- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05025332
An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001) (PTHS-001)
August 3, 2023 updated by: Neuren Pharmaceuticals Limited
An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Pitt Hopkins Syndrome.
Study Overview
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 Pitt Hopkins 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.
Study Type
Interventional
Enrollment (Estimated)
20
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: James Shaw
- Phone Number: +61 427 299 669
- Email: jshaw@neurenpharma.com
Study Locations
-
-
Alabama
-
Birmingham, Alabama, United States, 35294
- Recruiting
- University of Alabama at Birmingham
-
Contact:
- Cassandra Newsom
- Phone Number: 205-934-2352
- Email: Cassandranewsom@uabmc.edu
-
-
California
-
San Francisco, California, United States, 94143
- Recruiting
- University of California at San Francisco
-
Contact:
- Alison Walmsley
- Phone Number: 408-334-5630
- Email: alison.walmsley@ucsf.edu
-
-
Colorado
-
Aurora, Colorado, United States, 80045
- Recruiting
- Children's Hospital Colorado
-
Contact:
- Eliana Obando
- Phone Number: 720-777-5378
- Email: eliana.obando@childrenscolorado.org
-
-
Illinois
-
Chicago, Illinois, United States, 60612
- Recruiting
- Rush University Medical Center
-
Contact:
- Alison Bauman
- Phone Number: 312-942-2005
- Email: Allison_L_Baumann@rush.edu
-
-
Texas
-
Dallas, Texas, United States, 75390
- Recruiting
- UT Southwestern
-
Contact:
- Jessica Clark
- Phone Number: 972-655-4847
- Email: Jessica.Clark@UTSouthwestern.edu
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
3 years to 17 years (Child)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Clinical diagnosis of PTHS with a documented disease-causing genetic etiology for the disorder.
- Males or females aged 3-17 years.
- Body weight of 12kg or higher at screening
- Subjects with a Clinical Global Impression- Severity (CGI-S) score of 4 or greater at the Screening visit.
- Not actively undergoing regression or loss of skills, defined as no persistent loss of previously acquired developmental skills for a period within 3 months of the Screening visit
- Each subject must be able to swallow the study medication provided as a liquid solution.
- Caregiver(s) must have sufficient English language skills.
Exclusion Criteria:
- Body weight <12kg at screening
- Clinically significant abnormalities in safety laboratory tests and vital signs at Screening.
- Abnormal QTcF interval or prolongation at Screening.
- Any other clinically significant finding on ECG at the Screening visit.
- Positive for severe acute respiratory syndrome coronavirus 2 (SARSCoV-2) and previous COVID 19 infection with last 12 months that required hospitalization.
- Unstable or changes Psychotropic treatment 2 weeks prior to screening
- Excluded concomitant treatments.
- Actively undergoing regression or loss of skills.
- Unstable seizure profile.
- Current clinically significant renal conditions and abnormalities
- Current clinically significant cardiovascular, hepatic, gastrointestinal, respiratory, endocrine disease, or clinically significant organ impairment.
- 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 visual or uncorrected hearing impairment.
- Allergy to strawberry.
- Positive pregnancy test
- Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily dose for 13 weeks.
|
NNZ-2591 oral solution (50mg/mL) to be administered twice daily dose for 13 weeks.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Safety and Tolerability
Time Frame: 13 weeks
|
To examine the incidence, severity and frequency of adverse events (AEs), including serious adverse events (SAEs) during treatment with NNZ-2591.
|
13 weeks
|
Pharmacokinetic - Measurement of Cmax
Time Frame: 13 weeks
|
Maximum observed concentration (Cmax) of NNZ-2591
|
13 weeks
|
Pharmacokinetic - Measurement of AUC
Time Frame: 13 weeks
|
Area under the concentration-time curve of NNZ-2591
|
13 weeks
|
Pharmacokinetic - Measurement of time to Cmax
Time Frame: 13 weeks
|
Time to Cmax of NNZ-2591
|
13 weeks
|
Pharmacokinetic - Measurement of t1/2
Time Frame: 13 weeks
|
Apparent terminal elimination half-life of NNZ-2591
|
13 weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Pitt Hopkins syndrome-specific Clinical Global Impression Scale-Overall Improvement (CGI-I)
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Caregiver Impression of Improvement
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Pitt Hopkins syndrome-specific Clinical Global Impression Scales-Domain Improvement
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Pitt Hopkins syndrome-specific Clinical Global Impression Scale-Severity (CGI-S)-Overall and Domain
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Caregiver Top 3 Concerns Likert Scale
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by MacArthur-Bates Communicative Development Inventory (MB-CDI)
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Observer-Reported Communication Ability (ORCA)
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Aberrant Behavior Checklist-2 (ABC-2)
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Child Sleep Habits Questionnaire (CSHQ)
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Gastrointestinal Health Questionnaire (GIHQ)
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Vineland Adaptive Behavior Scales-3, Interview version
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Modified two-minute walk test
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Bayley Scales of Infant Development 4 (BSID 4) motor scale
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Caregiver Diaries
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Quality of Life Inventory-Disability (QI-Disability)
|
13 weeks
|
Exploratory efficacy measurement
Time Frame: 13 weeks
|
Assessed by Impact of Childhood Neurological Disability (ICND)-Overall quality of life rating
|
13 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: James Shaw, Neuren Pharmaceuticals
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
October 14, 2022
Primary Completion (Estimated)
April 30, 2024
Study Completion (Estimated)
April 30, 2024
Study Registration Dates
First Submitted
August 24, 2021
First Submitted That Met QC Criteria
August 24, 2021
First Posted (Actual)
August 27, 2021
Study Record Updates
Last Update Posted (Actual)
August 7, 2023
Last Update Submitted That Met QC Criteria
August 3, 2023
Last Verified
August 1, 2023
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Mental Disorders
- Pathologic Processes
- Nervous System Diseases
- Respiratory Tract Diseases
- Respiration Disorders
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Disease Attributes
- Disease
- Signs and Symptoms, Respiratory
- Neurodevelopmental Disorders
- Syndrome
- Intellectual Disability
- Facies
- Hyperventilation
Other Study ID Numbers
- NEU-2591-PTHS-001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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