4 completed, 1 active not recruiting, 1 approved for marketing, 1 recruiting. 3 Phase 3, 2 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 3 | Active not recruiting 1, Completed 1, Recruiting 1 |
| Phase 2 | 2 | Completed 2 |
| Phase 1 | 1 | Completed 1 |
Follow donidalorsen: new trials by email Literature Review of donidalorsen trials Research Report on donidalorsen
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Donidalorsen Treatment in Children With Hereditary Angioedema Donidalorsen - Hereditary Angioedema (HAE) | Phase 3 | Recruiting | Ionis Pharmaceuticals, Inc. | 20 | 2026-04-21 | 2026-11-02 |
| A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) Donidalorsen - Hereditary Angioedema | Phase 3 | Active not recruiting | Ionis Pharmaceuticals, Inc. | 154 | 2022-07-13 | 2025-12-12 |
| OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE) Donidalorsen, Placebo - Hereditary Angioedema | Phase 3 | Completed | Ionis Pharmaceuticals, Inc. | 91 | 2021-12-03 | 2025-03-06 |
| An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema Donidalorsen - Hereditary Angioedema | Phase 2 | Completed | Ionis Pharmaceuticals, Inc. | 20 | 2020-03-31 | 2026-05-08 |
| A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema Donidalorsen, Placebo - Hereditary Angioedema | Phase 2 | Completed | Ionis Pharmaceuticals, Inc. | 23 | 2020-01-07 | 2023-04-03 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers Placebo, Donidalorsen - Healthy Volunteers | Phase 1 | Completed | Ionis Pharmaceuticals, Inc. | 32 | 2017-08-28 | 2022-08-04 |
| Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema Donidalorsen - Hereditary Angioedema | Approved for marketing | Ionis Pharmaceuticals, Inc. | 2026-09-04 |
Ionis Pharmaceuticals, Inc. (7)
United States (5) · Netherlands (4) · Canada (3) · Italy (3) · Poland (3) · Spain (3) · Belgium (2) · Bulgaria (2) · France (2) · Germany (2) · Israel (2) · Turkey (Türkiye) (2) · United Kingdom (2) · Australia (1) · Denmark (1)
PMID 35294812, PMID 38009241, PMID 38819395, PMID 40673861, PMID 41208217, PMID 41767175, PMID 41802598
2019 (1), 2025 (1)