Infection, HIV, Others
29 Participants Needed
Eltrombopag for Aplastic Anemia
Bethesda, Maryland
Background:
* Moderate aplastic anemia is a blood disease which may require frequent blood and platelet transfusions. Sometimes patients with this disease can be treated with immunosuppressive drugs. Not all patients respond and not all patients are suitable for this treatment.
* Thrombopoietin (TPO) is a protein made by the body. The bone marrow needs TPO to produce platelets. TPO may also be able to stimulate bone marrow stem cells to produce red cells and white cells. However, TPO cannot be given by mouth. This has led researchers to develop the drug eltrombopag, which acts in the same way and can be given by mouth. Eltrombopag has been shown to safely increase platelet numbers in healthy volunteers and in patients with other chronic blood diseases, including severe aplastic anemia. Researchers are interested in looking at whether eltrombopag can be given to people with moderate aplastic anemia and significantly low blood cell counts.
Objectives:
- To evaluate the safety and effectiveness of eltrombopag in people with moderate aplastic anemia or patients with bone marrow failure and unilineage cytopenia who need treatment for significantly low blood cell counts.
Eligibility:
- People at least 2 years of age who have moderate aplastic anemia or bone marrow failure and unilineage cytopenia,and significantly low blood cell counts.
Design:
* Patients will be screened with a physical examination, medical history, blood tests, a bone marrow biopsy, and an eye exam.
* Patients will receive eltrombopag by mouth once a day.
* Patients will have weekly blood tests to monitor the effectiveness of the treatment and adjust the dose in response to possible side effects.
* Patients may continue to take eltrombopag if their platelet count or hemoglobin increases, their requirement for platelet or blood transfusion decreases after 16 to 20 weeks of treatment, and there have been no serious side effects. Access to the drug will continue until the study is closed. Patients will be asked to return for a follow-up visit 6 months after the last dose of medication.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:2 - 100
Key Eligibility Criteria
Disqualifiers:Fanconi Anemia, HIV, Cancer, Others
Must Not Be Taking:ATG, Campath, Cytokines, Others
34 Participants Needed
Imatinib for Bone Marrow Failure Syndrome
Bethesda, Maryland
Background:
Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise easily; they are also at higher risk of getting cancers of the blood, bone marrow, and lymph nodes.
Objective:
The purpose of the study includes determining which dose of imatinib is best for people with pathogenic or likely pathogenic RUNX1 mutations without blood cancers, and to determine whether there are any changes in platelet function and inflammatory markers.
Eligibility:
Adults aged 18 and older with RUNX1 mutations. Healthy people without this mutation, including family members of affected participants, are also needed.
Design:
Participants with the RUNX1 mutation will be screened. They will have a physical exam with blood tests. They will have a test of their heart function. They may need a new bone marrow biopsy if they haven't had one in the past year.
Imatinib is a tablet taken by mouth once a day, every day, at home. Affected participants in different parts of the study will take imatinib for either 28 days or up to 84 days. They will fill out questionnaires about how they are feeling.
For the first part of the study, participants will have blood tests every 2 weeks, either at home or at the NIH, while they are taking the imatinib. They will have a follow up visit, at home or at the NIH, when they are done taking imatinib on Day 28.
Participants on the second part of the study will come to NIH on days 1 and days 84. They will have blood tests every 2 weeks (at home or the NIH) while they are taking imatinib. They may opt to have a bone marrow biopsy repeated after they finish their course of imatinib.
Participants will have a follow-up visit (at home or the NIH) 30 days after they stop taking imatinib.
Participants who do not have the RUNX1 mutation will have 1 clinic visit. They will have blood tests. They will fill out questionnaires. They may opt to have a bone marrow biopsy.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Pregnancy, Hematologic Therapy, Cardiac Conditions, Others
Must Not Be Taking:Aspirin, Anti-platelet, CYP3A4 Inhibitors, Others
75 Participants Needed
Pacritinib for Myelodysplastic Syndrome
Bethesda, Maryland
Background:
Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults. MDS and MDS/MPN can also progress to acute myeloid leukemia. Treatments for these disorders are risky and not always effective. Better treatments are needed.
