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Clinical trial

A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy

ID
Name
201222
Description
This Phase I/II clinical trial consists of the application of lentiviral vector-based gene therapy to patients affected by Metachromatic Leukodystrophy (MLD), a rare inherited Lysosomal Storage Disorder (LSD) resulting from mutations in the gene encoding the Arylsulfatase A (ARSA) enzyme. The medicinal product consists of autologous CD34+ hematopoietic stem/progenitor cells in which a functional ARSA cDNA is introduced by means of 3rd generation VSV-G pseudotyped lentiviral vectors.
Trial arms
Trial start
2010-04-09
Estimated PCD
2018-04-09
Trial end
2025-03-15
Status
Active (not recruiting)
Phase
Early phase I
Treatment
OTL-200 Gene Therapy
Autologous hematopoietic stem/progenitor cells collected from the bone marrow and transduced ex vivo with a Lentiviral vector encoding the human ARSA cDNA
Arms:
OTL-200 Gene Therapy
Other names:
Previously GSK2696274
Size
20
Primary endpoint
Improvement of Gross Motor Function Measure (GMFM) score
24 months after treatment
Increase of residual Arylsulfatase A (ARSA) activity
24 months after treatment
Conditioning regimen-related safety
at +60 days after transplantation
Conditioning regimen-related toxicity
3 years after treatment
The short-term safety and tolerability of lentiviral-transduced cell infusion
48 hours after treatment infusion
The long-term safety of lentiviral-transduced cell infusion
baseline, 1, 3, 6, and 12 months after treatment, then once a year
The long-term safety of lentiviral-transduced cell infusion
baseline, 3, 6 and 12 months after treatment, then once a year
The long-term safety of lentiviral-transduced cell infusion
6 and 12 months after treatment, then once a year
Eligibility criteria
Inclusion Criteria: * Pre-symptomatic MLD patients with the late infantile variant; * Pre- or early-symptomatic MLD patients with the early juvenile variant; * Patients for whom parental/guardian signed informed consent has been obtained. Exclusion Criteria: * HIV RNA and/or HCV RNA and/or HBV DNA positive patients; * Patients affected by neoplastic diseases; * Patients with cytogenetic alterations typical of MDS/AML; * Patients with end-organ functions or any other severe disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study; * Patients enrolled in other trials/other therapeutic approaches that might become available; * Patient who underwent allogeneic hematopoietic stem cell transplantation in the previous six months; * Patient who underwent allogenic hematopoietic stem cell transplantation with evidence of residual cells of donor origin.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 20, 'type': 'ACTUAL'}}
Updated at
2023-11-24

1 organization

1 product

2 indications

Product
OTL-200
Indication
Lysosomal Storage Disease
Indication
Metachromatic Leukodystrophy
Organization
Orchard Therapeutics