Clinical Trials
13
Trial Phases
2 Phases
Drug Approvals
0
Drug Approvals
No drug approvals found
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Clinical Trials
Distribution across different clinical trial phases (13 trials with phase data)โข Click on a phase to view related trials
An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)
- First Posted Date
- 2023-05-30
- Last Posted Date
- 2024-12-09
- Lead Sponsor
- Neuren Pharmaceuticals Limited
- Target Recruit Count
- 20
- Registration Number
- NCT05879614
- Locations
- ๐บ๐ธ
Rady Children's Hospital San Diego, San Diego, California, United States
๐บ๐ธRare Disease Research, Atlanta, Georgia, United States
๐บ๐ธUncommon Cures, Chevy Chase, Maryland, United States
An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
- First Posted Date
- 2021-08-27
- Last Posted Date
- 2025-06-03
- Lead Sponsor
- Neuren Pharmaceuticals Limited
- Target Recruit Count
- 18
- Registration Number
- NCT05025241
- Locations
- ๐บ๐ธ
Rush University Medical Center, Chicago, Illinois, United States
๐บ๐ธMassachusetts General Hospital, Boston, Massachusetts, United States
๐บ๐ธBoston Children's Hospital, Boston, Massachusetts, United States
An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)
- First Posted Date
- 2021-08-27
- Last Posted Date
- 2025-06-18
- Lead Sponsor
- Neuren Pharmaceuticals Limited
- Target Recruit Count
- 28
- Registration Number
- NCT05025332
- Locations
- ๐บ๐ธ
University of Alabama at Birmingham, Birmingham, Alabama, United States
๐บ๐ธUniversity of California at San Francisco, San Francisco, California, United States
๐บ๐ธChildren's Hospital Colorado, Aurora, Colorado, United States
An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
- First Posted Date
- 2021-08-18
- Last Posted Date
- 2025-01-31
- Lead Sponsor
- Neuren Pharmaceuticals Limited
- Target Recruit Count
- 17
- Registration Number
- NCT05011851
- Locations
- ๐ฆ๐บ
Sydney Children's Hospital, Randwick, New South Wales, Australia
๐ฆ๐บCentre for Clinical Trials in Rare Neurodevelopmental Disorders at Children's Health Queensland Hospital and Health Service, South Brisbane, Queensland, Australia
๐ฆ๐บAustin Health, Heidelberg, Victoria, Australia
To Evaluate the Safety, Tolerability and Pharmacokinetics of Oral NNZ-2591 in Healthy Volunteers
- First Posted Date
- 2020-05-08
- Last Posted Date
- 2021-07-09
- Lead Sponsor
- Neuren Pharmaceuticals Limited
- Target Recruit Count
- 28
- Registration Number
- NCT04379869
- Locations
- ๐ฆ๐บ
Scientia Clinical Research, Sydney, New South Wales, Australia
๐ฆ๐บLinear Clinical Research, The Queen Elizabeth II Medical Centre, Nedlands, Western Australia, Australia
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News
Ionis Advances Antisense Therapy for Angelman Syndrome to Phase 3 Following Promising Clinical Results
Ionis Pharmaceuticals is moving forward with Phase 3 trials for ION582, an antisense therapy designed to unsilence the paternal UBE3A gene in Angelman syndrome patients, after Biogen declined to license the drug.
FDA Grants Fast Track Status to Neuren's Novel Pitt Hopkins Syndrome Treatment
Neuren Pharmaceuticals receives FDA Fast Track designation for their investigational treatment targeting Pitt Hopkins syndrome, a rare neurodevelopmental disorder affecting 1 in 34,000-41,000 people.
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