Main
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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:
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EUCTR |
Last refreshed on:
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7 January 2019 |
Main ID: |
EUCTR2018-002502-31-ES |
Date of registration:
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02/08/2018 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A
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Scientific title:
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A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A - FANCOLEN-II |
Date of first enrolment:
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31/10/2018 |
Target sample size:
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5 |
Recruitment status: |
Authorised-recruitment may be ongoing or finished |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-002502-31 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): yes
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): no
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Countries of recruitment
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Spain
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Contacts
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Name:
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Teresa Bricio
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Address:
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Pso. de la Castellana, 163 2ºIzq
28046
Madrid
Spain |
Telephone:
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34917452520 |
Email:
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teresa.bricio@alphabioresearch.com |
Affiliation:
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Alpha Bioresearch, S.L. |
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Name:
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Teresa Bricio
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Address:
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Pso. de la Castellana, 163 2ºIzq
28046
Madrid
Spain |
Telephone:
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34917452520 |
Email:
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teresa.bricio@alphabioresearch.com |
Affiliation:
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Alpha Bioresearch, S.L. |
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Key inclusion & exclusion criteria
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Inclusion criteria: Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of DEB or a similar DNA-crosslinking agent 2. Patients of the complementation group FA-A 3. Minimum age: 1 year and a minimum weight of 8 kg. 4. Maximum age: 12 years 5. At least one of the following hematologic parameters below lower limits of normal of • Hemoglobin • Absolute neutrophils • Platelets 6. At least 30 CD34+ cells/µL are determined in one BM aspiration within 3 months prior to CD34+ cell collection 7. If the number of C34+ cells/ µL in BM is in the range of 10-29, PB parameters should meet two of the three following criteria: • Hemoglobin: =11g/dL • Neutrophils: =900 cells/µL • Platelets: =60,000 cells/µL 8. Provide informed consent in accordance with current legislation 9. Women of childbearing age must have a negative urine pregnancy test at the baseline visit, and accept the use of an effective contraception method during participation in the trial Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria: Patients with an HLA-identical sibling donor 2. Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities predictive of these conditions in BM aspirate analysis. This assessment should be made by valid studies conducted within the 3 months before the patient enters the clinical trial 3. Patients with somatic mosaicism associated with stable or improved counts in all PB cell lineages 4. Lansky performance index = 60% 5. Any concomitant disease or condition that, in the opinion of the Principal Investigator, deems the patient unfit to participate in the trial 6. Pre-existing sensory or motor impairment > = grade 2 according to the criteria of the National Cancer Institute (NCI) 7. Pregnant or breastfeeding women 8. Hepatic dysfunction as defined by either: • Bilirubin > 1.5 x the upper limit of normal (ULN) • Alanine aminotransferase (ALT ) > 2.5 x ULN • Aspartate aminotransferase (ALT) > 2.5 x ULN. 9. Renal dysfunction requiring either hemodialysis or peritoneal dialysis 10. Pulmonary dysfunction as defined by either: • Need for supplemental oxygen during the prior 2 weeks (in absence of acute infection) • Oxygen saturation (by pulse oximetry) <90%. 11. Evidence of active metastatic or locoregionally advanced malignancy for which survival is anticipated to be less than 3 years
Age minimum:
Age maximum:
Gender:
Female: yes Male: yes
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Health Condition(s) or Problem(s) studied
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Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
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Fanconi anemia (subtype A) MedDRA version: 20.0
Level: LLT
Classification code 10055206
Term: Fanconi's anemia
System Organ Class: 100000004850
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Intervention(s)
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Product Name: RP-L102 Pharmaceutical Form: Suspension for injection INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS Concentration unit: IU/kg international unit(s)/kilogram Concentration type: not less then Concentration number: 500000-
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Primary Outcome(s)
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Primary end point(s): • Level of phenotypic correction of hematopoietic cells after the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A • Level of engraftment of gene-corrected hematopoietic cells after the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A • Clinical response (prevention or rescue of bone marrow failure (BMF)) after the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A • Ongoing evaluation of the short- and long-term safety of the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A
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Secondary Objective: Not applicable
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Main Objective: The main objective of this trial is to assess the therapeutic efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene in patients with Fanconi anemia subtype A (complementation group A, FA-A).
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Timepoint(s) of evaluation of this end point: Up to 3 years after infusion
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Secondary Outcome(s)
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Timepoint(s) of evaluation of this end point: Not applicable
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Secondary end point(s): Not applicable
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Secondary ID(s)
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RP-L102-0118
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Source(s) of Monetary Support
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Rocket Pharmaceuticals, Inc.
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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