NNZ-2591 for Pitt Hopkins Syndrome

(PTHS-001 Trial)

No longer recruiting at 4 trial locations

Overseen By Erin Norris

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Neuren Pharmaceuticals Limited

Must not be taking: Psychotropics

Disqualifiers: Diabetes, Cardiovascular, Seizures, Depression, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 13 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral treatment called NNZ-2591 for children and adolescents with Pitt Hopkins Syndrome, a rare genetic disorder that affects development and causes intellectual disability. The main goal is to determine the treatment's safety and effectiveness. Researchers seek children aged 3 to 17 diagnosed with Pitt Hopkins Syndrome who are not currently losing developmental skills. Participants must also be able to swallow a liquid solution. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications, but it mentions that unstable or changed psychotropic treatments within 2 weeks before screening are excluded. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that NNZ-2591 is likely to be safe for humans?

Research has shown that NNZ-2591 is safe and well tolerated in earlier studies with children and teenagers who have Pitt Hopkins Syndrome. These studies found no major safety issues. Doctors and caregivers observed improvements, suggesting the treatment is not only safe but also possibly beneficial. So far, the evidence supports NNZ-2591 as a safe option for this condition. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Pitt Hopkins Syndrome, which mainly focus on managing symptoms, NNZ-2591 offers a novel approach by potentially addressing the underlying causes of the condition. This experimental drug is an oral solution that is believed to work by enhancing brain cell communication and promoting neural health, targeting pathways that are not addressed by current therapies. Researchers are excited about NNZ-2591 because it holds the promise of not just relieving symptoms but potentially improving cognitive and developmental outcomes, which could significantly enhance the quality of life for individuals with Pitt Hopkins Syndrome.

What evidence suggests that NNZ-2591 might be an effective treatment for Pitt Hopkins Syndrome?

Research has shown that NNZ-2591, the treatment under study in this trial, may help treat Pitt Hopkins Syndrome. In earlier studies, children with this condition showed improvements. Specifically, nine out of 11 children demonstrated noticeable improvement on the Clinical Global Impression of Improvement (CGI-I) scale, indicating that both doctors and caregivers observed positive changes. Additionally, the treatment proved to be safe and well tolerated. These findings suggest NNZ-2591 could potentially help manage symptoms of Pitt Hopkins Syndrome. ^{1 2 3 4 6}

Who Is on the Research Team?

James Shaw

Principal Investigator

Neuren Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for children and adolescents aged 3-17 with Pitt Hopkins Syndrome, who can swallow liquid medication, weigh at least 12kg, have a certain severity of disease symptoms, and haven't lost skills in the past 3 months. They must not be pregnant or have significant health issues like heart or kidney problems.

Inclusion Criteria

I can swallow liquid medication.

I am between 3 and 17 years old.

You weigh at least 12 kilograms at the time of screening.

Exclusion Criteria

Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study

I am currently experiencing major or long-term depression.

I am scheduled for surgery during the study period.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive NNZ-2591 oral solution (50 mg/mL) twice daily for 13 weeks to assess safety, tolerability, and pharmacokinetics

13 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• NNZ-2591

Trial Overview The study tests NNZ-2591's safety and how well it works in kids with Pitt Hopkins Syndrome. It looks at how the body processes the drug (pharmacokinetics) and its effectiveness on various symptoms such as hyperventilation and intellectual disability.

How Is the Trial Designed?

Group I: NNZ-2591 Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Neuren Pharmaceuticals Limited

Lead Sponsor

Trials

13

Recruited

920+

Citations

1. neurenpharma.com neurenpharma.com/pdf/05375e89-43d1-41bc-bef8-fe89045539b6/Pitt-Hopkins-syndrome-Phase-2-trial-results-presentation.pdf

NNZ-2591 Pitt Hopkins syndrome Phase 2 trial top-line ... Positive data from Phelan McDermid syndrome and Pitt Hopkins syndrome Phase 2 trials support review and potential optimisation of existing ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05025332

An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins ... A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Pitt Hopkins Syndrome.

3. sciencedirect.com sciencedirect.com/science/article/pii/S2949774425001530

NNZ-2591, a synthetic IGF-1 metabolite analog: Phase 2 ... NNZ-2591 appeared to be safe and well tolerated in children and adolescents with PTHS. Improvements were observed by clinicians and caregivers in all PTHS- ...

4. clinicaltrialsarena.com clinicaltrialsarena.com/news/neuren-pitt-hopkins-trial/

Neuren Pharmaceuticals reports data from trial of NNZ-2591 Nine out of 11 children showed improvement on the PTHS Clinical Global Impression of Improvement (CGI-I), while eight demonstrated improvement ...

5. ctv.veeva.com ctv.veeva.com/study/an-open-label-study-of-oral-nnz-2591-in-pitt-hopkins-syndrome-pths001

An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins ... The primary purpose of this study is to investigate the safety, tolerability and pharmacokinetics of treatment with NNZ-2591 oral solution in ...

6. cdn.clinicaltrials.gov cdn.clinicaltrials.gov/large-docs/41/NCT05025241/Prot_000.pdf

CLINICAL TRIAL PROTOCOL This is a Phase 2, open-label study of the safety and tolerability of NNZ-2591 Oral Solution, 50 mg/mL in male and female children and ...