Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy

Launched by SHENZHEN SECOND PEOPLE'S HOSPITAL · Sep 23, 2015

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called gene therapy for two rare conditions: Metachromatic Leukodystrophy (MLD) and Adrenoleukodystrophy (ALD). The goal is to see if this treatment is safe and effective for children and young people who are experiencing advanced stages of these diseases. Gene therapy works by using modified cells from the patient's own body, which are designed to help improve their condition. The trial is currently looking for participants who are under 16 years old at the time their symptoms began and have specific diagnoses confirmed by genetic testing and imaging.

To be eligible for the trial, participants must have not reached the end stage of either condition and must meet certain health criteria. They should not have serious other health issues, like cancer or severe organ dysfunction, and individuals with certain infections will also be excluded. If someone joins the trial, they can expect close monitoring and support throughout the treatment process. It's important for families to know that this trial aims to provide hope for children suffering from these challenging conditions and could pave the way for future treatments.

Gender

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