Multiple clinical trials spanning Phase 1 through Phase 4 evaluate aficamten in healthy adult volunteers as well as adult and pediatric patients with symptomatic obstructive and non-obstructive hypertrophic cardiomyopathy (HCM). Sponsored by industry entities—such as Cytokinetics, Ji Xing Pharmaceuticals, Corxel Pharmaceuticals, and Bayer—and academic institutions like Oregon Health and Science University, these trials feature active recruitment, completed, and invitation-based statuses. Key studies include pivotal Phase 3 comparisons against standard care, pediatric Phase 2/3 trials, and open-label extensions evaluating long-term safety and therapeutic efficacy.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT06412666 | ACTIVE_NOT_RECRUITING | Pediatric; Symptomatic Obstructive Hypertrophic Cardiomyopathy |
Cytokinetics |
2024-05-29 | PHASE2; PHASE3 |
| NCT04848506 | ENROLLING_BY_INVITATION | Symptomatic Hypertrophic Cardiomyopathy (HCM) |
Cytokinetics |
2021-05-06 | PHASE2; PHASE3 |
| NCT07600177 | RECRUITING | Hypertrophic Cardiomyopathy (HCM) |
Oregon Health and Science University |
2026-05-05 | PHASE4 |
| NCT07023341 | ACTIVE_NOT_RECRUITING | Obstructive Hypertrophic Cardiomyopathy |
Bayer |
2025-06-30 | PHASE3 |
| NCT06081894 | ACTIVE_NOT_RECRUITING | Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy |
Cytokinetics |
2023-08-31 | PHASE3 |
| NCT06116968 | COMPLETED | Obstructive Hypertrophic Cardiomyopathy |
Corxel Pharmaceuticals |
2023-11-14 | PHASE3 |
| NCT05767346 | COMPLETED | Obstructive Hypertrophic Cardiomyopathy (oHCM) |
Cytokinetics |
2023-06-20 | PHASE3 |
| NCT05924815 | COMPLETED | Healthy Participants |
Cytokinetics |
2023-05-15 | PHASE1 |
| NCT05186818 | COMPLETED | Obstructive Hypertrophic Cardiomyopathy (oHCM) |
Cytokinetics |
2022-02-01 | PHASE3 |
| NCT06116968 | Recruiting | Obstructive Hypertrophic Cardiomyopathy |
Ji Xing Pharmaceuticals (Shanghai) Co. Ltd. |
2023-11-14 | Phase 3 |
| NCT04848506 | Enrolling by invitation | Symptomatic Hypertrophic Cardiomyopathy (HCM) |
Cytokinetics |
2021-05-06 | Phase 2 |
| NCT04783766 | Completed | Obstructive Hypertrophic Cardiomyopathy|Healthy Adult Subjects |
Ji Xing Pharmaceuticals (Shanghai) Co. Ltd.|Cytokinetics |
2021-04-10 | Phase 1 |
(data from https://clinicaltrials.gov, updated on 2026-08-18)
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>).