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93 Sickle-cell disease Trials Near You
Power is an online platform that helps thousands of Sickle-cell Disease 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 PowerRed Blood Cell Exchange for Sickle Cell Disease
Columbus, Ohio
The SCD-CARRE trial is a Phase 3, prospective, randomized, multicenter, controlled, parallel two-arm study aimed to determine if automated exchange blood transfusion and standard of care administered to high mortality risk adult SCD patients reduces the total number of episodes of clinical worsening of SCD requiring acute health care encounters (non-elective infusion center/ER/hospital visits) or resulting in death over 12 months as compared with standard of care.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Key Eligibility Criteria
Disqualifiers:RBC Alloimmunization, Hyper-hemolysis, Severe Transfusion Reaction, Others
173 Participants Needed
Etavopivat for Sickle Cell Disease
Columbus, Ohio
This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo. Which treatment the participants will get is decided by chance. Etavopivat is a new medicine and is currently being tested in other studies in addition to this one. The study will last for about 2 years.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:12+
Key Eligibility Criteria
Disqualifiers:More Than 15 VOCs, Chronic Transfusion, Hepatic Dysfunction, Severe Renal Dysfunction, Others
Must Be Taking:Antimalarial Prophylaxis
408 Participants Needed
Etavopivat for Sickle Cell Disease
Columbus, Ohio
This trial is testing a drug called etavopivat to see if it can help people with sickle cell disease. The drug aims to improve blood health and reduce pain by making red blood cells work better. The study includes patients aged 12 to 65.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12 - 65
Key Eligibility Criteria
Disqualifiers:HIV, Hepatitis B/C, Renal Dysfunction, Others
Must Not Be Taking:Voxelotor, Erythropoietin, CYP3A4/5 Inducers
450 Participants Needed
Transplant Method for Sickle Cell Disease
Columbus, Ohio
This trial uses a combination of drugs and mild radiation to help children with sickle cell disease receive treatment from a sibling. It aims to reduce side effects while maintaining high cure rates. The focus is on children who need a safer treatment option.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:2 - 25
Key Eligibility Criteria
Disqualifiers:HIV, Hepatitis B/C, Pregnancy, Others
100 Participants Needed
Gene Therapy for Sickle Cell Disease
Columbus, Ohio
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:12 - 40
Key Eligibility Criteria
Disqualifiers:Malignancy, Immunodeficiency, Infection, Pregnancy, Others
15 Participants Needed
mHealth App + Booklets for Sickle Cell Disease
Columbus, Ohio
In a hybrid type I effectiveness-implementation trial, our three-center research teams aim to examine whether empowering adults with sickle cell disease (SCD) with patient-facing SCD-specific guidelines through an mHealth application with booklets will decrease acute healthcare utilization and be cost-effective over booklets with the guidelines alone. Our team, head will test our hypotheses with the following aims: Aim 1: evaluate the effectiveness of the patient-facing guidelines mHealth app + booklet intervention to decrease acute healthcare utilization (hospitalizations, emergency room visits, and day hospital visits) in adults with SCD over the standard care in a randomized controlled trial, Aim 2: evaluate the implementation outcomes of the mHealth app + booklet using the capability, opportunity, and motivation-behavior (COM-B) and reach, effectiveness, adoption, implementation, and maintenance (RE-AIM) frameworks, and Aim 3: evaluate the cost-effectiveness of patient-facing mHealth app + booklets vs. standard care in adults with SCD. is hybrid effectiveness-implementation trial design, according to the COM-B and RE-AIM frameworks with a mixed-methods approach, will give valuable insights into the effects, facilitators, and barriers to the implementation that will influence the effects of the patient-facing guidelines intervention.
No Placebo Group
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Unphased
Key Eligibility Criteria
Disqualifiers:Non-English Speakers, Under 18, Others
287 Participants Needed
Video Directly Observed Therapy for Sickle Cell Disease
Columbus, Ohio
This study is for caregivers of young children with sickle cell disease and adolescents with sickle cell disease who are currently prescribed hydroxyurea and are receiving care at one of the study sites. The study will assess retention and engagement during a pilot randomized control trial comparing video directly observed therapy (VDOT) to attention control. We also hope to understand more about patient and family preferences longer-term adherence monitoring and intervention.
