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NNZ-2591 for Prader-Willi Syndrome (PWS-001 Trial)

Phase 2

Recruiting

Research Sponsored by Neuren Pharmaceuticals Limited

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Clinical diagnosis of PWS with a documented disease-causing genetic abnormality of the chromosome 15q11-q13 confirmed by DNA methylation and microarray

Body weight of 12 kg to 100kg (inclusive) at Baseline

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 13 weeks

Awards & highlights

PWS-001 Trial Summary

This trial explores the safety and effectiveness of a drug to treat Prader-Willi Syndrome in kids and teens.

Who is the study for?

Children with Prader-Willi Syndrome aged 4-12, who are on growth hormone treatment and can swallow liquid medication. They must weigh between 12 kg to 100kg, have a confirmed genetic diagnosis of PWS, and live in the US for at least 3 months. Exclusions include severe health conditions like heart or liver disease, recent COVID hospitalization, planned surgery during the study period, or uncontrolled diabetes.Check my eligibility

What is being tested?

The trial is testing NNZ-2591's safety and how well it's tolerated when taken orally by children with Prader-Willi Syndrome. It also looks at how the body processes the drug (pharmacokinetics) and its effectiveness in improving symptoms of PWS.See study design

What are the potential side effects?

While specific side effects for NNZ-2591 aren't listed here, common ones may include digestive issues like nausea or diarrhea; allergic reactions; changes in appetite or weight; fatigue; mood swings; skin rashes; headaches; sleep disturbances.

PWS-001 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My condition is confirmed Prader-Willi Syndrome with a specific genetic abnormality.

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My weight is between 12 kg and 100 kg.

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I am currently receiving growth hormone treatment.

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I can swallow liquid medication.

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I am between 4 and 12 years old.

PWS-001 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 13 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~13 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 13 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Pharmacokinetic - Measurement of AUC

Pharmacokinetic - Measurement of Cmax

Pharmacokinetic - Measurement of t1/2

+2 more

Secondary outcome measures

Exploratory efficacy measurement

PWS-001 Trial Design

1Treatment groups

Experimental Treatment

Group I: NNZ-2591Experimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

NNZ-2591

2020

Completed Phase 1

~30

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Neuren Pharmaceuticals LimitedLead Sponsor

12 Previous Clinical Trials

926 Total Patients Enrolled

James ShawStudy DirectorNeuren Pharmaceuticals

4 Previous Clinical Trials

88 Total Patients Enrolled

Jordan PressStudy DirectorNeuren Pharmaceuticals

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this medical research include adults aged 35 and over?

"The age range for eligibility in this trial is 4 to 12 years old."

Answered by AI

Does this research endeavor currently have any spots open for participation?

"As per the clinicaltrials.gov information, this experiment is currently enlisting participants. It was initially made available on September 1st 2023 and has been revised as recently as that same date."

Answered by AI

How many participants are enrolled in the current trial?

"Correct. According to clinicaltrials.gov, this trial is currently seeking participants; it was initially posted on September 1st 2023 and has since been updated recently. The research seeks the recruitment of 20 individuals at a single medical centre."

Answered by AI

What are the eligibility criteria for those seeking to join this clinical experiment?

"To be accepted into this research, participants must display a diagnosis of prader-willi syndrome and fall between the ages of 4 to 12. The total number of patients admitted is capped at 20 people."

Answered by AI

Does the Food and Drug Administration recognize NNZ-2591 as a safe treatment?

"There is some existing evidence that suggests NNZ-2591's safety, so we have ranked it a 2. However, no clinical data exists to support its efficacy yet."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2023. "An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05879614.