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History of Changes for Study: NCT05879614
An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001) (PWS-001)
Latest version (submitted April 10, 2024) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 May 18, 2023 None (earliest Version on record)
2 September 1, 2023 Recruitment Status, Study Status, Contacts/Locations and Oversight
3 January 8, 2024 Contacts/Locations and Study Status
4 February 15, 2024 Study Status and Contacts/Locations
5 April 10, 2024 Contacts/Locations and Study Status
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Changes (Merged) for Study: NCT05879614
May 18, 2023 (v1) -- February 15, 2024 (v4)

Changes in: Study Status, Oversight and Contacts/Locations

Open or close this module Study Identification
Unique Protocol ID: NEU-2591-PWS-001
Brief Title: An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001) (PWS-001)
Official Title: An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)
Secondary IDs:
Open or close this module Study Status
Record Verification: May 2023 February 2024
Overall Status: Not yet recruiting Recruiting
Study Start: June 30, 2023 September 1, 2023
Primary Completion: June 30, 2024 [Anticipated]
Study Completion: June 30, 2024 [Anticipated]
First Submitted: May 18, 2023
First Submitted that
Met QC Criteria:
May 18, 2023
First Posted: May 30, 2023 [Actual]
Last Update Submitted that
Met QC Criteria:
May 18, 2023 February 15, 2024
Last Update Posted: May 30 February 20, 2023 2024 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: Neuren Pharmaceuticals Limited
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: Yes
U.S. FDA-regulated Device: No
Data Monitoring: Yes
Open or close this module Study Description
Brief Summary: A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Prader-Willi Syndrome.
Detailed Description: The primary purpose of this study is to investigate the safety, tolerability and pharmacokinetics of treatment with NNZ-2591 oral solution in children and adolescents with Prader-Willi Syndrome. The secondary purpose is to investigate measures of efficacy. Subjects will receive treatment with NNZ-2591 oral solution (50 mg/mL) doses for a total of 13 weeks.
Open or close this module Conditions
Conditions: Prader-Willi Syndrome
Keywords: Prader-Willi Syndrome
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 2
Interventional Study Model: Single Group Assignment
Number of Arms: 1
Masking: None (Open Label)
Allocation: N/A
Enrollment: 20 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.
Drug: NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.
Other Names:
  • Cyclo-L-Glycyl-L-2-Allylproline
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Safety and Tolerability
[ Time Frame: 13 weeks ]

To examine the incidence, severity and frequency of adverse events (AEs), including serious adverse events (SAEs) during treatment with NNZ-2591.
2. Pharmacokinetic - Measurement of Cmax
[ Time Frame: 13 weeks ]

Maximum observed concentration (Cmax) of NNZ-2591
3. Pharmacokinetic - Measurement of AUC
[ Time Frame: 13 weeks ]

Area under the concentration-time curve of NNZ-2591
4. Pharmacokinetic - Measurement of time to Cmax
[ Time Frame: 13 weeks ]

Time to Cmax of NNZ-2591
5. Pharmacokinetic - Measurement of t1/2
[ Time Frame: 13 weeks ]

Apparent terminal elimination half-life of NNZ-2591
Secondary Outcome Measures:
1. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by PWS-specific Clinical Global Impression Scale & Domain -Overall Improvement Score (CGI-I)
2. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Caregiver Global Impression-Change Score
3. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by PWS-specific Clinical Global Impression Scale-Severity (CGI-S) Overall Score
4. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Caregiver Top 3 Concerns Likert Scale Scores
5. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by PWS Profile Score
6. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by PWS Anxiousness and Distress Behaviors Questionnaire Score (PADQ)
7. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Autism Diagnostic Observation Schedule (ADOS-2), Repetitive behaviors and Social scores
8. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Hyperphagia Questionnaire-Clinical Trials (HQ-CT) Score
9. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Food Safety Zone Questionnaire Score
10. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Vineland Adaptive Behavior Scales-3 Growth Scale Scores
11. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Zarit Burden Interview Score
12. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Child Sleep Habits Questionnaire (CSHQ)
13. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Impact of Childhood Neurological Disability Scale (ICND)
14. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Quality of Life Inventory-Disability (QI-Disability)
15. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Kaufman Brief Intelligence Test or Mullen Scales of Early Learning
16. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by PWS Suicidality Assessment
17. Exploratory efficacy measurement
[ Time Frame: 13 weeks ]

