ß-Thalassemia
Conditions
Interventions
RM-001 Treatment Group:None
Sponsors
The First Affiliated Hospital of Guangxi Medical University
Eligibility
Sex/Gender
All
Age
6 Years to 35 Years
Inclusion criteria
Inclusion criteria: 1. Subject (himself/herself or legal guardian) voluntarily signs the informed consent and is able to complete the process and assessments of the long-term follow-up study. 2. Treated with RM-001 cells for therapy of transfusion-dependent ß-thalassemia in the clinical studies initiated or supported by Guangzhou Reforgene Medicine Co., Ltd.. 3. Able to comply with the clinical study protocol for follow-up visits.
Exclusion criteria
Exclusion criteria: There are no exclusion criteria for this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| All-cause mortality/overall survival;Serious adverse events after RM-001 infusion;The number of subjects with new or worsening hematologic disorders;The number of subjects with malignancies;The number of subjects with malignancies related to gene editing;Proportion of subjects who achieved transfusion independence (TI); | — |
Secondary
| Measure | Time frame |
|---|---|
| Occurrence of RM-001-related adverse events;Immune-related adverse events;Characteristics of subjects who achieved transfusion independence;Characteristics of subjects who achieved transfusion reduction;The average Hb levels of subjects at Year 5, Year 10 and Year 15 after product infusion;Changes of iron overload;Iron chelation therapy;Erythrocyte development; | — |
Countries
China
Contacts
Public ContactYongrong Lai
The First Affiliated Hospital of Guangxi Medical University
Outcome results
None listed