Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
TERMINATED
PHASE1
3 participants
INTERVENTIONAL
2012-02-22
2024-02-15
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
NCT04069533
Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A
NCT03157804
FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector
NCT03351868
Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
NCT00258427
Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1
NCT02931071
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
STEM CELL MOBILIZATION FOR CELL COLLECTION: Patients receive filgrastim subcutaneously (SC) twice daily (BID) for up to 6 days (on days 1-6 of mobilization). Patients receive plerixafor SC once daily (QD) on days 4-6 of mobilization. Peripheral blood stem cell (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 intravenously (IV) or prednisone orally (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.
After completion of study treatment, patients are followed up periodically for 15 years.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Treatment (hematopoietic stem progenitor cells)
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.
Bone Marrow Aspiration
Undergo bone marrow harvest
Filgrastim
Given SC
Genetically Engineered Hematopoietic Stem Progenitor Cells
Undergo infusion of genetically modified hematopoietic progenitor cell therapy
Laboratory Biomarker Analysis
Correlative studies
Leukapheresis
Undergo leukapheresis
Methylprednisolone
Given IV
Plerixafor
Given SC
Prednisone
Given PO
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Bone Marrow Aspiration
Undergo bone marrow harvest
Filgrastim
Given SC
Genetically Engineered Hematopoietic Stem Progenitor Cells
Undergo infusion of genetically modified hematopoietic progenitor cell therapy
Laboratory Biomarker Analysis
Correlative studies
Leukapheresis
Undergo leukapheresis
Methylprednisolone
Given IV
Plerixafor
Given SC
Prednisone
Given PO
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
* Bone marrow analysis demonstrating normal cytogenetics, and no more than 5% of cells with a single clonal abnormality by fluorescence in situ hybridization (FISH) for myelodysplastic syndrome (MDS) panel within 3 months of stem cell collection
* Signed informed consent by the patient or legally authorized representative
* Absolute neutrophil count \>= 0.5 x 10\^9/L
* Hemoglobin \>= 8 g/dL
* Platelet count \>= 20 x 10\^9/L and able to achieve a platelet count of \>= 50 x 10\^9/L with transfusion support
* Adequate hepatic function with aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 5 x upper limit of normal (ULN)
* Adequate renal function with creatinine (Cre) =\< 1.5; if greater, then glomerular filtration rate (GFR) \> 60 mL/min/1.73 m\^2 as calculated by the Modification of Diet in Renal Disease equation
* Adequate pulmonary function with corrected diffusion capacity of carbon monoxide (DLCO) \> 50% in those for whom this study can be performed
* For subjects \< 17 years of age, Modified Lansky Play-Performance Score of \>= 70%; for subjects 17 and older, Karnofsky score of \>= 70%
Exclusion Criteria
* Myelodysplastic syndrome as defined by World Health Organization (WHO) criteria
* Acute myeloid leukemia as defined by WHO criteria
* Pregnancy or lactation; females of childbearing potential and males who are admitted to the study will be advised that the study procedures and study drugs may be teratogenic, and they will be required to take adequate measures to prevent conception for the duration of the study
* Concurrent enrollment in any other study using an investigational drug
* Physical or emotional status that would prevent informed consent, protocol compliance, or adequate follow-up
* Patients for whom an human leukocyte antigen (HLA) matched sibling donor bone marrow transplant is being actively pursued will not be eligible for study until it is determined that no sibling donor is available or that a stem cell transplant is not feasible during the time the patient might be on study
* No patient will be included in this study as an alternative to a clinically indicated HLA matched sibling donor stem cell transplant
* If an HLA matched sibling donor is identified, but stem cell or marrow collection is not feasible (e.g., donor is in utero, is a newborn from whom cord blood was not collected, or is unable to undergo a donation procedure because of ill health), a patient may be included in the study at the discretion of the investigators
* Significant associated diseases including documented human immunodeficiency virus (HIV) infection, uncontrolled hypertension (diastolic blood pressures \> 95%ile for age), unstable angina, congestive heart failure (\> New York \[NY\] class II), poorly controlled diabetes (hemoglobin A1c \[Hgb A1c\] \> 7%), coronary angioplasty within 6 months, myocardial infarction within the last 6 months, or uncontrolled atrial or ventricular cardiac arrhythmia, abnormal coagulation, persistent abnormal urinalysis reflecting intrinsic renal disease
* Active ongoing viral, bacterial, or fungal infection
4 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Rocket Pharma Limited
UNKNOWN
Fred Hutchinson Cancer Center
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Hans-Peter Kiem
Role: PRINCIPAL_INVESTIGATOR
Fred Hutch/University of Washington Cancer Consortium
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States
Countries
Review the countries where the study has at least one active or historical site.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
NCI-2011-00202
Identifier Type: REGISTRY
Identifier Source: secondary_id
RG9212015
Identifier Type: OTHER
Identifier Source: secondary_id
2097.00
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.