4 completed, 1 withdrawn. 3 Phase 2, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 3 | Completed 2, Withdrawn 1 |
| Phase 1 | 1 | Completed 1 |
| Phase 3 | 1 | Completed 1 |
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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 |
|---|---|---|---|---|---|---|
| Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose Trehalose - Oculopharyngeal Muscular Dystrophy | Phase 2 | Withdrawn | Bioblast Pharma Ltd. | 2017-06-15 | 2020-01-13 | |
| A Study to Establish Safety and Maximum Tolerated Dose of IV Trehalose in Healthy Subjects Trehalose for IV Infusion, Saline 0.9% IV - Healthy Volunteer Subjects | Phase 1 | Completed | Bioblast Pharma Ltd. | 24 | 2016-03-01 | 2016-11-22 |
| Continuation Protocol to Protocol BBCO-001 Tehalose 30gr - Muscular Dystrophy, Oculopharyngeal (OPMD) | Phase 3 | Completed | Bioblast Pharma Ltd. | 9 | 2015-01-01 | 2019-07-17 |
| Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease Cabaletta for IV infusion once weekly during 24 weeks - Machado-Joseph Disease / Spinocerebellar Ataxia 3 | Phase 2 | Completed | Bioblast Pharma Ltd. | 15 | 2014-07-01 | 2016-11-22 |
| Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients Cabaletta - Oculopharyngeal Muscular Dystrophy | Phase 2 | Completed | Bioblast Pharma Ltd. | 25 | 2014-02-01 | 2017-10-17 |
Muscular Dystrophy, Oculopharyngeal (3) · Deglutition Disorders (1) · Machado-Joseph Disease (1) · Muscular Dystrophies (1)
Trehalose (2) · Infusions, Intravenous (1) · Sodium Chloride (1)
Canada (3) · Israel (2) · United States (2)
Bernard Brais, MD (2) · Bernard Brais (1) · Carlos Gordon, Prof. (1) · Yoseph Caraco, M.D (1)