A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
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ClinicalTrials.gov Identifier: NCT01896102 |
Recruitment Status
:
Recruiting
First Posted
: July 11, 2013
Last Update Posted
: January 10, 2018
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Condition or disease | Intervention/treatment | Phase |
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Cerebral Adrenoleukodystrophy (CALD) | Genetic: Lenti-D Drug Product | Phase 2 Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 30 participants |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD) |
Study Start Date : | August 2013 |
Estimated Primary Completion Date : | August 2019 |
Estimated Study Completion Date : | December 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: Lenti-D Drug Product |
Genetic: Lenti-D Drug Product
Lenti-D Drug Product (autologous CD34+ cell-enriched population that contains cells transduced with Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein, suspended in a cryopreservative solution) is administered intravenously. Lenti-D Drug Product is administered by IV infusion following myeloablative conditioning with busulfan and cyclophosphamide.
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- Proportion of subjects who are alive and have none of the 6 major functional disabilities (MFDs) at Month 24 (i.e. Month 24 MFD-free survival). [ Time Frame: 24 months (±1 months) post-transplant ]
MFDs are:
- loss of communication
- cortical blindness
- tube feeding
- total incontinence
- wheelchair dependence
- complete loss of voluntary movement
- The proportion of subjects who experience either acute (≥Grade II) or chronic graft versus host disease (GVHD) at Month 24. [ Time Frame: 24 months (±1 months) post-transplant ]
- Incidence of resolution of gadolinium positivity on MRI (i.e., GdE-). [ Time Frame: 24 months (±1 months) post-transplant ]
- Time to sustained resolution of gadolinium positivity on MRI (i.e., GdE-). [ Time Frame: 24 months (±1 months) post-transplant ]Sustained is defined as gadolinium resolution without a subsequent evaluation indicating gadolinium positivity.
- Change in total NFS from Baseline to Month 24. [ Time Frame: 24 months (±1 months) post-transplant ]
- MFD-free survival over time [ Time Frame: 24 months (±1 months) post-transplant ]
- Overall survival [ Time Frame: 24 months (±1 months) post-transplant ]

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Ages Eligible for Study: | up to 17 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Informed consent is obtained from a competent custodial parent or guardian with legal capacity to execute a local 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.
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Active cerebral ALD as defined by:
- Elevated VLCFA values, and
- Active CNS disease established by central radiographic review of brain magnetic resonance imaging (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.
- Neurologic Function Score (NFS) ≤ 1.
Exclusion Criteria:
- 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 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).
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Hematological compromise as evidenced by:
- Peripheral blood ANC count < 1500 cells/mm3,
- Platelet count < 100,000 cells/mm3, or
- Hemoglobin < 10 g/dL.
- Uncorrected bleeding disorder.
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Hepatic compromise as evidenced by:
- Aspartate transaminase (AST) value > 2.5×ULN
- Alanine transaminase (ALT) value > 2.5×ULN
- 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 (actual or calculated 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 (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome, and familial adenomatous polyposis).
- Clinically significant active bacterial, viral, fungal, parasitic, or prion-associated infection
- Positive for human immunodeficiency virus type 1 or 2 (HIV-1, HIV-2); hepatitis B; hepatitis C; human T lymphotrophic virus 1 (HTLV-1). (Note that subjects who have been vaccinated against hepatitis B [hepatitis B surface antibody-positive] who are negative for other markers of prior hepatitis B infection [eg, negative for hepatitis B core antibody (Ab)] are eligible. Subjects with past exposure to HBV [HBcAb positive and/or HBeAb positive] are also eligible for the study provided they have a negative test for HBV DNA. Also note that subjects who are positive for anti-hepatitis C antibody are eligible as long as they have a negative hepatitis C viral load).
- Any clinically significant cardiovascular 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. Male subjects and their female partners are required to use two different effective methods of contraception from Screening through at least 6 months after drug product infusion.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01896102
Contact: bluebird bio | (339) 499-9300 | clinicaltrials@bluebirdbio.com |
United States, California | |
Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center | Recruiting |
Los Angeles, California, United States, 90095 | |
Contact: Dayna Terrazas 310-825-6708 drterrazas@mednet.ucla.edu | |
Principal Investigator: Satiro N De Oliveira, MD | |
United States, Massachusetts | |
Boston Children's Hospital/Massachusetts General Hospital | Recruiting |
Boston, Massachusetts, United States, 02115 | |
Contact: Colleen Dansereau 617-919-7008 Colleen.Dansereau@childrens.harvard.edu | |
United States, Minnesota | |
University of Minnesota | Recruiting |
Minneapolis, Minnesota, United States, 55455 | |
Contact: Kim Nelson 612-273-2925 knelso62@fairview.org | |
Contact: Maggie Kumbalek 612-273-2178 mmartyn1@fairview.org | |
Principal Investigator: Paul Orchard, MD | |
France | |
Hôpital Bicêtre | Recruiting |
Le Kremlin-Bicêtre Cedex, France, 94275 | |
Contact: Patrick Aubourg, MD patrick.aubourg@inserm.fr | |
Contact: Caroline Sevin, MD caroline.sevin@inserm.fr | |
Principal Investigator: Patrick Aubourg, MD | |
United Kingdom | |
Great Ormond Street Hospital for Children NHS Foundation Trust | Recruiting |
London, United Kingdom, WC1N3JH | |
Contact: Katie Snell k.snell@ucl.ac.uk | |
Principal Investigator: Adrian Thrasher, MD, PhD |
Study Director: | Mohammed Asmal, MD., PhD. | bluebird bio, Inc. | |
Principal Investigator: | David Williams, MD | Boston Children’s Hospital | |
Principal Investigator: | Christine Duncan, MD | Boston Children’s Hospital | |
Principal Investigator: | Florian Eichler, MD | Massachusetts General Hospital | |
Principal Investigator: | Satiro de Oliveira, MD | University of California, Los Angeles | |
Principal Investigator: | Paul Orchard, MD | University of Minnesota - Clinical and Translational Science Institute | |
Principal Investigator: | Adrian Thrasher, MD, PhD | Great Ormond Street Hospital for Chidren NHS Foundation Trust | |
Principal Investigator: | Patrick Aubourg, MD, PhD | Hôpital Bicêtre |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | bluebird bio |
ClinicalTrials.gov Identifier: | NCT01896102 History of Changes |
Other Study ID Numbers: |
ALD-102 |
First Posted: | July 11, 2013 Key Record Dates |
Last Update Posted: | January 10, 2018 |
Last Verified: | January 2018 |
Keywords provided by bluebird bio:
Adrenoleukodystrophy X-linked adrenoleukodystrophy Gene therapy Hematopoietic stem cell |
Additional relevant MeSH terms:
Adrenoleukodystrophy Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Hereditary Central Nervous System Demyelinating Diseases Leukoencephalopathies Demyelinating Diseases Mental Retardation, X-Linked Intellectual Disability |
Neurobehavioral Manifestations Neurologic Manifestations Genetic Diseases, X-Linked Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolism, Inborn Errors Peroxisomal Disorders Metabolic Diseases Adrenal Insufficiency Adrenal Gland Diseases Endocrine System Diseases |