Objective:
To test a study drug (pacritinib) in adults and children with MDS or MDS/MPN.
Eligibility:
Children (aged 12 to 17 years) and adults (aged 18 years and older) with MDS or MDS/MPN.
Design:
Participants will be screened. They will have a physical exam with blood tests. They will have tests of their heart function. They may have a bone marrow biopsy: An area over the hip will be numbed; a needle will be inserted to remove a sample of soft tissue from inside the hipbone.
Pacritinib is a capsule taken by mouth. All participants will take the study drug 2 times a day, every day, in 28-day cycles. They will write down the date and time they take each capsule. Doctors will assign varying dosages of the drug to different participants.
Participants will have clinic visits each week during cycle 1; every 2 weeks during cycle 2; and gradually increasing to every 3 months after cycle 13. Treatment will continue for up to 8 years.
Bone marrow biopsies, heart tests, and other tests will be repeated at intervals throughout the study. Participants will also fill out questionnaires about their quality of life, the symptoms of their disease, and other topics.
No Placebo Group
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 1, 2
Age:12+
Key Eligibility Criteria
Disqualifiers:Bleeding Disorders, Cardiac Conditions, Infections, Others
Must Not Be Taking:Anticoagulants, Antiplatelets, CYP3A4 Inhibitors
160 Participants Needed
Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), inherited bone marrow failure syndromes (IBMFS), short telomere syndromes, Fanconi anemia, and non-Fanconi DNA double-strand break (DNA-dsb) repair disorder.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:4 - 50
Key Eligibility Criteria
Disqualifiers:HIV, Hepatitis B/C, Pregnancy, Others
27 Participants Needed
Cord Blood Transplant for Blood Diseases
Rochester, New York
This trial uses stem cells from a baby's umbilical cord to treat patients who need new healthy stem cells. Patients first get strong medicine to clear out unhealthy cells, then receive the new stem cells, and take medications to prevent complications. Umbilical cord blood has been used in the treatment of various diseases, including leukemias, lymphomas, and immune system disorders.
No Placebo Group
Trial Details
Trial Status:Recruiting
Age:2 - 75
Key Eligibility Criteria
Disqualifiers:Pregnancy, HIV, Uncontrolled Infection, Others
30 Participants Needed
Let's Get REAL Tool for Pediatric Blood Cancers
St Louis, Missouri
The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular therapy decisions (SCTCT). The investigators will pilot the intervention among 24 youth and their parents, stratified by youth age (stratum 1, 8-12 years of age and stratum 2, 13-17 years of age).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:8+
Key Eligibility Criteria
Disqualifiers:Severe Medical Problems, Cognitive Incapacity, Others
60 Participants Needed
Fludarabine RIC for Bone Marrow Failure Syndrome
Philadelphia, Pennsylvania
This is a pilot study to determine whether fludarabine-based reduced intensity conditioning (RIC) regimens facilitate successful donor engraftment of patients with acquired aplastic anemia (AA) and Inherited bone marrow failure (iBMF) syndromes undergoing Matched related donor bone marrow transplant (MRD-BMT).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Early Phase 1
Age:< 22
Key Eligibility Criteria
Disqualifiers:Uncontrolled Infections, Myelodysplastic Syndrome, Others
25 Participants Needed
Stem Cell Transplant for Bone Marrow Failure Syndrome
Philadelphia, Pennsylvania
The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia IRB approved protocols that utilize CliniMACs technology for T depletion.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Early Phase 1
Age:< 30
Key Eligibility Criteria
Disqualifiers:Infections, HLA Matched Sibling, Pregnancy
100 Participants Needed
CD45RA Depleted Stem Cell Addback for Leukemia
Philadelphia, Pennsylvania
