A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral … (NCT03852498) | Clinical Trial Compass
CompletedPhase 3
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
United States, France, Germany35 participantsStarted 2019-01-24
Plain-language summary
The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and fludarabine in participants with CALD. A participant's blood stem cells will be 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 will be transplanted back into the participant following myeloablative conditioning. Enrollment and treatment in Study ALD-104 have been completed and further enrollment in this study is not expected, although participants follow-up remains ongoing in the long-term follow-up Study LTF-304 (NCT02698579).
Who can participate
Age range17 Years
SexMALE
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Inclusion criteria
✓. 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 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 CALD as defined by:
✓. Elevated very long chain fatty acids (VLCFA) values, and
✓. Active central nervous system (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 (GdE) on MRI of demyelinating lesions.
✓. NFS \< or = 1.
Exclusion criteria
✕. Prior receipt of an allogeneic transplant or gene therapy.
✕. Use of statins, Lorenzo's Oil, or dietary regimens used to lower VLCFA levels. Note: participants must discontinue use of these medications at time of consent.
What they're measuring
1
Percentage of Participants Who Were Alive and Have None of the 6 Major Functional Disabilities (MFDs) at Month 24
Timeframe: At Month 24
2
Percentage of Participants Who Achieved Neutrophil Engraftment After Drug Product Infusion
✕. 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:
✕. Peripheral blood absolute neutrophil count (ANC) count \<1500 cells/ cubic millimeter (mm\^3), and either