PRN1008 (Rilzabrutinib) has been evaluated across numerous Phase 1, Phase 2, and Phase 3 clinical trials sponsored primarily by Sanofi and Principia Biopharma in collaboration with academic institutions such as Massachusetts General Hospital. Spanning completed, recruiting, active, and terminated statuses, these investigations evaluate the compound's safety, pharmacokinetics, and therapeutic efficacy in healthy volunteers, subjects with hepatic impairment, and patients with autoimmune and inflammatory conditions, including immune thrombocytopenia, pemphigus vulgaris, IgG4-related disease, chronic spontaneous urticaria, and asthma.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT07216079 | ACTIVE_NOT_RECRUITING | Immune Thrombocytopenia |
Sanofi |
2025-10-15 | PHASE3 |
| NCT04562766 | ACTIVE_NOT_RECRUITING | Immune Thrombocytopenia |
Principia Biopharma, a Sanofi Company |
2020-12-14 | PHASE3 |
| NCT04520451 | COMPLETED | Immunoglobulin G4 Related Disease |
Principia Biopharma, a Sanofi Company |
2020-08-22 | PHASE2 |
| NCT03395210 | COMPLETED | Immune Thrombocytopenia |
Principia Biopharma, a Sanofi Company |
2018-03-22 | PHASE2 |
| NCT05104892 | Completed | Asthma |
Sanofi |
2021-12-12 | Phase 2 |
| NCT05107115 | Completed | Chronic Spontaneous Urticaria |
Sanofi |
2021-11-24 | Phase 2 |
| NCT03762265 | TERMINATED | Pemphigus |
Principia Biopharma, a Sanofi Company |
2019-01-08 | PHASE3 |
| NCT04748926 | COMPLETED | Healthy Volunteers |
Principia Biopharma, a Sanofi Company |
2021-04-07 | PHASE1 |
| NCT06444191 | COMPLETED | Healthy Volunteers |
Principia Biopharma, a Sanofi Company |
2021-01-04 | PHASE1 |
| NCT04748926 | Completed | Healthy Volunteers |
Principia Biopharma a Sanofi Company|Sanofi |
2021-04-07 | Phase 1 |
| NCT06444204 | COMPLETED | Hepatic Function Abnormal; Healthy Volunteers |
Principia Biopharma, a Sanofi Company |
2020-11-02 | PHASE1 |
| NCT04562766 | Recruiting | Immune Thrombocytopenia |
Principia Biopharma a Sanofi Company|Sanofi |
2020-12-14 | Phase 3 |
| NCT04520451 | Active not recruiting | Immunoglobulin G4 Related Disease |
Principia Biopharma a Sanofi Company|Massachusetts General Hospital|Sanofi |
2020-08-22 | Phase 2 |
| NCT02704429 | COMPLETED | Pemphigus Vulgaris |
Principia Biopharma, a Sanofi Company |
2016-01-22 | PHASE2 |
(data from https://clinicaltrials.gov, updated on 2025-11-06)
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>).