An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)

NCT05879614 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: NEU-2591-PWS-001
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 4

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.

Conditions

Prader-Willi Syndrome

Keywords

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (5)

• Safety and Tolerability

  To examine the incidence, severity and frequency of adverse events (AEs), including serious adverse events (SAEs) during treatment with NNZ-2591.

  13 weeks

• Pharmacokinetic - Measurement of Cmax

  Maximum observed concentration (Cmax) of NNZ-2591

  13 weeks

• Pharmacokinetic - Measurement of AUC

  Area under the concentration-time curve of NNZ-2591

  13 weeks

• Pharmacokinetic - Measurement of time to Cmax

  Time to Cmax of NNZ-2591

  13 weeks

• Pharmacokinetic - Measurement of t1/2

  Apparent terminal elimination half-life of NNZ-2591

  13 weeks

Secondary Outcomes (17)

• Exploratory efficacy measurement

  13 weeks

• Exploratory efficacy measurement

  13 weeks

• Exploratory efficacy measurement

  13 weeks

• Exploratory efficacy measurement

  13 weeks

• Exploratory efficacy measurement

  13 weeks

Study Arms (1)

NNZ-2591

EXPERIMENTAL

NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.

Drug: NNZ-2591

Interventions

NNZ-2591 DRUG

NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.

Also known as: Cyclo-L-Glycyl-L-2-Allylproline

NNZ-2591

Eligibility Criteria

• Age: 4 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• Body weight \<12 kg or \>100 kg at Baseline.
• HbA1c values above 7% at the Screening visit.
• Clinically significant abnormalities in safety laboratory tests and vital signs at Screening.
• Positive pregnancy test at the Screening visit.
• Positive drugs of abuse screen not explained by concomitant medications.
• Abnormal QTcF interval or prolongation at Screening.
• Any other clinically significant finding on ECG at the Screening visit.
• Positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at Screening or Baseline.
• Previous COVID 19 infection with last 12 months that required hospitalization.
• Previous COVD-19 infection involving multi-organ systems, resulting in Multisystem Inflammatory Syndrome in Children (MIS-C) or with clinically significant long term effects.
• COVID-19 infection associated with acute kidney injury (AKI) or renal conditions.
• Renal conditions or abnormalities identified in laboratory testing, imaging or medical history.
• Liver conditions and Hepatic abnormalities.
• Vision abnormalities and Ocular conditions.
• Excluded concomitant treatments.

Sponsors & Collaborators

• Neuren Pharmaceuticals Limited: lead

Study Sites (4)

Rady Children's Hospital San Diego

San Diego, California, 92123, United States

Location

Rare Disease Research

Atlanta, Georgia, 30329, United States

Location

Uncommon Cures

Chevy Chase, Maryland, 20815, United States

Location

Suburban Research

Media, Pennsylvania, 19063, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

cyclo-L-glycyl-L-2-allylproline

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Study Officials

• Jordan Press

  Neuren Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 18, 2023
• First Posted: May 30, 2023
• Study Start: September 1, 2023
• Primary Completion: June 30, 2025
• Study Completion: June 30, 2025
• Last Updated: December 9, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)