A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

  • End date
    Feb 27, 2024
  • participants needed
  • sponsor
    bluebird bio
Updated on 27 January 2021


Study ALD-104 is an international, non-randomized, open-label, multi-site study in male participants (<or=17 years of age at enrollment) with cerebral adrenoleukodystrophy (CALD). Approximately 35 participants will be infused with Lenti-D Drug Product after myeloablative conditioning with busulfan and fludarabine.

This trial will evaluate the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of CALD. A subject'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 subject following myeloablative conditioning.

Condition Cerebral Adrenoleukodystrophy, cald
Treatment Genetic, Genetic, Lenti-D; elivaldogene autotemcel, Lenti-D
Clinical Study IdentifierNCT03852498
Sponsorbluebird bio
Last Modified on27 January 2021


Yes No Not Sure

Inclusion Criteria

Is your age less than or equal to 17 yrs?
Are you male?
Do you have Cerebral Adrenoleukodystrophy?
Do you have any of these conditions: cald or Cerebral Adrenoleukodystrophy?
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
Males aged 17 years and younger, at the time of parental/guardian consent and, where appropriate, subject assent
Active cerebral ALD 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
Loes score between 0.5 and 9 (inclusive) on the 34-point scale, and ii. Gadolinium enhancement on MRI of demyelinating lesions
NFS less than or equal to (<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: subjects 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
Peripheral blood absolute neutrophil count (ANC) count <1500 cells/ cubic millimeter (mm3), and either
Platelet count <100,000 cells/mm3, or
Hemoglobin <10 gram per deciliter (g/dL)
Hepatic compromise as evidenced by
Aspartate transaminase (AST) value >2.5 upper limit of normal (ULN)
Alanine transaminase (ALT) value >2.5 ULN
Total bilirubin value >3.0 milligram per deciliter (mg/dL), except if there is a diagnosis of Gilbert's Syndrome and the subject is otherwise stable
Baseline estimated glomerular filtration rate <70 milliliter per minute (mL/min)/1.73 square meter (m2)
Cardiac compromise as evidenced by left ventricular ejection fraction <40 percent (%)
Immediate family member with a known or suspected Familial Cancer Syndrome
Clinically significant uncontrolled, active bacterial, viral, fungal, parasitic, or prion associated infection
Positive for HIV, hepatitis B or C virus, or human T lymphotrophic virus 1 (HTLV-1)
Any clinically significant cardiovascular, hematological, or pulmonary disease, or other disease or condition that would be contraindicated for any of the other study procedures
Absence of adequate contraception for fertile subjects
Any contraindications to the use of Granulocyte colony-stimulating factor (G-CSF) or plerixafor during the mobilization of hematopoietic stem cells, and any contraindications to the use of busulfan or fludarabine, including known hypersensitivity to the active substances or to any of the excipients in their formulations
Known hypersensitivity to protamine sulfate
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