Clinical Trials
Tolebrutinib is being evaluated across several Phase 1, Phase 2, and Phase 3 clinical trials for neuroinflammatory and autoimmune conditions, including myasthenia gravis, renal or hepatic impairment, and relapsing, primary progressive, and non-relapsing secondary progressive multiple sclerosis. Predominantly sponsored by Sanofi with academic contributions from the National Institute of Neurological Disorders and Stroke, these studies assess safety, pharmacokinetics, and therapeutic efficacy, with recruitment statuses encompassing completed, active not recruiting, and terminated protocols.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT06372145 | ACTIVE_NOT_RECRUITING | Relapsing Multiple Sclerosis; Secondary Progressive Multiple Sclerosis; Progressive Relapsing Multiple Sclerosis |
Sanofi |
2024-04-16 | PHASE3 |
| NCT04458051 | COMPLETED | Primary Progressive Multiple Sclerosis |
Sanofi |
2020-08-13 | PHASE3 |
| NCT03996291 | COMPLETED | Relapsing Multiple Sclerosis |
Sanofi |
2019-09-23 | PHASE2 |
| NCT04411641 | COMPLETED | Non-relapsing Secondary Progressive Multiple Sclerosis |
Sanofi |
2020-09-24 | PHASE3 |
| NCT04410991 | COMPLETED | Relapsing Multiple Sclerosis |
Sanofi |
2020-06-11 | PHASE3 |
| NCT04410978 | COMPLETED | Relapsing Multiple Sclerosis |
Sanofi |
2020-06-30 | PHASE3 |
| NCT04742400 | UNKNOWN | Multiple Sclerosis |
National Institute of Neurological Disorders and Stroke (NINDS) |
2021-04-15 | PHASE2 |
| NCT05132569 | TERMINATED | Myasthenia Gravis |
Sanofi |
2021-12-03 | PHASE3 |
| NCT05282030 | COMPLETED | Renal Impairment |
Sanofi |
2022-03-10 | PHASE1 |
| NCT05283915 | COMPLETED | Hepatic Function Abnormal |
Sanofi |
2022-03-18 | PHASE1 |
| NCT06106074 | COMPLETED | Multiple Sclerosis |
Sanofi |
2020-08-10 | PHASE1 |
| NCT05283915 | Completed | Hepatic Function Abnormal |
Sanofi |
2022-03-18 | Phase 1 |
| NCT05282030 | Completed | Renal Impairment |
Sanofi |
2022-03-23 | Phase 1 |
| NCT04742400 | Active not recruiting | Multiple Sclerosis |
National Institute of Neurological Disorders and Stroke (NINDS)|National Institutes of Health Clinical Center (CC) |
2021-04-15 | Phase 2 |
| NCT06106074 | Completed | Multiple Sclerosis |
Sanofi |
2020-08-10 | Phase 1 |
| NCT06064539 | COMPLETED | Multiple Sclerosis |
Sanofi |
2020-05-18 | PHASE1 |
| NCT06064539 | Completed | Multiple Sclerosis |
Sanofi |
2020-05-18 | Phase 1 |
(data from https://clinicaltrials.gov, updated on 2026-08-17)
Compliance for Clinical Use
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>).