Participants will use an electronic adherence monitor (provided by the study team) to measure how often they are taking their hydroxyurea. Participants will also be asked to complete questionnaires throughout the study period to provide information about their expectations for, experience with, and satisfaction with the study materials.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:11+
Key Eligibility Criteria
Disqualifiers:Previous VDOT Study, Others
Must Be Taking:Hydroxyurea
60 Participants Needed
Arginine Therapy for Sickle Cell Disease
Columbus, Ohio
The trial of IV arginine therapy in children with Vaso-occlusive painful episodes (VOE) in sickle cell disease (SCD) is designed to further knowledge on efficacy and safety of the therapy.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Age:3 - 21
Key Eligibility Criteria
Disqualifiers:Not Listed
360 Participants Needed
EDIT-301 for Sickle Cell Disease
Columbus, Ohio
This trial is testing a new treatment called EDIT-301 for people aged 12 to 50 with severe sickle cell disease. The treatment uses the patient's own stem cells, which are changed in a lab and then put back into their body to help fight the disease.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:12 - 50
Key Eligibility Criteria
Disqualifiers:HLA-matched Donor, Prior HSCT, Vasculopathy, Malignancy, Others
Must Not Be Taking:Hydroxyurea, Voxelotor, Crizanlizumab, L-glutamine
45 Participants Needed
Donor Matching Strategies for Blood Cancer Transplants
Columbus, Ohio
The purpose of this study is to determine if a search strategy of searching for an HLA-matched unrelated donor for allogeneic transplantation if possible then an alternative donor if an HLA-matched unrelated donor is not available versus proceeding directly to an alternative donor transplant will result in better survival for allogeneic transplant recipients within 2 years after study enrollment.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Key Eligibility Criteria
Disqualifiers:Not Listed
1753 Participants Needed
SCThrive for Sickle Cell Anemia
Cincinnati, Ohio
The goal of this clinical trial is to evaluate the impact of SCThrive (a behavioral self-management intervention) on patient activation, self-management behaviors, daily functioning, and emergency room visits in 260 adolescents and young adults with sickle cell disease (SCD) ages 13-21 receiving care at 1 of 4 pediatric SCD clinics.
The main question\[s\]it aims to answer are:
* Does SCThrive improve patient activation?
* Does SCThrive improve self-management behaviors, daily functioning, and decrease emergency room visits?
* Are any improvements maintained 3 months after treatment?
Participants will complete self-management related surveys before, after, and 3 months following their participation in an 8- week, virtual group intervention with an accompanying mobile app (SCThrive).
Researchers will compare outcomes for participants who receive SCThrive and participants who receive uniform standard care (SCHealthED which = standard of care plus SCD educational text messages) to see if there are differences in patient activation, self-management behaviors, daily functioning, and emergency room visits.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:13 - 21
Key Eligibility Criteria
Disqualifiers:Chronic Disease, Non-English, Cognitive Disorder
310 Participants Needed
Losartan for Sickle Cell Disease
Cincinnati, Ohio
This trial is testing losartan, a blood pressure medication, in patients aged 6 and older with Sickle Cell Disease. The goal is to see if it can safely reduce fluid buildup in the body over time.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:6+
Key Eligibility Criteria
Disqualifiers:Chronic Transfusion, Congenital Heart Disease, Others
Must Be Taking:Sickle Cell Therapies
24 Participants Needed
Hydroxyurea for Sickle Cell Anemia
Cincinnati, Ohio
This study will 1) Evaluate the prevalence of elevated (conditional or abnormal) transcranial Doppler (TCD) velocities in a cross-sectional analysis of children with Sickle Cell Anemia (SCA) living in Tanzania; 2) Obtain longitudinal data on TCD velocities in this population; and 3) Measure the effects of hydroxyurea therapy on TCD velocities and associated primary stroke risk.
