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 July 17, 2026 · source: ClinicalTrials.gov
All areas OncologyImmunologyMetabolicCNSCardiovascularInfectious DiseaseRespiratoryRare DiseaseOphthalmologyDermatologyMusculoskeletalRenalPain
22
Next 6 months
32
Next 12 months
13
Results posted
109
Phase 3 Rare Disease
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.
What rare disease trials are reading out soon?
The "upcoming" section shows industry-sponsored Phase 3 rare disease trials with primary completion ahead, soonest first, each graded by how credible that date is.
Which rare disease trials have already read out?
The "recently read out" section shows Phase 3 rare disease trials whose primary endpoint completed in the last 12 months — results posted, or completed and awaiting topline.
How are these readout dates sourced?
Every date is the ClinicalTrials.gov primary completion date — the only public, structured signal. We grade each by whether it is actual or sponsor-estimated, and by how recently the trial record was updated.
Where can I see readouts for other therapeutic areas?
The full cross-area calendar is on the main readout calendar; this page is the Rare Disease slice of it.
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 4mo 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 3mo 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 4mo 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 3mo 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 5mo 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 3mo ago
| NCT05031780 |
Upcoming rare disease readouts — next 12 months
· 32Industry 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 |
|---|---|---|---|---|---|---|
| Biogen | A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD) Asset: Nusinersen | Muscular Atrophy, Spinal | Active Not Recruiting | Q3 2026 2026-07-31 (est.) | Estimated · fresh
Updated 5mo ago
| NCT04729907 |
| Qilu Pharmaceutical Co., Ltd. | QL0911 for the Treatment of Cancer Treatment-Induced Thrombocytopenia Asset: QL0911 | Cancer Treatment-Induced Thrombocytopenia | Recruiting | Q3 2026 2026-07 (est.) | Estimated · fresh
Updated 6mo ago
| NCT06456528 |
| SK Plasma Co., Ltd. | An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients Asset: SKP-0141 | Hemophilia A, Severe | Not Yet Recruiting | Q3 2026 2026-07-31 (est.) | Estimated · stale
Updated 19mo ago
| NCT06738901 |
| Genzyme, a Sanofi Company | Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B Asset: Fitusiran | Hemophilia | Active Not Recruiting | Q3 2026 2026-08-15 (est.) | Estimated · aging
Updated 11mo ago
| NCT03974113 |
| Kedrion S.p.A. | A Study Investigating Intravenous Human Normal Immunoglobulin 10% in Adults With Chronic Immune Thrombocytopenia (ITP) Asset: Kedrion IVIG 10% | Chronic Primary Immune Thrombocytopenia (ITP) | Recruiting | Q3 2026 2026-08 (est.) | Estimated · fresh
Updated 4mo ago
| NCT07059000 |
| 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 3mo ago
| NCT05653349 |
Unlock 26 more rare disease readouts · plus filters on the full calendar
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.