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15 Fanconi Anemia Trials
Power is an online platform that helps thousands of Fanconi Anemia 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.
Chemotherapy + Stem Cell Transplant for Fanconi Anemia
Cincinnati, OhioThe purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:3+
Sex:All
Key Eligibility Criteria
Disqualifiers:Active CNS Leukemia, HIV, Others
70 Participants Needed
Quercetin for Squamous Cell Cancer in Fanconi Anemia
Cincinnati, OhioFanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leukemia (AML) and squamous cell carcinoma (SCC). Improved transplant outcomes are modifying the natural history of Fanconi Anemia. Improved transplant survival, no radiation exposure, and almost no GVHD increases the importance of addressing later SCC even further. The investigators hypothesize that quercetin will prevent or delay the development of SCC and associated complications, there by ameliorating or delaying the need for potentially lethal treatment with chemotherapy and/or radiation therapy for the same.
Funding Source - FDA Office of Orphan Products Development (OOPD)
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:2+
Sex:All
48 Participants Needed
Abatacept for Graft-versus-Host Disease Prevention
Chicago, IllinoisThis trial will see if extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will prevent acute and chronic graft-versus-host disease (GVHD) in children and adolescents receiving unrelated donor (URD) hematopoietic stem cell transplantation (HSCT), without compromising their engraftment or reconstitution of protective immunity to infection.
The study will enroll 30 pediatric patients with serious non-malignant hematologic diseases (NMHD) undergoing URD HSCT. The trial will include patients with 7/8 donors and those with 8/8 (matched) donors. All participants will receive 8 doses of abatacept. Recruitment is expected to last for about 2 years and participants will be followed for up to 3 years.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:< 20
Sex:All
30 Participants Needed
Eltrombopag for Fanconi Anemia
Bethesda, MarylandBackground:
Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly. These people may need blood transfusions for anemia (low red blood cells) or low platelet counts or bleeding. Researchers want to see if a new drug will help people with this disease.
Objective:
To find out if a new drug, eltrombopag, is effective in people with Fanconi anemia. To know how long the drug needs to be given to improve blood counts.
Eligibility:
People at least 6 years old with Fanconi anemia with reduced blood cell counts.
Design:
Participants will be screened with blood and urine tests. They will repeat this before starting to take the study drug.
Participants will take eltrombopag pills by mouth once a day for 24 weeks. They will be monitored closely for side effects.
Participants will have blood tests every 2 weeks while on eltrombopag.
Participants will visit NIH 3 months and 6 months after starting eltrombopag. At these visits, participants will:
Answer questions about their medical history, how they are feeling, and their quality of life
Have a physical exam
Have blood and urine tests
Have a bone marrow sample taken by needle from the hip. The area will be numbed.
If participants blood cell counts improve, they might join the extended access part of the study. They will continue taking eltrombopag for 3 years and sign a different consent.
After 24 weeks of treatment, if there is no improvement in blood cell counts, participants will stop taking eltrombopag. They will return for an optional follow-up visit that repeats the study visits....
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:6 - 99
Sex:All
25 Participants Needed
CD45RA Depleted Stem Cell Addback for Leukemia
Philadelphia, PennsylvaniaThe 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
Sex:All
100 Participants Needed
FP-045 for Fanconi Anemia
New York, New YorkThis is a multi-center, Phase 1/2 study to determine the Optimal Biologic Dose (OBD) and to evaluate the safety, tolerability, PK, and preliminary activity of FP 045 when administered orally in young adult/adolescent and pediatric patients with Fanconi anemia. The study will enroll a total of 4 young adult/adolescent patients and a minimum of 8 and up to 12 pediatric patients with mild-moderate bone marrow failure who have not undergone hematopoietic cell transplant. This makes the total patient number between 12-16 total. Dose escalation will occur individually for each patient, within each age group. Each patient will receive each of 3 dose levels of FP 045 (intra-patient dose escalation), beginning with Dose Level 1, followed by Dose Levels 2 and 3. Each dose level will be administered for 28 days prior to escalation to the next higher dose level for that patient.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:3 - 35
Sex:All
16 Participants Needed
Olaparib for Biliary Tract Cancer
New York, New YorkThis trial studies how well olaparib works in patients with advanced biliary tract cancer and specific DNA repair gene mutations. Olaparib may help stop cancer growth by blocking enzymes needed for cell repair. The trial aims to see if this treatment can improve survival and response rates.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Congestive Heart Failure, Uncontrolled Hypertension, Seizure Disorder, Others
