EUCTR2011-001953-10-FR
Active, not recruiting
Phase 1
A phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti D lentiviral vector for the treatment of childhood cerebral adrenoleukodystrophy (CCALD)
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Childhood Cerebral Adrenoleukodystrophy (CCALD)
- Sponsor
- bluebird bio, Inc.
- Enrollment
- 15
- Status
- Active, not recruiting
- Last Updated
- 4 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\.Informed consent is obtained from a competent custodial parent or guardian with legal capacity to execute a local Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved consent. (Informed assent will be sought from capable subjects, in accordance with the directive of the IRB/IEC and with local requirements.)
- •2\.Boys aged 17 years and younger, at the time of parental/guardian consent and, where appropriate, subject assent.
- •3\.Active cerebral ALD as defined by:
- •a.Elevated plasma VLCFA levels, and
- •b.Active CNS disease established by central radiographic review of brain MRI demonstrating
- •i.Loes score between 0\.5 and 9 (inclusive) on the 34\-point scale, and
- •ii.Gadolinium enhancement on MRI of demyelinating lesions.
- •4\.NFS \= 1 .
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 15
Exclusion Criteria
- •1\.Receipt of an allogeneic transplant or gene therapy.
- •2\.Availability of a willing 10/10 HLA\-matched sibling donor (excluding female heterozygote).
- •3\.Use of statins, Lorenzo’s Oil, or dietary regimens used to lower VLCFA levels. Note: subjects must discontinue use of these medications at time of consent.
- •4\.Receipt of an investigational study drug or procedure within 3 months before Screening that might counfound study outcomes. Use of investigational study drugs is prohibited throughout the course of the study .
- •5\.Any conditions that make it impossible to perform MRI studies (including allergies to anesthetics or contrast agents).
- •6\.Hematological compromise as evidenced by:
- •a.Peripheral blood absolute neutrophil count (ANC) \< 1500 cells/mm3,
- •b.Platelet count \< 100,000 cells/mm3, or
- •c.Hemoglobin \< 10 g/dL.
- •d.Uncorrected bleeding disorder.
Outcomes
Primary Outcomes
Not specified
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