The major morbidities of allogeneic hematopoietic stem cell transplant (HSCT) using donors that are not human leukocyte antigen (HLA) matched siblings are graft vs host disease (GVHD) and life- threatening infections. T cell receptor alpha beta (TCRαβ) T lymphocyte depletion and CD19+ B lymphocyte depletion of alternative donor hematopoietic stem cell (HSC) grafts is effective in preventing GVHD, but immune reconstitution may be delayed, increasing the risk of infections. The central hypothesis of this study is that an addback of CD45RO memory T lymphocytes, derived from a fraction of the original donor peripheral stem cell product depleted of CD45RA naïve T lymphocytes, will accelerate immune reconstitution and help decrease the risk of infections in TCRab/CD19 depleted PSCT.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:1 - 25
Key Eligibility Criteria
Disqualifiers:Hodgkin Lymphoma, Non-Burkitt Lymphoma, Others
Must Not Be Taking:Alemtuzumab
100 Participants Needed
Stem Cell Transplant for Leukemia
New York, New York
The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults. CD34+ stem cells are the cells that make all the types of blood cells in the body. GVHD is a condition that results from a reaction of transplanted donor T-lymphocytes (a kind of white blood cell) against the recipient's body and organs. Study subjects will be offered treatment involving the use of the CliniMACS® Reagent System (Miltenyi Biotec), a CD34+ selection device to remove T-cells from a peripheral blood stem cell transplant in order to decrease the risk of acute and chronic GVHD.
This study involves subjects who are diagnosed with a malignant disease, that has either failed standard therapy or is unlikely to be cured with standard non-transplant therapy, who will receive a peripheral blood stem cell transplant. A malignant disease includes the following: Chronic Myeloid Leukemia (CML) in chronic phase, accelerated phase or blast crisis; Acute Myelogenous Leukemia (AML); Myelodysplastic Syndrome (MDS); Juvenile Myelomonocytic Leukemia (JMML); Acute Lymphoblastic Leukemia (ALL); or Lymphoma (Hodgkin's and Non-Hodgkin's).
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:< 22
Key Eligibility Criteria
Disqualifiers:Uncontrolled Infection, Pregnancy, Breastfeeding
14 Participants Needed
CD34+ Stem Cell Selection for Bone Marrow Failure Syndromes
New York, New York
This trial uses a special machine to filter out harmful cells from donor blood to make stem cell transplants safer for young patients with non-cancerous diseases. By removing specific cells, it aims to prevent a serious immune reaction.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:< 40
Key Eligibility Criteria
Disqualifiers:Uncontrolled Infection, Pregnancy, HIV, Others
37 Participants Needed
Cord Blood Transplant for Blood Cancers
New York, New York
This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 21
Key Eligibility Criteria
Disqualifiers:Advanced Metabolic Disease, CNS Leukemic, Others
31 Participants Needed
Why Other Patients Applied
"I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."
WR
Obesity PatientAge: 58
"As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."
IZ
Healthy Volunteer PatientAge: 38
"My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."
HZ
Arthritis PatientAge: 78
"I've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."
ZS
Depression PatientAge: 51
"I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."
AG
Paralysis PatientAge: 50
Haploidentical Transplant for Severe Aplastic Anemia
Memphis, Tennessee
This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not fully matched to the patient. This includes studying the effects of the chemotherapy, radiation, the transplanted cell product and additional white blood cell (lymphocyte) infusions on the patient's body, disease and overall survival. The primary objective is to assess the rate of engraftment at 30 days and overall survival (OS) and event free survival (EFS) at 1 year post-hematopoietic cell transplantation (HCT).
Primary Objectives
* To estimate the rate of engraftment at 30 days after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide.
* To estimate the overall survival and event free survival at 1-year post transplantation.
Secondary Objectives
* To calculate the incidence of acute and chronic GVHD after HCT.
* To calculate the rate of secondary graft rejection at 1-year post transplantation
* To calculate the cumulative incidence of viral reactivation (CMV, EBV and adenovirus).
* To describe the immune reconstitution after TCR αβ+ T-cell-depleted graft infusion at 1 month, 3 months, 6 months, 9 months, and 1 year.
