Multiple clinical trials evaluate DM1-associated therapies across various conditions, including breast cancer, leukemia, head and neck neoplasms, and myotonic dystrophy type 1. Spanning Phase 1 through Phase 3 alongside non-applicable phase designations, these studies are sponsored by both pharmaceutical developers and academic institutions. Recruitment statuses across the portfolio range from active recruitment to completed or terminated trials, reflecting the broad therapeutic evaluation of mertansine as an antibody-drug conjugate payload.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT06138743 | Recruiting | Myotonic Dystrophy 1 |
Arrowhead Pharmaceuticals |
2024-03-04 | Phase 1 |
| NCT05532813 | Not yet recruiting | Steinert''s Disease|Myotonic Dystrophy 1|Metformin |
Assistance Publique - Hôpitaux de Paris |
2024-03 | Phase 3 |
| NCT06204809 | Recruiting | Myotonic Dystrophy 1 |
PepGen Inc |
2023-12-12 | Phase 1 |
| NCT06270186 | Withdrawn | Myotonic Dystrophy 1 |
Centre Hospitalier Universitaire de Nice |
2023-11 | Not Applicable |
| NCT05945927 | Recruiting | Breast Cancer |
Hoffmann-La Roche |
2023-09-13 | -- |
| NCT02452554 | COMPLETED | Pleuropulmonary Blastoma; Recurrent Malignant Peripheral Nerve Sheath Tumor; Recurrent Neuroblastoma; Recurrent Rhabdomyosarcoma; Recurrent Synovial Sarcoma; Wilms Tumor |
Children's Oncology Group |
2015-10-12 | PHASE2 |
| NCT02420873 | COMPLETED | Leukemia |
M.D. Anderson Cancer Center |
2015-05-12 | PHASE2 |
| NCT01237678 | TERMINATED | Small Cell Lung Cancer |
ImmunoGen, Inc. |
2010-11 | PHASE1; PHASE2 |
| NCT02254018 | COMPLETED | Head and Neck Neoplasms |
Boehringer Ingelheim |
2002-09-01 | PHASE1 |
| NCT02254031 | TERMINATED | Breast Neoplasms |
Boehringer Ingelheim |
2003-07-01 | PHASE1 |
| NCT02254005 | COMPLETED | Breast Neoplasms |
Boehringer Ingelheim |
2002-10-01 | PHASE1 |
| NCT02254044 | TERMINATED | Carcinoma, Squamous Cell |
Boehringer Ingelheim |
2003-10 | PHASE1 |
(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>).