Metachromatic Leukodystrophy
Conditions
Interventions
1:Lentiviral Hematopoietic Stem Cell Gene Therapy
2:No Lentiviral Hematopoietic Stem Cell Gene Therapy
Sponsors
Shenzhen Second People's Hospital, The First Affiliated Hospital of Shenzhen University
Eligibility
Sex/Gender
All
Age
1 Years to 35 Years
Inclusion criteria
Inclusion criteria: For MLD inclusion criteria 1.Confirmed diagnosis as MLD by ARSA genetic diagnosis , MRI imaging and low ARSA A activity ( below 20% or normal level) 2.The patient' symptoms and lesions have not been developed to the end stage of MLD. For ALD inclusion criteria 1.Confirmed diagnosis as MLD by ABCD1genetic diagnosis , MRI imaging and high VLCFA level, abnormal highlevel of adrenocorticotropic hormone,ACTH. 2. The patient' symptoms and lesions have not been developed to the end stage of ALD
Exclusion criteria
Exclusion criteria: For MLD exclusion criteria. 1.No clinical symptoms of MLD. 2.ARSA Activity >50% of healthy controls 3.end stage of MLD 4. Other complications .ie. Cancer. 5.HIV RNA and/or HCV RNA and/or HBV DNA positive patients 6.Patient who underwent allogenic hematopoietic stem cell transplantation with evidence of residual cells of donor origin For ALD exclusion criteria. 1.No clinical symptoms 2.Normal level of VLCFAs in blood 3.End stage of ALD 4.Other complications .ie. Cancer. 5.HIV RNA and/or HCV RNA and/or HBV DNA positive patients. 6.Patient who underwent allogenic hematopoietic stem cell transplantation with evidence of residual cells of donor origin.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Neural scores;ARSA activity (MLD);VLCFA level ( ALD); | — |
Contacts
Public ContactZhou JiaCai
Outcome results
None listed