Exploratory Objectives
* To longitudinally assess the phenotype and epigenetic profile of T-cells in SAA patients receiving HCT for SAA.
* To assess the phenotype and epigenetic profile of T-cells in DLI administered to SAA patients post HCT.
* To longitudinally assess CD8 T cell differentiation status in SAA patients using an epigenetic atlas of human CD8 T cell differentiation.
* To examine the effector functions and proliferative capacity of CD8 T cells isolated from SAA patients before and after DLI.
* Quantify donor derived Treg cells at different time points in patients received HCT.
* Determine Treg activation status at different stages after HCT.
* Are specific features of the DLI product associated with particular immune repertoire profiles post-transplant?
* How does the diversity and functional profile of the DLI product alter the response to pathogens in the recipient?
* Do baseline features of the recipient's innate and adaptive immune cells correlate with post-transplant immune repertoires and response profiles?
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 21
Key Eligibility Criteria
Disqualifiers:Fanconi Anemia, Dyskeratosis Congenita, MDS, Others
Must Not Be Taking:Alemtuzumab, ATG
21 Participants Needed
This clinical trial tests next generation sequencing (NGS) for the detection of precursor features of pre-myeloid cancers and bone marrow failure syndromes. NGS is a procedure that looks at relevant cancer associated genes and what they do. Finding genetic markers for pre-malignant conditions may help identify patients who are at risk of pre-myeloid cancers and bone marrow failure syndromes and lead to earlier intervention.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Key Eligibility Criteria
Disqualifiers:Under 18 Years
2000 Participants Needed
HSCT for Leukemia and Lymphoma
Minneapolis, Minnesota
This is a Phase II study of allogeneic hematopoietic stem cell transplant (HCT) using a myeloablative preparative regimen (of either total body irradiation (TBI); or, fludarabine/busulfan for patients unable to receive further radiation). followed by a post-transplant graft-versus-host disease (GVHD) prophylaxis regimen of post-transplant cyclophosphamide (PTCy), tacrolimus (Tac), and mycophenolate mofetil (MMF).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 60
Key Eligibility Criteria
Disqualifiers:Active CNS Malignancy, Uncontrolled Infection, Others
300 Participants Needed
TCD HCT for Fanconi Anemia
Minneapolis, Minnesota
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:< 65
Key Eligibility Criteria
Disqualifiers:Pregnancy, Uncontrolled Infection, Malignant Cancer, Others
48 Participants Needed
Gene Therapy for Fanconi Anemia
Minneapolis, Minnesota
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A).
Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:1+
Key Eligibility Criteria
Disqualifiers:HLA-identical Donor, Myelodysplastic Syndrome, Leukemia, Others
Must Not Be Taking:Androgens, Investigational Therapy
5 Participants Needed
UCB Transplant After Non-Myeloablative Prep for Blood Cancers
Minneapolis, Minnesota
This is a phase II trial using a non-myeloablative cyclophosphamide/ fludarabine/total body irradiation (TBI) preparative regimen with modifications based on factors including diagnosis, disease status, and prior treatment. Single or double unit selected according to current University of Minnesota umbilical cord blood graft selection algorithm.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:< 75
Key Eligibility Criteria
Disqualifiers:Pregnancy, Active Infection, HIV, Others
16 Participants Needed
Deoxycytidine + Deoxythymidine for Telomere Biology Disorder
Boston, Massachusetts
The goal of this clinical trial is to learn if a combination therapy of deoxycytidine (dC) plus deoxythymidine (dT) is safe in patients with telomere biology disorders. The main questions it aims to answer are:
* Is the therapy safe with tolerable side effects in patients with telomere biology disorders?
* Are problems with the bone marrow or blood or lungs changed after 6 months of dC+dT treatment in patients with telomere biology disorders?
Participants will:
* Take study drug by mouth three times daily for 24 weeks
* Make approximately 2 visits to Boston Children's Hospital during the 24 weeks: once at the beginning of treatment and once at the end of treatment.
* Go to a lab for a blood draw an additional 6 times during treatment.
* Have 9 phone calls with a research nurse, including one 4 weeks after treatment ends.
