A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral… (NCT01896102) | Clinical Trial Compass
CompletedPhase 2/3
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
United States, Argentina, Australia32 participantsStarted 2013-08-21
Plain-language summary
This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blood stem cells were collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells were transplanted back into the participant following myeloablative conditioning. Participants in this study will be continuously followed in study LTF-304.
Who can participate
Age range17 Years
SexMALE
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Inclusion criteria
✓. Informed consent was 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 participants, in accordance with the directive of the IRB/IEC and with local requirements).
✓. Males aged 17 years and younger, at the time of parental/guardian consent and, where appropriate, participant assent.
✓. Active cerebral adrenoleukodystrophy (ALD) as defined by:
✓. Elevated very long chain fatty acids (VLCFA) values, and
✓. Active CNS disease established by central radiographic review of brain magnetic resonance imaging (MRI) demonstrating:
✓. Loes score between 0.5 and 9 (inclusive) on the 34-point scale, and
✓. Gadolinium enhancement on MRI of demyelinating lesions.
✓. NFS less than or equal to (\<or=) 1.
Exclusion criteria
What they're measuring
1
Percentage of Participants Who Were Alive and Have None of the 6 Major Functional Disabilities (MFDs) at Month 24 and Without Allo-HSCT or Rescue Cell Administration
Timeframe: At Month 24
2
Proportion of Participants Who Had Experienced Either Acute ([>or=] Grade II) or Chronic Graft Versus Host Disease (GVHD) by Month 24
. Receipt of an allogeneic transplant or gene therapy.
✕. Availability of a willing 10/10 HLA-matched sibling donor (excluding female heterozygotes).
✕. Use of statins, Lorenzo's Oil, or dietary regimens used to lower very long chain fatty acids (VLCFA) levels. Note: participants must discontinue use of these medications at time of consent.
✕. Receipt of an investigational study drug or procedure within 3 months before Screening that might confound study outcomes. Use of investigational study drugs is prohibited throughout the course of the study.
✕. Any conditions that make it impossible to perform MRI studies (including allergies to anesthetics or contrast agents).
✕. Hematological compromise as evidenced by:
✕. Hepatic compromise as evidenced by:
✕. Renal compromise as evidenced by abnormal renal function (actual or calculated creatinine clearance \< 50 milliliter per minute \[mL/min\])