Must Not Be Taking:CYP3A Inhibitors, CYP3A Inducers
36 Participants Needed
Gene Therapy for Fanconi Anemia
Minneapolis, MinnesotaThe 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+
Sex:All
5 Participants Needed
TCD HCT for Fanconi Anemia
Minneapolis, MinnesotaThis 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
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, Uncontrolled Infection, Malignant Cancer, Others
48 Participants Needed
Alcohol Exposure for Oral Cancer Risk
Minneapolis, MinnesotaThis is a minimal risk intervention study where healthy volunteers and individuals with Fanconi anemia will consume a single dose of alcohol and provide primarily non-invasive biological samples at various time points. Biospecimens to be collected include saliva, oral cells collected via mouthwash and cheek brush, and urine. The collection of two blood samples (5 mL each) will be optional and banked for future use.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:18 - 45
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, Alcoholism, Tobacco Use, Others
Must Not Be Taking:Antibiotics, Others
170 Participants Needed
This is an open label, phase II trial in subjects with treatment naïve, metastatic hormone sensitive prostate cancer (mHSPC) with deleterious homologous recombination repair (HRR) alteration(s). These include pathologic alterations in BRCA 1/2, BRIP1, CHEK2, FANCA, PALB2, RAD51B, and/or RAD54L. A total of 64 people will be enrolled to the study.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18+
Sex:Male
Key Eligibility Criteria
Disqualifiers:Neuroendocrine Features, Brain Metastases, Others
Must Not Be Taking:Second Generation ARIs, Chemotherapy
64 Participants Needed
Fluorescent Imaging for Premalignant Oral Lesions
Houston, TexasThis clinical trial studies widefield fluorescence and reflectance imaging, fluorescence spectroscopy, and tissue samples in regularly examining (monitoring) participants at risk for developing oral cancer. All tissue and cells are made of tiny particles. Some of these particles give off small amounts of light. This light is called fluorescence. Fluorescent imaging use instruments that shine different wavelengths (colors) of light in the mouth taking fluorescence pictures through a portable head light or by taking fluorescent and reflectance pictures through a dental microscope using a digital camera. Fluorescent spectroscopy uses a small probe placed gently against the lining of the mouth and the tissue is exposed to small amounts of fluorescent light that is then collected with a special camera and a computer to be analyzed. Checking mouth tissue samples under a microscope may also help detect abnormal cells. Diagnostic procedures, such as fluorescence and reflectance imaging, fluorescence spectroscopy imaging, and tissue samples, may help doctors detect pre-cancer or early cancer when it may be easier to treat.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:18+
Sex:All
338 Participants Needed
Stem Cell Transplant for Leukemia
Houston, TexasThis 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
Trial Phase:Unphased
Age:< 55
Sex:All
47 Participants Needed
Pre-Surgery Niraparib for Prostate Cancer
Sacramento, CaliforniaThis phase II trial studies how well niraparib, when given before surgery, works in treating patients with high risk prostate cancer that has not spread to other parts of the body (localized) and alterations in deoxyribonucleic acid (DNA) repair pathways. Niraparib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18+
Sex:Male
Key Eligibility Criteria
Disqualifiers:Prior Prostate Treatment, Severe Infection, Others
Must Not Be Taking:PARP Inhibitors, Antiandrogens
30 Participants Needed
Stem Cell Transplant + JSP191 for Fanconi Anemia
Stanford, CaliforniaThe objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor after using an experimental antibody treatment called JSP-191 as a part of conditioning. This experimental treatment will hopefully cause fewer side effects than chemotherapy (the current standard of care method).
Participants will be administered the conditioning regimen, are assessed until they receive the depleted stem cell infusion, and will be followed for up to 2 years after the cell infusion.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:2+
Sex:All
Key Eligibility Criteria
Disqualifiers:HIV, Active Infections, Malignancies, Others
Must Not Be Taking:Investigational Agents
12 Participants Needed
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Frequently Asked Questions
How much do Fanconi Anemia 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 Fanconi Anemia 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 Fanconi Anemia 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 Fanconi Anemia 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 Fanconi Anemia 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 Fanconi Anemia clinical trials ?
Most recently, we added CD45RA Depleted Stem Cell Addback for Leukemia, Niraparib + Abiraterone Acetate + Prednisone for Prostate Cancer and Stem Cell Transplant for Leukemia to the Power online platform.