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)
gene therapy for CCALD
Cerebral ALD phenotype (cerALD)
Study Chair(s)/Principal Investigator(s):
David Williams, MD
Florian Eichler, MD
Paul Orchard, MD
Ami Shah, MD
Drug Trial, Biomarkers/imaging, Gene Therapy
Active, currently recruiting
ALD Connect Affiliated Study
Interventional Trialwith active agents/drugs only
This is an international, non-randomized, open-label, multi-site, single-dose study in boys with CCALD designed to demonstrate that disease progression can be stabilized. Up to 15 subjects will be enrolled in order to obtain at least 12 evaluable subjects in a Transplant Population (TP), defined as all subjects for whom transplantation is performed.
Approximately 26 months.
Subjects will be asked to participate in a long-term follow up study at the conclusion of this study.
# of Subjects:
Enrollment Start Date:
Enrollment End Date:
Posting Last Modified Date:
Date Study Added to ALDConnect.org:
bluebird bio, Inc.
bluebird bio, Inc.
150 Second Street
Cambridge, Massachusetts 02141
Maximum Age: 17
Time since Symptom Onset: Not applicable
Time since Diagnosis: Not applicable
Other Eligibility 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 subjects, in accordance with the directive of the IRB/IEC and with local requirements).
• Boys aged 17 years and younger, at the time of parental/guardian consent and, where appropriate, subject assent.
• Active cerebral ALD as defined by: Elevated VLCFA levels, 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 of demyelinating lesions on MRI.
• NFS ≤ 1.
• Receipt of an allogeneic transplant or gene therapy.
• Availability of a willing 10/10 human leukocyte antigen (HLA)-matched sibling donor.
• 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 Day -60 that might confound study outcomes.
• Any conditions that make it impossible to perform MRI studies (including allergies to anesthetics or contrast agents).
• Hematological compromise as evidenced by:
i. Peripheral blood absolute neutrophil count (ANC) < 1500 cells/mm^3 or, ii. Platelet count < 100,000 cells/mm^3 or, iii. Hemoglobin < 10 g/dL or, iv. Uncorrected bleeding disorder.
• Hepatic compromise as evidenced by:
i. Aspartate transaminase (AST) value > 2.5 × the upper limit of normal (ULN) or, ii. Alanine transaminase (ALT) value > 2.5 × ULN or, iii. Total bilirubin value > 3.0 mg/dL, except if there is a diagnosis of Gilbert's Syndrome and the subject is otherwise stable.
• Renal compromise as evidenced by abnormal renal function (creatinine clearance < 50 mL/min).
• Cardiac compromise as evidenced by left ventricular ejection fraction < 40%.
• Immediate family member with a known or suspected Familial Cancer Syndrome.
• Clinically significant active bacterial, viral, fungal, or parasitic infection.
• Positive for presence of human immunodeficiency virus type 1 or 2 (HIV 1, HIV 2), hepatitis B, hepatitis C, or human T lymphotrophic virus 1 (HTLV 1).
• Any clinically significant cardiovascular or pulmonary, or other disease or condition that would be contraindicated for any of the other study procedures.
Site Contact Information
University of California at Los Angeles
757 Westwood Plaza
Los Angeles, California 90095
Boston Children's Hospital/Massachusetts General Hospital
300 Longwood Avenue
Boston, Massachusetts 02115
University of Minnesota, Amplatz Children's Hospital
2450 Riverside Ave
Minneapolis, Minnesota 55455