Multiple clinical trials spanning Phases 1 through 3 evaluate tamibarotene across hematologic malignancies—including acute myeloid leukemia, myelodysplastic syndrome, and acute promyelocytic leukemia—as well as non-oncology conditions such as lupus nephritis and Alzheimer's disease. Sponsored by pharmaceutical companies and academic institutions, these studies encompass various recruitment stages, ranging from actively recruiting combination therapy protocols to completed, terminated, or withdrawn trials. Together, these investigations highlight the ongoing therapeutic evaluation of tamibarotene across oncologic and autoimmune indications.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT06289998 | ACTIVE_NOT_RECRUITING | Autosomal Dominant Polycystic Kidney Disease (ADPKD) |
Rege Nephro Co., Ltd. |
2023-12-22 | PHASE2 |
| NCT04797780 | TERMINATED | Myelodysplastic Syndromes |
Syros Pharmaceuticals |
2021-02-08 | PHASE3 |
| NCT04905407 | TERMINATED | Acute Myeloid Leukemia |
Syros Pharmaceuticals |
2021-08-26 | PHASE2 |
| NCT06085638 | WITHDRAWN | Chronic Myelomonocytic Leukemia |
M.D. Anderson Cancer Center |
2023-09-14 | PHASE1; PHASE2 |
| NCT02807558 | COMPLETED | Acute Myeloid Leukemia; Myelodysplastic Syndrome |
Syros Pharmaceuticals |
2016-09-20 | PHASE2 |
| NCT04905407 | Recruiting | Acute Myeloid Leukemia |
Syros Pharmaceuticals |
2021-08-26 | Phase 2 |
| NCT02807558 | Completed | Acute Myeloid Leukemia|Myelodysplastic Syndrome |
Syros Pharmaceuticals |
2016-09-20 | Phase 2 |
| NCT01337154 | TERMINATED | Stage IIIB Non-small Cell Lung Cancer With Pleural Effusion; Stage IV Non-small Cell Lung Cancer |
CytRx |
2011-04 | PHASE2 |
| NCT01226147 | UNKNOWN | Lupus Nephritis |
Kinki University |
2010-09 | PHASE2 |
| NCT01120002 | UNKNOWN | Alzheimer's Disease |
Osaka City University |
2010-05 | PHASE2 |
| NCT00520208 | COMPLETED | Acute Promyelocytic Leukemia |
CytRx |
2007-09 | PHASE2 |
| NCT01343355 | UNKNOWN | HTLV-I-Associated Myelopathy |
St. Marianna University School of Medicine |
2011-01 | PHASE2; PHASE3 |
| NCT00985530 | TERMINATED | Acute Promyelocytic Leukemia |
Northwestern University |
2009-10 | PHASE1 |
| NCT00985530 | Terminated | Acute Promyelocytic Leukemia |
Northwestern University|CytRx|Cephalon |
2009-10 | Phase 1 |
| NCT00417391 | COMPLETED | Crohn's Disease |
R&R Inc. |
2006-12 | PHASE2 |
| NCT00520208 | Completed | Acute Promyelocytic Leukemia |
CytRx |
2007-09 | Phase 2 |
(data from https://clinicaltrials.gov, updated on 2025-04-04)
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>).