A Phase I/II Clinical Trial of Lentiviral Hematopoietic Stem Cell Gene Therapy for Treatment of Developed Metachromatic Leukodystrophy and Adrenoleukodystrophy
Who is this study for? Child to adult patients with Adrenoleukodystrophy or Metachromatic Leukodystrophy
What treatments are being studied? CD34-Positive Hematopoietic Stem Cell Transplant
Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
Evaluating the safety and efficacy of Lentiviral Hematopoietic Stem Cell Gene Therapy for advanced stage of Metachromatic Leukodystrophy and adrenoleukodystrophy.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 1
Maximum Age: 16
Healthy Volunteers: f
View:
• Confirmed diagnosis as MLD by ARSA genetic diagnosis, MRI（Magnetic Resonance Imaging）and low ARSA A activity (below 20% of normal level);
• The patient' symptoms and lesions have not been developed to the end stage of MLD.
• age \< 16.0 years at symptom onset
• 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);
• The patient' symptoms and lesions have not been developed to the end stage of ALD.
• age \< 16.0 years at symptom onset
Locations
Other Locations
China
Shenzhen Second People's Hospital, The First Affiliated Hospital of Shenzhen University
RECRUITING
Shenzhen
Contact Information
Primary
JiaCai zhou, M.D.,Ph.D
jiacai8199@163.com
+8613923406652
Backup
Qizhou Lian, M.D.,Ph.D.
dalilian2000@aliyun.com
Time Frame
Start Date: 2015-01
Estimated Completion Date: 2025-10
Participants
Target number of participants: 50
Treatments
Experimental: transduced CD34+ hematopoietic stem cell
Transplantation of autologous CD34+ hematopoietic stem cells transduced with ARSA/ABCD1 encoding lentiviral vector. Dosage: 2x10\^6/Kg （Minimum）to 20x10\^6/Kg （Maximum） transduced CD34+ cells at bedside for infusion
Related Therapeutic Areas
Sponsors
Leads: Shenzhen Second People's Hospital
Collaborators: Shenzhen University, Guangzhou Women and Children's Medical Center