Assessed by Caregiver Diary
Open or close this module Eligibility
Minimum Age: 4 Years
Maximum Age: 12 Years
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

  1. Clinical diagnosis of PWS with a documented disease-causing genetic abnormality of the chromosome 15q11-q13 confirmed by DNA methylation and microarray.
  2. Males or females aged 4-12 years, inclusive.
  3. Body weight of 12 kg to 100kg (inclusive) at Baseline.
  4. Subjects with a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at the Screening visit.
  5. Must currently be on treatment with growth hormone.
  6. Each subject must be able to swallow the study medication provided as a liquid solution.
  7. Caregiver(s) must have sufficient English language skills.
  8. Subject and caregiver must reside in the US and have been resident in the US for at least 3 months prior to screening.

Exclusion Criteria:

  1. Body weight <12 kg or >100 kg at Baseline.
  2. HbA1c values above 7% at the Screening visit.
  3. Clinically significant abnormalities in safety laboratory tests and vital signs at Screening.
  4. Positive pregnancy test at the Screening visit.
  5. Positive drugs of abuse screen not explained by concomitant medications.
  6. Abnormal QTcF interval or prolongation at Screening.
  7. Any other clinically significant finding on ECG at the Screening visit.
  8. Positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at Screening or Baseline.
  9. Previous COVID 19 infection with last 12 months that required hospitalization.
  10. Previous COVD-19 infection involving multi-organ systems, resulting in Multisystem Inflammatory Syndrome in Children (MIS-C) or with clinically significant long term effects.
  11. COVID-19 infection associated with acute kidney injury (AKI) or renal conditions.
  12. Renal conditions or abnormalities identified in laboratory testing, imaging or medical history.
  13. Liver conditions and Hepatic abnormalities.
  14. Vision abnormalities and Ocular conditions.
  15. Excluded concomitant treatments.
  16. Unstable seizure profile.
  17. Current clinically significant cardiovascular, gastrointestinal, or respiratory disease, or clinically significant organ impairment, or endocrine disease with the exception of obesity and controlled hypothyroidism.
  18. Current clinically significant hypo or hyperthyroidism, Type 1 or Type 2 diabetes mellitus requiring insulin (whether well controlled or uncontrolled), or uncontrolled Type 1 or Type 2 diabetes.
  19. Has planned surgery during the study.
  20. History of, or current, cerebrovascular disease or brain trauma.
  21. History of, or current catatonia or catatonia-like symptoms.
  22. History of, or current, malignancy.
  23. Current major or persistent depressive disorder (including bipolar depression).
  24. Significant uncorrected hearing impairment.
  25. Allergy to strawberry.
  26. Has participated in another interventional clinical study within 30 days prior to start of Screening.
  27. Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study.
Open or close this module Contacts/Locations
Central Contact Person: James Shaw
Telephone: +61 427 299 669
Email: jshaw@neurenpharma.com
Henry Nickson
Telephone: +1 (470) 883-2822
Email: PWStrialreferral@precisionformedicine.com
Study Officials: James Shaw Jordan Press
Study Director
Neuren Pharmaceuticals
Locations: United States, Georgia
Rare Disease Research
[Recruiting]
Atlanta, Georgia, United States, 30329
Contact:Contact: 470-883-2822 PWStrialreferral@precisionformedicine.com
Contact:Principal Investigator: Han C Phan, MD
United States, Maryland
Uncommon Cures
[Recruiting]
Chevy Chase, Maryland, United States, 20815
Contact:Contact: 470-883-2822 PWStrialreferral@precisionformedicine.com
Contact:Principal Investigator: Roshan Lal, MD
United States, Pennsylvania
Suburban Research
[Recruiting]
Media, Pennsylvania, United States, 19063
Contact:Contact: 470-883-2822 PWStrialreferral@precisionformedicine.com
Contact:Principal Investigator: Shivkumar Hatti, MD
Open or close this module IPDSharing
Plan to Share IPD: No
Open or close this module References
Citations:
Links:
Available IPD/Information:

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