Skip to main content
MedPathMedPath Home
Clinical Trials/EUCTR2018-002502-31-ES
EUCTR2018-002502-31-ES
Active, not recruiting
Phase 1

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

Rocket Pharmaceuticals, Inc.0 sites5 target enrollmentAugust 2, 2018
Share to TwitterShare to FacebookShare to LinkedInShare to Reddit
ConditionsFanconi anemia (subtype A)MedDRA version: 20.0Level: LLTClassification code 10055206Term: Fanconi's anemiaSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Overview

Phase
Phase 1
Intervention
Not specified
Conditions
Fanconi anemia (subtype A)
Sponsor
Rocket Pharmaceuticals, Inc.
Enrollment
5
Status
Active, not recruiting
Last Updated
7 years ago
OverviewInvestigatorsEligibility CriteriaOutcomesSimilar Trials

Overview

Brief Summary

No summary available.

Registry
who.int
Start Date
August 2, 2018
End Date
TBD
Last Updated
7 years ago
Study Type
Interventional clinical trial of medicinal product
Sex
All

Investigators

Eligibility Criteria

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:

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

Outcomes

Primary Outcomes

Not specified

Similar Trials

Active, not recruiting
Not Applicable
Clinical Trial testing the combination of MK-5172 and MK-8742 with Ribavirin in people with Hepatitis CHepatitis C Virus Genotype 2, 4, 5 and 6MedDRA version: 16.1Level: PTClassification code 10019744Term: Hepatitis CSystem Organ Class: 10021881 - Infections and infestationsMedDRA version: 16.1Level: LLTClassification code 10047457Term: Viral hepatitis CSystem Organ Class: 10021881 - Infections and infestationsTherapeutic area: Diseases [C] - Virus Diseases [C02]
EUCTR2013-002169-21-GBMerck Sharp & Dohme Corp.100
Active, not recruiting
Phase 1
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 AFanconi anemia (subtype A)MedDRA version: 20.0Level: LLTClassification code 10055206Term: Fanconi's anemiaSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
EUCTR2018-002502-31-GBRocket Pharmaceuticals, Inc.5
Active, not recruiting
Phase 1
Clinical Trial testing the combination of MK-5172 with/without MK-8742 and/or Ribavirin in people with Hepatitis CHepatitis C Virus Genotype 2/4/5/6MedDRA version: 17.0Level: PTClassification code 10019744Term: Hepatitis CSystem Organ Class: 10021881 - Infections and infestationsMedDRA version: 17.0Level: LLTClassification code 10047457Term: Viral hepatitis CSystem Organ Class: 10021881 - Infections and infestationsTherapeutic area: Diseases [C] - Virus Diseases [C02]
EUCTR2013-002169-21-BEMerck Sharp & Dohme Corp., a subsidiary of Merck & Co.,100
Active, not recruiting
Not Applicable
Clinical Trial testing the combination of MK-5172 and MK-8742 with Ribavirin in people with Hepatitis CHepatitis C Virus Genotype 2MedDRA version: 14.1Level: PTClassification code 10019744Term: Hepatitis CSystem Organ Class: 10021881 - Infections and infestationsMedDRA version: 14.1Level: LLTClassification code 10047457Term: Viral hepatitis CSystem Organ Class: 10021881 - Infections and infestationsTherapeutic area: Diseases [C] - Virus Diseases [C02]
EUCTR2013-002169-21-ESMerck Sharp & Dohme Corp.70
Active, not recruiting
Phase 1
Pembrolizumab (MK-3475) and Pembrolizumab in combination with other agents in High Risk Non-muscle Invasive Bladder Cancer (NMIBC) Patients Unresponsive to Bacillus Calmette-Guerin (BCG) Therapyon-Muscle Invasive Bladder Cancer (NMIBC)MedDRA version: 20.0Level: PTClassification code 10005003Term: Bladder cancerSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)Therapeutic area: Diseases [C] - Cancer [C04]
EUCTR2014-004026-17-FIMerck Sharp & Dohme LLC320