Multiple clinical trials are currently evaluating procedures and therapeutic strategies for hematologic malignancies, severe aplastic anemia, and sickle cell disease in the context of hematopoietic stem cell transplantation. Ranging from Phase II feasibility studies to non-applicable phase protocols, these trials encompass recruitment statuses of recruiting, active not recruiting, and not yet recruiting. Academic institutions and clinical consortia, including Siriraj Hospital, Nationwide Children's Hospital, Assistance Publique - Hôpitaux de Paris, and the Center for International Blood and Marrow Transplant Research, serve as study sponsors.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT06286228 | Not yet recruiting | Multiple Myeloma |
Siriraj Hospital|Faculty of Medicine Siriraj Hospital |
2024-04-01 | -- |
| NCT06082947 | Recruiting | Hematopoietic Stem Cell Transplantation|Hematologic Malignancy |
Nationwide Children''s Hospital |
2023-12-18 | Not Applicable |
| NCT05419843 | Not yet recruiting | Idiopathic Aplastic Anemia|Severe Aplastic Anemia|Moderate Aplastic Anemia Requiring Transfusions |
Assistance Publique - Hôpitaux de Paris |
2022-06 | Phase 2 |
| NCT03904134 | Active not recruiting | Acute Myeloid Leukemia|Acute Lymphoblastic Leukemia|Myelodysplastic Syndromes|Non-hodgkin Lymphoma|Hodgkin Lymphoma|Acquired Aplastic Anemia|Sickle Cell Disease |
Center for International Blood and Marrow Transplant Research|National Heart Lung and Blood Institute (NHLBI)|National Cancer Institute (NCI)|Blood and Marrow Transplant Clinical Trials Network|National Marrow Donor Program|Medical College of Wisconsin |
2019-06-14 | Not Applicable |
(data from https://clinicaltrials.gov, updated on 2024-05-22)
Mechanism and Biochemical Profile
Appendix RUO and cGMP Quality Standards
| Quality Dimension | RUO (Research Use Only) | cGMP (Current Good Manufacturing Practice) |
|---|---|---|
| Clinical Applicability | Prohibited in human clinical trials or medical diagnostics. | Mandatory for human clinical trials (Phase I–III) and therapies. |
| Regulatory Status | Non-regulated grade; exempt from drug manufacturing laws. | Legally enforced by health authorities (e.g., FDA, EMA, NMPA). |
| Facility Environment | Unclassified analytical or research laboratories. | Validated Cleanrooms (ISO Class 5–8) with continuous monitoring. |
| Quality Control | Basic purity and activity testing. | Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma). |
| Process Validation | Basic equipment calibration; no process validation required. | Full qualification (IQ/OQ/PQ) and complete batch records. |
| Quality Assurance | Vendor self-declared without required formal QMS. | Mandatory QA/QC unit, Change Control, CAPA, and vendor audits. |
| Regulatory Impact | High risk of IND rejection if used as a critical raw material. | Required for IND/NDA filings, supported by Drug Master Files (DMF). |
Footnotes
Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).
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