NNZ-2591 for Pitt Hopkins Syndrome

(PTHS-001 Trial)

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

28 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a liquid medicine called NNZ-2591 in children and teenagers with Pitt Hopkins Syndrome. The goal is to see if the medicine is safe, how the body handles it, and if it helps with symptoms. Participants will take the medicine for several weeks.

Research Team

James Shaw

Principal Investigator

Neuren Pharmaceuticals

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+