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49 Bone Marrow Failure Trials Near You
Power is an online platform that helps thousands of Bone Marrow Failure 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 PowerHaploidentical 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
Stem Cell Mobilization with Plerixafor for Abnormal Endometrium
Orange, Connecticut
This trial tests if a new treatment can help women with uterine lining issues by moving their own stem cells into their blood to repair the uterus. It aims to improve pregnancy chances for women with specific reproductive challenges.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Early Phase 1
Age:18 - 40
Sex:Female
Key Eligibility Criteria
Disqualifiers:Hydrosalpinx, Endometriosis, Diminished Ovarian Reserve, Others
30 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
Stem Cell Transplant for Aplastic Anemia
Minneapolis, Minnesota
Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who have bone marrow failure characterized by a requirement for red blood cell and platelet transfusions. Three different preparative regimens are included based on disease and donor type.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:0 - 70
Key Eligibility Criteria
Disqualifiers:Not Listed
57 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
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
Hematopoietic Cell Transplant for Aplastic Anemia
Minneapolis, Minnesota
A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT) utilizing population pharmacokinetic (popPK)-guided individual dosing of pre-transplant conditioning and differential dosing of low dose total body irradiation based on age, presence of myelodysplasia and/or clonal hematopoiesis.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:0 - 75
Key Eligibility Criteria
Disqualifiers:Pregnancy, Uncontrolled Infection, Advanced MDS, Others
60 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
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
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 peripheral blood counts improved in patients with telomere biology disorders who have cytopenias?
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:Not Yet Recruiting
Trial Phase:Phase 1
Age:1 - 60
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
Haploidentical Stem Cell Transplant for Blood Diseases
Saint 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
Mucositis is a normal side effect of stem cell transplant which happens as a result of chemotherapy being given prior to a new donor cell infusion (bone marrow transplant). The chemotherapy will kill cancer cells, but good cells, such as those in the mouth, are killed too. The mouth cells going away causes the areas in the mouth to be blistered, irritated, sore, and extremely painful. Pain medication (usually morphine or hydromorphone if allergic to morphine) are given when oral blisters are seen or felt by patient in patient's mouth. However, one pain medication given through a vein in the patient may or may not be effective and providers are often challenged with providing good pain control while waiting for the new donor cells to grow, which will then heal the mouth. This is a period of waiting that is 6-8 weeks.
The investigators know that methadone, a second pain medication, may decrease pain in a different way than morphine. This is because methadone works in a different way in the brain than morphine. By giving these pain medicines together, the hope of the study is to show decreased pain while waiting for new cells to grow.
The goal of this clinical trial is to hope to learn whether adding methadone (second pain medication) to the current pain medication which is morphine alone (all patients will receive this pain medication) will help reduce the pain experience of participant. Current treatment of morphine alone is sometimes not entirely effective and so any improvement of pain while waiting for new cells to grow is one of the goals of this study. If methadone is effective in decreasing pain, then patients may benefit in the future from using these two medications up front when getting a transplant.
Participant in this study between 6-18 years of age and is needing a stem cell transplant for a disease that can potentially be cured by transplantation.
Participant in this study is receiving chemotherapy and/or radiation conditioning that can cause mucositis. Participants are being asked to participate in this study because participants meet criteria to receive methadone that may or may not reduce pain experience versus just being given morphine alone, which is what all patients are given when the participants have mucositis.
The main goal of the study is to see if less opioid (pain medication) when methadone is added in comparison to participant who uses PCA only. The investigators also want to learn if patient's overall function is improved if given methadone. Another goal would be to see the number of TPN days the participant received and if the participant who was given methadone began to eat sooner. Other smaller goals include learning about side effects of methadone, and if the hospital stay is less for those who receive the study medication.
This medication will be given at Children's Medical Center of Dallas while participant is admitted for the stem cell transplant. There is no sponsor that is funding the study and this drug will be given free of charge in exchange for participation in the study
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 3
Age:6 - 18
Key Eligibility Criteria
Disqualifiers:QT Prolongation, Neurological, Psychiatric, Others
Must Be Taking:Opioids
60 Participants Needed
CK0801 for Bone Marrow Disease
Houston, Texas
The goal of this clinical research study is to determine whether it is safe and practical to give CK0801 (a Cord blood-derived T-regulatory cell product) to patients with bone marrow failure syndrome. Researchers want to determine the highest possible dose that is safe to be given. Researchers also want to learn if CK0801 may improve the symptoms of bone marrow failure syndrome.
