Rare Disease Phase 3 Readout Calendar
Industry-sponsored rare disease trials with primary completion in the next 12 months or last 12 months.
Data last updated September 20, 2026 · source: ClinicalTrials.gov
What you can answer with this page
A confidence-graded calendar of every industry Phase 3 rare disease trial reading out in the next 12 months — with a per-row staleness flag so you know which completion dates are still credible.
Recently read-out results are free; the upcoming calendar shows a free preview. See the full cross-area calendar → or the Rare Disease landscape →
Recently read out & awaiting topline — Rare Disease
· 6Phase 3 rare disease trials whose primary endpoint completed in the last 12 months — results posted, or completed and awaiting topline. Most recent first.
| Sponsor | Trial / Asset | Indication | Status | Primary completion | Confidence | NCT |
|---|---|---|---|---|---|---|
| argenx | A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP). Asset: efgartigimod | Primary Immune Thrombocytopenia | Completed | Q1 2026 2026-03-11 | Completed · awaiting
Updated 6mo ago
| NCT04225156 |
| Amgen | Study of Romiplostim for Chemotherapy-induced Thrombocytopenia in Adult Subjects With Non-small Cell Lung Cancer (NSCLC), Ovarian Cancer, or Breast Cancer Asset: Romiplostim | Chemotherapy-induced Thrombocytopenia | Active Not Recruiting | Q1 2026 2026-03-10 | Completed · awaiting
Updated 5mo ago
| NCT03937154 |
| PTC Therapeutics | Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) Asset: Ataluren | Duchenne Muscular Dystrophy | Completed | Q1 2026 2026-02-10 | Completed · awaiting
Updated 6mo ago
| NCT01247207 |
| Forma Therapeutics, Inc. | A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS) Asset: Etavopivat Tablets Low dose | Sickle Cell Disease | Active Not Recruiting | Q1 2026 2026-02-06 | Completed · awaiting
Updated 5mo ago
| NCT04624659 |
| Sanofi | A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Asset: Venglustat (GZ402671) | Fabry Disease | Active Not Recruiting | Q4 2025 2025-12-17 | Completed · awaiting
Updated 7mo ago
| NCT05206773 |
| Agios Pharmaceuticals, Inc. | A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) Asset: Mitapivat | Sickle Cell Disease | Active Not Recruiting | Q4 2025 2025-10-30 | Completed · awaiting
Updated 5mo ago
| NCT05031780 |
Upcoming rare disease readouts — next 12 months
· 29Industry Phase 3 rare disease trials with primary completion ahead, soonest first — confidence-graded by date type and CT.gov staleness.
| Sponsor | Trial / Asset | Indication | Status | Primary completion | Confidence | NCT |
|---|---|---|---|---|---|---|
| Jiangsu BioJeTay Biotechnology Co.… | Phase III Clinical Trial of STSP-0601 for Injection in Hemophilia Patients Asset: STSP-0601 for Injection | Hemophilia | Active Not Recruiting | Q3 2026 2026-09 (est.) | Estimated · fresh
Updated 5mo ago
| NCT06922045 |
| argenx | A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia Asset: efgartigimod PH20 SC | Primary Immune Thrombocytopenia | Active Not Recruiting | Q4 2026 2026-10-01 (est.) | Estimated · aging
Updated 8mo ago
| NCT04812925 |
| Suzhou Alphamab Co., Ltd. | Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B Without Inhibitors Asset: KN057 | Hemophilia A Without Inhibitor +1 | Active Not Recruiting | Q4 2026 2026-10-15 (est.) | Estimated · aging
Updated 12mo ago
| NCT06569108 |
| CSL Behring | Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Participants With Hemophilia A Previously Treated With FVIII Products Asset: Recombinant single-chain factor VIII (rVIII-SingleChain) | Congenital Hemophilia A | Active Not Recruiting | Q4 2026 2026-10-26 (est.) | Estimated · fresh
Updated 5mo ago
| NCT06738485 |
| Novartis Pharmaceuticals | Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP) Asset: Ianalumab | Primary Immune Thrombocytopenia (ITP) | Active Not Recruiting | Q4 2026 2026-10-29 (est.) | Estimated · fresh
Updated 5mo ago
| NCT05653349 |
About this calendar
Dates anchor on the ClinicalTrials.gov primary completion date — the only public, structured signal — and are graded by date type (actual vs sponsor-estimated) and how recently the record was updated. Sponsor-estimated dates can slip silently, so treat each as a current target rather than a guarantee. See the full readout calendar for all areas and filters.