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 2
Age:2 - 16
Key Eligibility Criteria
Disqualifiers:Febrile Illness, Hospitalization, Transfusion, Others
Must Be Taking:Hydroxyurea
202 Participants Needed
Hydroxyurea for Sickle Cell Anemia
Cincinnati, Ohio
This trial studies how to best use hydroxyurea, a medication that helps blood cells stay round and flexible, in children with sickle cell anemia. It aims to find the right dose for each child and monitor their health. The study also looks at ways to check if children are taking their medication regularly. Hydroxyurea has been used for over 25 years to treat sickle cell anemia, showing efficacy in both adults and children.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:6 - 21
Key Eligibility Criteria
Disqualifiers:Family Unwillingness
Must Be Taking:Hydroxyurea
150 Participants Needed
Integrative Training for Sickle Cell Disease Pain
Cincinnati, Ohio
This research aims to answer the question: does a group training program specifically for teens with chronic sickle cell disease (SCD) pain that teaches skills to strengthen the mind and body help improve everyday functioning and reduce pain symptoms?
The program will be tailored to address challenges related to frequent or chronic sickle cell pain and may improve participants' physical and emotional health.
The program, called I-STRONG for SCD (Integrative Strong Body and Mind Training for Sickle Cell Disease), may help improve everyday functioning and pain symptoms in teens with chronic pain related to SCD. The research team aims to determine how participants (teens and parents) respond to this program.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:12 - 18
Key Eligibility Criteria
Disqualifiers:Rheumatologic Disorders, Inflammatory Bowel Disease, Others
Must Be Taking:Disease-modifying Treatments
155 Participants Needed
Imatinib for Sickle Cell Anemia
Cincinnati, Ohio
This trial tests a pill used for other diseases on patients with sickle cell disease. The goal is to see if it can lower harmful proteins and particles in their blood. Researchers hope this will reduce pain crises and other complications over several months.
No Placebo Group
Trial Details
Trial Status:Recruiting
Age:18 - 25
Key Eligibility Criteria
Disqualifiers:Not Listed
20 Participants Needed
Etavopivat for Sickle Cell Disease
Cincinnati, Ohio
This trial is testing a new oral medicine called etavopivat in patients with sickle cell disease or thalassemia. The medicine helps red blood cells produce energy more efficiently. The goal is to reduce the need for blood transfusions and increase hemoglobin levels.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:12 - 65
Key Eligibility Criteria
Disqualifiers:Hepatic Dysfunction, HIV, Hepatitis B/C, Others
60 Participants Needed
Minocycline for Cognitive Decline in Sickle Cell Disease
Cincinnati, Ohio
Sickle cell disease (SCD) is a common, inherited blood disorder that primarily affects people of African Ancestry. It has a lot of complications including neurological complications. The neurological complications of SCD are particularly devastating and lead to cognitive decline even in the absence of overt brain injury. In such cases, it is thought that inflammation in the brain maybe partly responsible for the cognitive decline.
The main reasons for this research study are to see 1) how safe and 2) how well minocycline works to try to stop/reverse cognitive decline in people with SCD. People with SCD are at risk for changes in their brain over time that can cause problems with learning, memory, and attention. Part of the reason for this is inflammation within the brain. Minocycline may be able to stop these brain changes by stopping this brain inflammation.
Minocycline is a second-generation tetracycline antibiotic that has been shown to both inhibit neuroinflammation and improve cognitive function in a variety of neurodegenerative and psychiatric disorders but has not yet been studied in SCD. We are proposing here, a pilot double-blinded, randomized controlled trial to examine the tolerability and early efficacy of minocycline in adults with SCD at two dosing regimens (200 mg and 300 mg daily) versus placebo over one year. Participants will undergo a neuropsychological exam using the NIH Toolbox Cognition Battery at both study enrollment and exit (after one year) to assess for changes/stability of cognition. Participants will receive monthly phone calls/text messages to assess for adverse events and will be seen every three months for pill counts and routine laboratory monitoring. The primary outcome will be a comparison of adverse events across the two dosing strategies versus placebo. Early evidence for cognitive benefit will also be assessed from the results of the NIH Toolbox.
