Contact SCGE




Gene Therapy Trial Report

Summary

Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy


NCTID NCT02559830 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Metachromatic Leukodystrophy, Adrenoleukodystrophy
Disease Ontology Term DOID:10588; DOID:10581
Compound Name CD34+ hematopoietic stem cells transduced with ABCD1 or ARSA
Sponsor Shenzhen Second People's Hospital
Funder Type Other
Recruitment Status
Unknown
Enrollment Count 50 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant ABCD1 or ARSA
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type LV
Editor Type none
Dose 1 2E6 transduced CD34+ cells/kg (maximum 20E6)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2015-08-12
Completion Date 2025-10
Last Update 2022-05-31

Participation Criteria


Eligible Age 1 Year - 16 Years
Standard Ages Child
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: Inclusion Criteria For MLD: 1. Confirmed diagnosis as MLD by ARSA genetic diagnosis, MRI(Magnetic Resonance Imaging)and low ARSA A activity (below 20% of normal level); 2. The patient' symptoms and lesions have not been developed to the end stage of MLD. 3. age \< 16.0 years at symptom onset Inclusion Criteria For ALD: 1. Confirmed diagnosis as ALD by ABCD1 genetic diagnosis, abnormal MRI imaging, abnormal high level of very long chain fatty acid (VLCFA) and adrenocorticotropic hormone (ACTH); 2. The patient' symptoms and lesions have not been developed to the end stage of ALD. 3. age \< 16.0 years at symptom onset Exclusion Criteria: Exclusion Criteria For MLD: 1. At a pre-symptomatic stage of of MLD; 2. ARSA activity \>50% compared to healthy individuals; 3. End stage of MLD; 4. Other complications, ie. Cancer; 5. human immunodeficiency virus(HIV) RNA and/or hepatitis C virus RNA and/or hepatitis B virus DNA positive patients; 6. Patients who underwent allogenic hematopoietic stem cell transplantation with evidence of residual cells of donor origin. 7. Serious organ dysfunction; 8. were enrolled in other clinical trials in the 6 months prior to screening; 9. had any other concern that hampered the compliance or safety as judged by the investigator; 10. Adult Exclusion Criteria For ALD: 1. No evidence of brain lesions; 2. Normal level of VLCFAs in blood; 3. End stage of ALD; 4. Other complications, ie. Cancer; 5. human immunodeficiency virus(HIV) RNA and/or hepatitis C virus RNA and/or hepatitis B virus DNA positive patients; 6. Patients who underwent allogenic hematopoietic stem cell transplantation with evidence of residual cells of donor origin. 7. Serious organ dysfunction; 8. were enrolled in other clinical trials in the 6 months prior to screening; 9. had any other concern that hampered the compliance or safety as judged by the investigator; 10. Adult
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND
FDA Designations
Recent Updates

Resources/Links


Resources/Links

No External Links Available.