Patients enrolled in this study will all have been diagnosed with treatment refractory bone marrow failure syndrome (which includes aplastic anemia, myelodysplastic syndrome, or myelofibrosis). Participants eligible to participate in this study are unable or unwilling to be treated with standard therapy or have failed standard therapy.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:HIV, Uncontrolled Infection, Pregnancy, Others
18 Participants Needed
Veto Cell Therapy for Leukemia and Lymphoma
Houston, Texas
This phase I/II trial studies how well cytokine-treated veto cells work in treating patients with hematologic malignancies following stem cell transplant. Giving chemotherapy and total-body irradiation before a stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Cytokine-treated veto cells may help the transplanted donor cells to develop and grow in recipients without causing graft-versus-host-disease (GVHD - when transplanted donor tissue attacks the tissues of the recipient's body).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:12 - 75
Key Eligibility Criteria
Disqualifiers:HIV, Uncontrolled Infection, CNS Malignancy, Others
24 Participants Needed
This phase II trial studies methylprednisolone, horse anti-thymocyte globulin, cyclosporine, filgrastim, and/or pegfilgrastim or pegfilgrastim biosimilar in treating patients with aplastic anemia or low or intermediate-risk myelodysplastic syndrome. Horse anti-thymocyte globulin is made from horse blood and targets immune cells known as T-lymphocytes. Since T-lymphocytes are believed to be involved in causing low blood counts in aplastic anemia and in some cases of myelodysplastic syndromes, killing these cells may help treat the disease. Methylprednisolone and cyclosporine work to suppress immune cells called lymphocytes. This may help to improve low blood counts in aplastic anemia and myelodysplastic syndromes. Filgrastim and pegfilgrastim are designed to cause white blood cells to grow. This may help to fight infections and help improve the white blood cell count. Giving methylprednisolone and horse anti-thymocyte globulin together with cyclosporine, filgrastim, and/or pegfilgrastim may be an effective treatment for patients with aplastic anemia or myelodysplastic syndrome.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Pregnancy, HIV, Uncontrolled Illness, Others
140 Participants Needed
Stem Cell Transplant for Leukemia
Houston, Texas
This trial tests a special stem cell transplant for patients with serious conditions. It removes harmful immune cells from the donor's blood to lower the risk of complications, potentially reducing the need for strong medications afterward.
No Placebo Group
Trial Details
Trial Status:Recruiting
Age:< 55
Key Eligibility Criteria
Disqualifiers:Not Listed
47 Participants Needed
Ruxolitinib for Aplastic Anemia
Seattle, Washington
This phase II trial tests how well a ruxolitinib-based graft versus host disease (GVHD) prevention (prophylaxis) regimen works before, during, and after bone marrow/stem cell transplantation (hematopoietic cell transplantation \[HCT\]) in patients with acquired aplastic anemia. Acquired aplastic anemia (AA) is a condition in which the bone marrow is unable to produce blood cells. Affected patients typically present with infections due to abnormally low number of neutrophils, bleeding due to low platelet count, and/or fatigue due to a lower-than-normal number of red blood cells (anemia). Its incidence varies with age, occurring most frequently in patients aged 2-5 years, 20-25 years, and 55 years and older. Treatment of AA includes either immunosuppressive therapy (IST) or bone marrow/stem cell transplantation (HCT) with first-line therapy in younger adults often being HCT, while adults over 40 still frequently trial IST first due to the morbidity and mortality concerns with HCT. GVHD is a common complication after donor stem cell transplantation, resulting from donor immune cells recognizing recipients' cells and attacking them. Ruxolitinib, a drug in a class of oral medications called JAK inhibitors has been approved for the treatment of acute and chronic GVHD. It has also been shown to decrease GVHD when used in the prevention setting in patients with myelofibrosis. The current study aims to assess whether adding ruxolitinib to a standard GVHD prevention regimen may reduce the risk of Grade II-IV acute and chronic GVHD after bone marrow/stem cell transplantation in older patients with acquired aplastic anemia.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Myocardial Infarction, HIV, Others
Must Be Taking:JAK Inhibitors
20 Participants Needed
Why Other Patients Applied
"I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."
FF
ADHD PatientAge: 31
"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
"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
"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
"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
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We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.
Bask GillCEO at Power
Frequently Asked Questions
How much do Bone Marrow Failure 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 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 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 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 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 clinical trials?
Most recently, we added Methadone for Pain Management in Stem Cell Transplants, Pacritinib for Myelodysplastic Syndrome and Ruxolitinib for Aplastic Anemia to the Power online platform.
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