What is the mechanism of action of this treatment?

Common treatments for Prader-Willi Syndrome (PWS) include growth hormone therapy, which primarily works by increasing levels of insulin-like growth factor 1 (IGF-1). IGF-1 plays a crucial role in growth and development, and its modulation can help improve muscle mass, reduce body fat, and enhance physical strength in PWS patients. This is particularly important for PWS patients who often suffer from hypotonia (low muscle tone), obesity, and short stature. By targeting IGF-1 activity, treatments like NNZ-2591 aim to address these core symptoms, potentially improving overall quality of life and physical health for individuals with PWS.

What side effects are associated with this type of intervention?

Common treatments for Prader-Willi Syndrome often include growth hormone therapy, which can have side effects such as edema, joint pain, and insulin resistance. Treatments modulating IGF-1 activity, like NNZ-2591, may similarly cause side effects related to growth hormone pathways, including potential impacts on glucose metabolism and fluid retention. Other general treatments for PWS, such as appetite suppressants and behavioral therapies, can also have side effects like gastrointestinal discomfort and mood changes.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved treatments for Prader-Willi Syndrome that focus on the modulation of IGF-1 activity similar to NNZ-2591. The primary FDA-approved treatment for PWS is growth hormone therapy, which helps improve growth, body composition, and physical strength. Other treatments are symptomatic and include interventions for hyperphagia, behavioral issues, and hormone deficiencies.

What other types of research exist?

Promising novel alternatives to NNZ-2591 for Prader-Willi Syndrome patients include vosoritide, a C-type natriuretic peptide analogue, which has shown efficacy in related conditions like achondroplasia. Additionally, targeting pathways such as the caspase cascade and endoplasmic reticulum stress response, as seen with NNU219 in multiple myeloma, could offer new therapeutic avenues. Further research into these and other emerging treatments is warranted to determine their potential benefits for PWS patients.

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

Phase 2

Waitlist Available

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

Must not have

Significant uncorrected hearing impairment

History of, or current, malignancy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 13 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a drinkable medicine called NNZ-2591 in children and teenagers with Prader-Willi Syndrome. The goal is to see if it is safe and effective. The treatment lasts for a few months.

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.

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have significant hearing loss that hasn't been treated.

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I have or had cancer before.

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I have had a stroke or brain injury.

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I had COVID-19 that led to MIS-C or long-term health issues.

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I have kidney problems due to a COVID-19 infection.

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I do not have serious thyroid issues or diabetes needing insulin that is not under control.

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I am scheduled for surgery during the study period.

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I have kidney issues identified through tests or my medical history.

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My weight is either below 12 kg or above 100 kg.

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I was hospitalized due to COVID-19 in the last 12 months.

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I tested positive for drugs not prescribed to me.

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I have a long-term or severe depression.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Pharmacokinetic - Measurement of AUC

Pharmacokinetic - Measurement of Cmax

Pharmacokinetic - Measurement of t1/2

+2 more

Secondary study objectives

Exploratory efficacy measurement

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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 2

~80

0 / 17Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Prader-Willi Syndrome (PWS) include growth hormone therapy, which primarily works by increasing levels of insulin-like growth factor 1 (IGF-1). IGF-1 plays a crucial role in growth and development, and its modulation can help improve muscle mass, reduce body fat, and enhance physical strength in PWS patients. This is particularly important for PWS patients who often suffer from hypotonia (low muscle tone), obesity, and short stature. By targeting IGF-1 activity, treatments like NNZ-2591 aim to address these core symptoms, potentially improving overall quality of life and physical health for individuals with PWS.

The effects of stimulant medication on the growth of hyperkinetic children.Clinical effects of fenfluramine on children with autism: a review of the research.An open-label pilot trial of minocycline in children as a treatment for Angelman syndrome.