|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
1 April 2024 |
|
Main ID: |
NCT03157804 |
|
Date of registration:
|
26/04/2017 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A
FANCOLEN-1 |
|
Scientific title:
|
Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FANCOLEN-1) |
|
Date of first enrolment:
|
January 7, 2016 |
|
Target sample size:
|
9 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/ct2/show/NCT03157804 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
|
|
Phase:
|
Phase 1/Phase 2
|
|
|
Countries of recruitment
|
|
Spain
| | | | | | | |
|
Contacts
|
|
Name:
|
Juan A Bueren |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
CIEMAT/CIBERER/IIS.FJD |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Patients diagnosed with Fanconi Anemia complementation group A (FA-A)
- Minimum age 1 year
- Maximum age 21 years
- Lansky Index> 60%.
- Informed consent in accordance with current legal regulations.
- Number of cells to be transduced: At least 3x10^5 purified CD34+ / kg body weight.
- Negative result in the urine pregnancy test at the baseline visit for women of
childbearing age, who should be committed to using an effective contraceptive method
during the period of study participation.
Exclusion Criteria:
- Patients with an human leukocyte antigen (HLA) identical family donor.
- Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities
predicting the same in bone marrow aspirates. In this case, the studies carried out
two months in advance of the patient's entry into the clinical trial will be
considered valid.
- Evidence that the patient to be infused has signs of somatic mosaicism, with
hematologic improvement.
- Any illness or concomitant process that in the opinion of the investigator
incapacitates the subject for their participation in the study.
- Pre-existing sensory or motor impairment> = grade 2 according to the National Cancer
Institute (NCl) criteria.
- Pregnant or lactating women.
Age minimum:
1 Year
Age maximum:
21 Years
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Fanconi Anemia
|
|
Intervention(s)
|
|
Drug: Plerixafor
|
|
Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs)
|
|
Procedure: Bone Marrow Aspiration
|
|
Biological: Filgrastim
|
|
Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells
|
|
Other: Laboratory Biomarker Analysis
|
|
Primary Outcome(s)
|
|
Proportion of patients with at least 0.1 copy of the therapeutic vector per nucleated bone marrow or peripheral blood cells two years after infusion.
[Time Frame: 2 years after infusion of transduced cells]
|
|
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
[Time Frame: Up to 3 years after infusion of transduced cells]
|
|
Secondary Outcome(s)
|
|
Proportion of patients with clinical hematological response after the infusion of autologous CD34 + cells transduced with the therapeutic lentiviral vector
[Time Frame: 2 years after infusion of transduced cells]
|
|
Secondary ID(s)
|
|
2011-006100-12
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|