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Gene Therapy
Gene Therapy for Fanconi Anemia
Phase 1
Waitlist Available
Led By Hans-Peter Kiem
Research Sponsored by Fred Hutchinson Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
FA demonstrated by a positive test for increased sensitivity to chromosomal breakage with mitomycin C or diepoxybutane performed by a Clinical Laboratory Improvement Amendments (CLIA) or College of American Pathologists (CAP) approved laboratory
FA complementation group A as determined by somatic cell hybrids, molecular characterization, western blot analysis, acquisition of mitomycin C resistance after in vitro lentiviral transduction with a vector bearing the complementary deoxyribonucleic acid (cDNA) for Fanconi complementation group A, or other clinically certified method of complementation group analysis
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 15 years
Awards & highlights
Study Summary
This trial will test how safe and effective it is to give patients with Fanconi anemia gene-modified cells. The cells are from the patient's own blood and have been corrected in the lab to have the normal gene.
Who is the study for?
This trial is for patients with Fanconi anemia who have normal bone marrow cytogenetics, adequate organ function, and specific blood count levels. They must not be pregnant or lactating, have acute myeloid leukemia, severe diseases like uncontrolled hypertension or HIV, or a suitable sibling donor available for stem cell transplant.Check my eligibility
What is being tested?
The trial tests the safety and effectiveness of infusing gene-modified blood stem cells into patients with Fanconi anemia. These cells are corrected in the lab to improve blood counts. The process includes bone marrow aspiration and drugs like Plerixafor and Filgrastim.See study design
What are the potential side effects?
Potential side effects may include discomfort from bone marrow aspiration, reactions to medications used during treatment such as Prednisone (like weight gain, mood changes), Methylprednisolone (immune suppression), and general risks associated with stem cell transplantation.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I tested positive for Fanconi anemia with a specific sensitivity test.
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My condition is confirmed to be FA group A by a specific test.
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I am mostly active and can care for myself regardless of my age.
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My bone marrow test shows normal genetics and less than 5% abnormal cells.
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My kidney function is within the normal range.
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My platelet count is at least 20 and can reach 50 with help.
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My liver tests, AST and ALT, are within normal limits.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 15 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 15 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Development of insertional mutagenesis or hematologic malignancy
Development of replication competent lentivirus
Hematological and non-hematological organ toxicity
+1 moreSecondary outcome measures
Demonstrable functional expression by growth of recipient cells in mitomycin C
Detectable levels of transduced cells in blood and marrow
Efficacy of G-CSF and plerixafor mobilization in Fanconi anemia (FA) patients
+3 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: Treatment (hematopoietic stem progenitor cells)Experimental Treatment8 Interventions
STEM CELL MOBILIZATION FOR CELL COLLECTION: Patients receive filgrastim SC BID for up to 6 days (on days 1-6 of mobilization). Patients receive plerixafor SC QD on days 4-6 of mobilization. PBSC count will be checked daily starting on day 4 of mobilization. Patients who have a PBSC count of >= 5 CD34+ cells/mcL will undergo up to 2 apheresis collections on consecutive days.
BONE MARROW HARVEST FOR CELL COLLECTION: Patients with inadequate PBSC counts undergo bone marrow harvest for collection of stem/progenitor cells.
REINFUSION: Patients receive methylprednisolone IV or prednisone PO on days -1 to 7 followed by a rapid taper over approximately 1 week and undergo reinfusion of genetically modified hematopoietic stem/progenitor cells on day 0.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Bone Marrow Aspiration
2011
Completed Phase 2
~1740
Leukapheresis
2016
Completed Phase 2
~690
Prednisone
2014
Completed Phase 4
~2370
Plerixafor
2011
Completed Phase 3
~720
Methylprednisolone
2015
Completed Phase 4
~2280
Filgrastim
2000
Completed Phase 3
~3670
Find a Location
Who is running the clinical trial?
Fred Hutchinson Cancer CenterLead Sponsor
556 Previous Clinical Trials
1,343,265 Total Patients Enrolled
5 Trials studying Fanconi Anemia
148 Patients Enrolled for Fanconi Anemia
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,833 Previous Clinical Trials
47,306,493 Total Patients Enrolled
10 Trials studying Fanconi Anemia
334 Patients Enrolled for Fanconi Anemia
Rocket Pharma LimitedUNKNOWN
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My doctor expects I have less than 2 years to live due to my cancer.I tested positive for Fanconi anemia with a specific sensitivity test.My condition is confirmed to be FA group A by a specific test.My lungs work well enough for the study, with a DLCO over 50%.I do not have significant diseases like uncontrolled HIV, heart issues, or poorly controlled diabetes.I am mostly active and can care for myself regardless of my age.If a family member is a good match to donate stem cells, but it's not possible to collect the cells, a patient may still be included in the study, depending on what the researchers decide.My bone marrow test shows normal genetics and less than 5% abnormal cells.My kidney function is within the normal range.I am not currently being considered for a bone marrow transplant from a sibling.I am not considering this study as a substitute for a stem cell transplant from a sibling.I currently have an active infection.My platelet count is at least 20 and can reach 50 with help.I have been diagnosed with a bone marrow disorder according to WHO standards.My liver tests, AST and ALT, are within normal limits.I have been diagnosed with acute myeloid leukemia.Your hemoglobin level is at least 8 grams per deciliter.You have enough infection-fighting white blood cells in your body.
Research Study Groups:
This trial has the following groups:- Group 1: Treatment (hematopoietic stem progenitor cells)
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Fanconi Anemia Patient Testimony for trial: Trial Name: NCT01331018 — Phase 1
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
For what illnesses is leukapheresis typically employed?
"To alleviate the symptoms of scalp structure, thyroiditis and ulcerative colitis, Leukapheresis can be employed."
Answered by AI
Has the Food and Drug Administration given its stamp of approval to Leukapheresis?
"Due to the preliminary nature of this study, Leukapheresis received a score of 1 as an indicator of its safety. This is because Phase 1 trials generally lack substantial data regarding efficacy and safety."
Answered by AI
How many participants have volunteered to join this medical experiment?
"This investigation is not currently open to recruitment. Initially posted on February 22nd 2012 and recently updated June 21st 2022, it has since been closed off from the public. However, if you are seeking out other trials there are presently 225 studies recruiting participants with Fanconi Anemia and 559 clinical tests that use leukapheresis which are accepting patients."
Answered by AI
Are there any opportunities to join the trial for participants?
"Unfortunately, no more participants are being accepted into this clinical trial. It was initially posted on February 22nd 2012 and last edited six months ago on June 21st 2022. If you're searching for other studies, there currently exists 225 trials actively enrolling patients with fanconi anemia and 559 experiments recruiting those needing leukapheresis."
Answered by AI
Could you mention any prior experiments that have employed Leukapheresis?
"Currently, there exists a considerable quantity of studies investigating Leukapheresis with 144 trials in the final stage. The majority are based in Duarte, California but 20927 locations globally have active trials on this subject."
Answered by AI
Who else is applying?
What site did they apply to?
Fred Hutch/University of Washington Cancer Consortium
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
1
What questions have other patients asked about this trial?
Can I do initial screening here in South Africa, and if I'm approved then travel? Do I stay in a hospital that I have to pay for?
PatientReceived 1 prior treatment
Why did patients apply to this trial?
I was diagnosed with leukemia, currently undergoing chemo that will hopefully cure the cancer. I want to help create better treatments and hopefully further the research into gene therapy.
PatientReceived no prior treatments
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