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 1
Key Eligibility Criteria
Disqualifiers:Other SCD Genotypes, Stroke, Autoimmune, Others
Must Be Taking:Hydroxyurea
30 Participants Needed
Etavopivat for Sickle Cell Anemia
Cincinnati, Ohio
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Key Eligibility Criteria
Disqualifiers:Other Disorders, Treatment Withdrawal, Others
Must Be Taking:Hydroxyurea, Crizanlizumab, Endari
325 Participants Needed
Medical Cannabis for Chronic Pain
Sandusky, Ohio
This trial will investigate if medical cannabis can effectively reduce pain and improve quality of life for patients with chronic conditions. The study will gather data through an online questionnaire about patients' use of cannabis and its effects. Medical cannabis interacts with the body's natural system to help manage pain and other symptoms. Medical cannabis has been increasingly studied and used as an alternative treatment for managing chronic pain, with numerous studies supporting its potential benefits.
Stay on current meds
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:7+
Key Eligibility Criteria
Disqualifiers:Pregnancy, Breastfeeding, Suicidality, Psychosis, Others
200000 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
"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 was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."
ID
Pancreatic Cancer PatientAge: 40
"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
"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
This study aims to enroll 58 pre-adolescent (\<13 years) pediatric participants with sickle cell disease (SCD) who have a pre-adolescent sibling bone marrow donor. All participants will go through a pre-transplant evaluation to find out if there are health problems that will keep them from being able to receive the transplant. It usually takes 2 to 3 months to complete the pre-transplant evaluation and make the arrangements for the transplant. Once they are found to be eligible for transplant, participants will be admitted to the hospital and will start transplant conditioning. Conditioning is the chemotherapy and other medicines given to prepare them to receive donor cells. It prevents the immune system from rejecting donor cells. Conditioning will start 21 days before transplant. Once they complete conditioning, participants will receive the bone marrow transplant. After the transplant, participants will stay in the hospital for 4-6 weeks. After they leave the hospital, participants will be followed closely in the clinic. Outpatient treatment and frequent clinic visits usually last 6 to 12 months. Routine medical care includes at least a yearly examination for many years after transplant by doctors and nurses familiar with sickle cell disease and transplant. The researchers will collect and study information about participants for 2 years after transplant.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:2 - 13
Key Eligibility Criteria
Disqualifiers:Liver Fibrosis, Lung Disease, Renal Dysfunction, Cardiac Dysfunction, Neurologic Impairment, Others
43 Participants Needed
Mitapivat for Sickle Cell Disease
Cleveland, Ohio
This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of hemoglobin in the blood and to reduce or prevent the occurrence of sickle cell pain crises. In addition, the long-term effect of mitapivat on efficacy and safety will be explored in an open-label extension portion.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:16+
Key Eligibility Criteria
Disqualifiers:Pregnant, Transfusions, Liver, Kidney, Others
Must Be Taking:Hydroxyurea
286 Participants Needed
BEAM-101 for Sickle Cell Disease
Cleveland, Ohio
This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18 - 35
Key Eligibility Criteria
Disqualifiers:Moyamoya Syndrome, Stroke, Others
Must Be Taking:Hydroxyurea
15 Participants Needed
Music Therapy for Sickle Cell Disease
Cleveland, Ohio
This is a multi-site, multi-visit feasibility RCT of music therapy (MT) among adolescent and adult patients (aged 14 and older) with sickle cell disease (SCD).
Subjects will be randomized into one of three groups, either (1) 6 visits of in- person MT (InMT:); (2) 1 visit of in-person MT and 5 visits of virtual MT (HybMT); or (3) 1 visit of in-person health education and 5 visits of virtual health education (HybHE).
Cohorts of 15 participants (10 at site 1 and 5 site 2) will be recruited each quarter for 6 quarters to reach 90 participants. Cohorts will maintain a semi-structured recruitment, consenting, assessment, and intervention schedule.
The primary objective of the study is to examine the feasibility of study. This is defined by 6 metrics: (1) completeness of data collection, (2) participant screening, (3) participant recruitment, (4) participant retention, (5) Individual attendance and (6) Home practice. A final determination of "feasibility" for the study will be met if any 4 of the 6 metrics described above are met.
