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Prader-Willi SyndromePhase 2PWS-001NCT05879614

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

A Phase 2 study testing NNZ-2591 in people with Prader-Willi Syndrome, run by Neuren Pharmaceuticals Limited at 4 sites in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Prader-Willi Syndrome. Read more about this study

Who is it for?
Ages 4 to 12 years
Conditions the study is for
Prader-Willi Syndrome
What is being tested?
NNZ-2591
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
4 sitesUnited States
Study dates
1 Sep 2023to 30 Jun 2025
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 to 12 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.

Who cannot join

  • 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.
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  • 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.
  • Unstable seizure profile.
  • Current clinically significant cardiovascular, gastrointestinal, or respiratory disease, or clinically significant organ impairment, or endocrine disease with the exception of obesity and controlled hypothyroidism.
  • 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.
  • Has planned surgery during the study.
  • History of, or current, cerebrovascular disease or brain trauma.
  • History of, or current catatonia or catatonia-like symptoms.
  • History of, or current, malignancy.
  • Current major or persistent depressive disorder (including bipolar depression).
  • Significant uncorrected hearing impairment.
  • Allergy to strawberry.
  • Has participated in another interventional clinical study within 30 days prior to start of Screening.
  • Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study.

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Rare Disease Research

    Atlanta, Georgia, 30329

  • Uncommon Cures

    Chevy Chase, Maryland, 20815

  • Suburban Research

    Media, Pennsylvania, 19063

  • Rady Children's Hospital San Diego

    San Diego, California, 92123

Other trials in United States

About this study

Official title: An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)

A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Prader-Willi Syndrome.

Read the 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.

Being tested
NNZ-2591
Keywords
Prader-Willi Syndrome
Medical subject headings
Prader-Willi Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NNZ-2591Drug

    Also called Cyclo-L-Glycyl-L-2-Allylproline

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

Study groups in NCT05879614
Study groupRoleWhat they receive
NNZ-2591ExperimentalNNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.
Receives: Drug: NNZ-2591

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety and Tolerability

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

    Measured: 13 weeks

  2. Pharmacokinetic - Measurement of Cmax

    Maximum observed concentration (Cmax) of NNZ-2591

    Measured: 13 weeks

  3. Pharmacokinetic - Measurement of AUC

    Area under the concentration-time curve of NNZ-2591

    Measured: 13 weeks

  4. Pharmacokinetic - Measurement of time to Cmax

    Time to Cmax of NNZ-2591

    Measured: 13 weeks

  5. Pharmacokinetic - Measurement of t1/2

    Apparent terminal elimination half-life of NNZ-2591

    Measured: 13 weeks

Secondary outcomes (17)

  1. Exploratory efficacy measurement

    Assessed by PWS-specific Clinical Global Impression Scale \& Domain -Overall Improvement Score (CGI-I)

    Measured: 13 weeks

  2. Exploratory efficacy measurement

    Assessed by Caregiver Global Impression-Change Score

    Measured: 13 weeks

  3. Exploratory efficacy measurement

    Assessed by PWS-specific Clinical Global Impression Scale-Severity (CGI-S) Overall Score

    Measured: 13 weeks

  4. Exploratory efficacy measurement

    Assessed by Caregiver Top 3 Concerns Likert Scale Scores

    Measured: 13 weeks

  5. Exploratory efficacy measurement

    Assessed by PWS Profile Score

    Measured: 13 weeks

  6. Exploratory efficacy measurement

    Assessed by PWS Anxiousness and Distress Behaviors Questionnaire Score (PADQ)

    Measured: 13 weeks

  7. Exploratory efficacy measurement

    Assessed by Autism Diagnostic Observation Schedule (ADOS-2), Repetitive behaviors and Social scores

    Measured: 13 weeks

  8. Exploratory efficacy measurement

    Assessed by Hyperphagia Questionnaire-Clinical Trials (HQ-CT) Score

    Measured: 13 weeks

  9. Exploratory efficacy measurement

    Assessed by Food Safety Zone Questionnaire Score

    Measured: 13 weeks

  10. Exploratory efficacy measurement

    Assessed by Vineland Adaptive Behavior Scales-3 Growth Scale Scores

    Measured: 13 weeks

  11. Exploratory efficacy measurement

    Assessed by Zarit Burden Interview Score

    Measured: 13 weeks

  12. Exploratory efficacy measurement

    Assessed by Child Sleep Habits Questionnaire (CSHQ)

    Measured: 13 weeks

  13. Exploratory efficacy measurement

    Assessed by Impact of Childhood Neurological Disability Scale (ICND)

    Measured: 13 weeks

  14. Exploratory efficacy measurement

    Assessed by Quality of Life Inventory-Disability (QI-Disability)

    Measured: 13 weeks

  15. Exploratory efficacy measurement

    Assessed by Kaufman Brief Intelligence Test or Mullen Scales of Early Learning

    Measured: 13 weeks

  16. Exploratory efficacy measurement

    Assessed by PWS Suicidality Assessment

    Measured: 13 weeks

  17. Exploratory efficacy measurement

    Assessed by Caregiver Diary

    Measured: 13 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 May 2023
  2. Study start1 Sep 2023
  3. Primary completion30 Jun 2025 (estimated)
  4. Study completion30 Jun 2025 (estimated)

Why it stopped: Revised development strategy

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05879614
Sponsor's protocol ID
NEU-2591-PWS-001
Acronym
PWS-001
Status last verified
Dec 2024
First submitted
18 May 2023
Last updated
9 Dec 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing NNZ-2591 in people with Prader-Willi Syndrome, run by Neuren Pharmaceuticals Limited at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 9 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety and Tolerability (time frame: 13 weeks). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05879614.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Dec 2024.

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