* Keep a diary to track doses of study drug that were taken or missed.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:1 - 70
Key Eligibility Criteria
Disqualifiers:Severe Aplastic Anemia, Chronic Diarrhea, Kidney Disease, High-risk Myelodysplastic Syndrome, Others
Must Not Be Taking:Androgens, Growth Factors
36 Participants Needed
Decitabine + Filgrastim for Acute Myeloid Leukemia
Boston, Massachusetts
The purpose of this study is to examine if it is feasible to administer decitabine and filgrastim after allogenic hematopoietic stem cell transplant (HCT) in children and young adults with myelodysplastic syndrome, acute myeloid leukemia and related myeloid disorders, and if the treatment is effective in preventing relapse after HCT.
The names of the study drugs involved in this study are:
* Decitabine (a nucleoside metabolic inhibitor)
* Filgrastim (a recombinant granulocyte colony-stimulating factor (G-CSF)
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:1 - 39
Key Eligibility Criteria
Disqualifiers:Uncontrolled Illness, FLT3/ITD Mutations, Others
37 Participants Needed
Bone Marrow/Kidney Transplant for Blood Disorders & Chronic Kidney Disease
Boston, Massachusetts
The main purpose of this study is to examine the outcome of a combined bone marrow and kidney transplant from a partially matched related (haploidentical or "haplo") donor. This is a pilot study, you are being asked to participate because you have a blood disorder and kidney disease. The aim of the combined transplant is to treat both your underlying blood disorder and kidney disease. We expect to have about 10 people participate in this study.
Additionally, because the same person who is donating the kidney will also be donating the bone marrow, there may be a smaller chance of kidney rejection and less need for long-term use of anti-rejection drugs.
Traditionally, very strong cancer treatment drugs (chemotherapy) and radiation are used to prepare a subject's body for bone marrow transplant. This is associated with a high risk for serious complications, even in subjects without kidney disease. This therapy can be toxic to the liver, lungs, mucous membranes, and intestines. Additionally, it is believed that standard therapy may be associated with a higher risk of a complication called graft versus host disease (GVHD) where the new donor cells attack the recipient's normal body. Recently, less intense chemotherapy and radiation regimens have been employed (these are called reduced intensity regimens) which cause less injury and GVHD to patients, and thus, have allowed older and less healthy patients to undergo bone marrow transplant. In this study, a reduced intensity regimen of chemotherapy and radiation will be used with the intent of producing fewer toxicities than standard therapy.
Typical therapy following a standard kidney transplant includes multiple lifelong medications that aim to prevent the recipient's body from attacking or rejecting the donated kidney. These are called immunosuppressant drugs and they work by "quieting" the recipient's immune system to allow the donated kidney to function properly. One goal in our study is to decrease the duration you will need to be on immunosuppressant drugs following your kidney transplant as the bone marrow transplant will provide you with the donor's immune system which should not attack the donor kidney.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Key Eligibility Criteria
Disqualifiers:Active Infection, HIV, HCV, Others
10 Participants Needed
Haploidentical Stem Cell Transplant for Blood Diseases
St. Petersburg, Florida
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:0 - 21
Key Eligibility Criteria
Disqualifiers:Pregnancy, HIV, Active Infections, Others
17 Participants Needed
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Frequently Asked Questions
How much do Bone Marrow Failure Disorders clinical trials pay?
Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.
How do Bone Marrow Failure Disorders clinical trials work?
After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Bone Marrow Failure Disorders trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Bone Marrow Failure Disorders is 12 months.
How do I participate in a study as a "healthy volunteer"?
Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.
What does the "phase" of a clinical trial mean?
The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.
Do I need to be insured to participate in a Bone Marrow Failure Disorders medical study?
Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.
What are the newest Bone Marrow Failure Disorders clinical trials?
Most recently, we added Pacritinib for Myelodysplastic Syndrome, Deoxycytidine + Deoxythymidine for Telomere Biology Disorder and Methadone for Pain Management in Stem Cell Transplants to the Power online platform.
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