This study will also include a secondary objective of conducting qualitative interviews to assess feasibility of implementation.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:14+
Key Eligibility Criteria
Disqualifiers:Visual, Hearing, Cognitive Impairment, Others
90 Participants Needed
L-Citrulline for Sickle Cell Disease
Detroit, Michigan
This study is being done to learn more about a possible new treatment for pain episodes (called vaso-occlusive crises or VOCs) in children, teens, and young adults with sickle cell disease (SCD). The study will include about 120 participants between the ages of 6 and 21 who come to the emergency department (ED) with a VOC.
A VOC is a painful episode that happens with no clear cause and no signs of infection or major problems with organs like the liver or kidneys.
Before joining the study, patients and their families may be asked to learn about it and give permission (called consent or assent) while at a regular clinic visit. If that hasn't happened yet, the consent/assent process will happen at the emergency department when the patient comes in for care. If the patient meets all the study requirements, they can join the treatment part of the study.
Participants will be randomly assigned (like flipping a coin) to receive either:
L-citrulline, the study drug, or A placebo, which looks the same but has no active ingredients.
Everyone has an equal chance of getting either one. The study drug is given through an IV. It starts with one larger dose, followed by a steady infusion for up to 12 hours.
All patients in the study will still receive the usual pain treatment (called standard of care), which may include opioids. However, some patients may need fewer opioids if the study treatment helps with their pain.
If any medicines are not allowed during the study, the doctor will explain this during the consent process.
Patients can go home once:
Their pain is controlled with oral (by mouth) pain medicine, They're eating and drinking well, and They've been given a personal pain management plan to use at home.
After leaving the hospital, the study team will follow up with patients by phone about 2 days later (within a 12-hour window), again around Day 7, and again around Day 30 to check how they're doing.
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 3
Age:6 - 21
Key Eligibility Criteria
Disqualifiers:Sepsis, Stroke, Severe Anemia, Others
Must Not Be Taking:Steroids, Nitrates, Sildenafil, Others
120 Participants Needed
Epeleuton for Sickle Cell Anemia
Detroit, Michigan
To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged ≥18 years.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Key Eligibility Criteria
Disqualifiers:Blood Transfusions, Pregnancy, Others
Must Be Taking:Hydroxyurea
30 Participants Needed
Stem Cell Transplant for Sickle Cell Disease
Detroit, Michigan
This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell transplantation using BIVV003.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18 - 40
Key Eligibility Criteria
Disqualifiers:Organ Dysfunction, Active Malignancy, HIV, Others
Must Not Be Taking:Prohibited Medications
8 Participants Needed
Crovalimab for Sickle Cell Disease
Detroit, Michigan
This trial is testing crovalimab to see if it can safely and effectively prevent painful episodes in people with Sickle Cell Disease by stopping the immune system from causing blood vessel blockages.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:12 - 55
Key Eligibility Criteria
Disqualifiers:Stem Cell Transplant, Chronic Transfusion, HIV, Others
90 Participants Needed
Bone Marrow Transplant for Sickle Cell Disease
Pittsburgh, Pennsylvania
This trial uses stem cells from mismatched donors with certain immune cells removed to treat patients with severe blood disorders who lack a perfect donor match. The approach aims to replace damaged cells, reduce complications, and support recovery with additional immune cells. A new technique has been developed to improve the treatment process.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:5 - 40
Key Eligibility Criteria
Disqualifiers:Pregnancy, CNS Event, Infections, Others
Must Be Taking:Hydroxyurea
5 Participants Needed
BMS-986470 for Sickle Cell Disease
Pittsburgh, Pennsylvania
The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Key Eligibility Criteria
Disqualifiers:Severe VOCs, Acute Chest Syndrome, Others
Must Be Taking:Hydroxyurea
184 Participants Needed
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Frequently Asked Questions
How much do Sickle-cell disease 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 Sickle-cell disease 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 Sickle-cell disease 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 Sickle-cell disease 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 Sickle-cell disease 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 Sickle-cell disease clinical trials?
Most recently, we added Hemanext ONE System for Sickle Cell Anemia, L-Citrulline for Sickle Cell Disease and CSL889 for Sickle Cell Disease During Pain Crisis to the Power online platform.
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