3 recruiting, 2 completed, 2 unknown, 1 active not recruiting. 5 Phase 2, 3 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 2, Unknown 2, Recruiting 1 |
| Phase 1 | 3 | Recruiting 2, Unknown 1 |
Follow Pitt Hopkins Syndrome: new trials by email Literature Review of Pitt Hopkins Syndrome trials Research Report on Pitt Hopkins Syndrome
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 |
|---|---|---|---|---|---|---|
| An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome Vorinostat (SAHA), Placebo - Pitt Hopkins Syndrome | Phase 1 | Recruiting | Unravel Biosciences, Inc. | 5 | 2026-03-15 | 2026-09-17 |
| Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome MZ-1866 - Pitt Hopkins Syndrome | Phase 1 / Phase 2 | Recruiting | Mahzi Therapeutics | 12 | 2025-12-19 | 2026-07-31 |
| Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders MTP-101P, Vancomycin, Magnesium Citrate - Pitt Hopkins Syndrome | Phase 2 | Unknown | Gut-Brain-Axis Therapeutics Inc. | 20 | 2024-02-27 | 2024-03-20 |
| An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001) NNZ-2591 - Pitt Hopkins Syndrome | Phase 2 | Completed | Neuren Pharmaceuticals Limited | 28 | 2022-10-14 | 2025-06-18 |
| Safety & Efficacy of AlloRx SC® in PTHS Patients AlloRx Stem Cells®, Placebo control - Pitt Hopkins Syndrome | Phase 1 / Phase 2 | Unknown | Vitro Biopharma Inc. | 26 | 2021-12-01 | 2021-12-21 |
| MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders vancomycin, magnesium citrate, microbiota, placebo vancomycin, real magnesium citrate, placebo microbiota - Pitt Hopkins Syndrome | Phase 2 | Completed | Arizona State University | 6 | 2019-09-30 | 2024-08-26 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford Rare Disorders, Undiagnosed Disorders | Recruiting | Sanford Health | 20000 | 2010-07-01 | 2025-05-29 |
Pitt-Hopkins syndrome (8) · Ataxia with vitamin E deficiency (2) · Cystinosis (2) · Hyperargininemia (2) · Hypophosphatasia (2) · Leukodystrophy, Metachromatic (2) · Multiple Endocrine Neoplasia Type 2b (2) · Stickler syndrome, type 1 (2) · Stickler syndrome, type 2 (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1) · 3-Methylglutaconic Aciduria, Type V (1) · 3-methylcrotonyl CoA carboxylase 1 deficiency (1) · 3-methylcrotonyl CoA carboxylase 2 deficiency (1) · Achalasia Addisonianism Alacrimia syndrome (1)
Abnormalities, Multiple (2) · Amino Acid Metabolism, Inborn Errors (2) · Anemia (2) · Anemia, Hemolytic (2) · Aortic Valve Disease (2) · Aortic Valve Stenosis (2) · Basal Ganglia Diseases (2) · Blood Platelet Disorders (2) · Bone Diseases (2) · Bone Diseases, Developmental (2) · Bone Diseases, Metabolic (2) · Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Carbohydrate Metabolism, Inborn Errors (2)
Vancomycin (2) · magnesium citrate (2) · Antacids (1) · Microbiota (1) · Vorinostat (1) · cyclo-L-glycyl-L-2-allylproline (1)
Arizona State University (1) · Gut-Brain-Axis Therapeutics Inc. (1) · Mahzi Therapeutics (1) · Neuren Pharmaceuticals Limited (1) · RTI International (1) · Sanford Health (1) · Unravel Biosciences, Inc. (1) · Vitro Biopharma Inc. (1)
United States (6) · Australia (1) · Colombia (1) · Israel (1)
Curt Scharfe, MD, PhD (1) · James Shaw (1) · Neal I Muni, M.D., MSPH (1)