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An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001) (PTHS-001)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05025332
Recruitment Status : Recruiting
First Posted : August 27, 2021
Last Update Posted : March 15, 2023
Sponsor:
Information provided by (Responsible Party):
Neuren Pharmaceuticals Limited

Study Description
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Brief Summary:
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Pitt Hopkins Syndrome.

Condition or disease Intervention/treatment Phase
Pitt Hopkins Syndrome Drug: NNZ-2591 Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)
Actual Study Start Date : October 14, 2022
Estimated Primary Completion Date : May 18, 2023
Estimated Study Completion Date : May 18, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily dose for 13 weeks.
 Drug: NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily dose for 13 weeks.
Other Name: Cyclo-L-Glycyl-L-2-Allylproline


Outcome Measures
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Primary Outcome Measures :
  1. 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.

  2. Pharmacokinetic - Measurement of Cmax [ Time Frame: 13 weeks ]
    Maximum observed concentration (Cmax) of NNZ-2591

  3. Pharmacokinetic - Measurement of AUC [ Time Frame: 13 weeks ]
    Area under the concentration-time curve of NNZ-2591

  4. Pharmacokinetic - Measurement of time to Cmax [ Time Frame: 13 weeks ]
    Time to Cmax of NNZ-2591

  5. Pharmacokinetic - Measurement of t1/2 [ Time Frame: 13 weeks ]
    Apparent terminal elimination half-life of NNZ-2591


Secondary Outcome Measures :
  1. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Pitt Hopkins syndrome-specific Clinical Global Impression Scale-Overall Improvement (CGI-I)

  2. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Caregiver Impression of Improvement

  3. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Pitt Hopkins syndrome-specific Clinical Global Impression Scales-Domain Improvement

  4. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Pitt Hopkins syndrome-specific Clinical Global Impression Scale-Severity (CGI-S)-Overall and Domain

  5. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Caregiver Top 3 Concerns Likert Scale

  6. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by MacArthur-Bates Communicative Development Inventory (MB-CDI)

  7. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Observer-Reported Communication Ability (ORCA)

  8. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Aberrant Behavior Checklist-2 (ABC-2)

  9. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Child Sleep Habits Questionnaire (CSHQ)

  10. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Gastrointestinal Health Questionnaire (GIHQ)

  11. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Vineland Adaptive Behavior Scales-3, Interview version

  12. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Modified two-minute walk test

  13. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Bayley Scales of Infant Development 4 (BSID 4) motor scale

  14. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Caregiver Diaries

  15. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Quality of Life Inventory-Disability (QI-Disability)

  16. Exploratory efficacy measurement [ Time Frame: 13 weeks ]
    Assessed by Impact of Childhood Neurological Disability (ICND)-Overall quality of life rating


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Clinical diagnosis of PTHS with a documented disease-causing genetic etiology for the disorder.
  2. Males or females aged 3-17 years.
  3. Body weight of 12kg or higher at screening
  4. Subjects with a Clinical Global Impression- Severity (CGI-S) score of 4 or greater at the Screening visit.
  5. 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
  6. Each subject must be able to swallow the study medication provided as a liquid solution.
  7. Caregiver(s) must have sufficient English language skills.

Exclusion Criteria:

  1. Body weight <12kg at screening
  2. Clinically significant abnormalities in safety laboratory tests and vital signs at Screening.
  3. Abnormal QTcF interval or prolongation at Screening.
  4. Any other clinically significant finding on ECG at the Screening visit.
  5. Positive for severe acute respiratory syndrome coronavirus 2 (SARSCoV-2) and previous COVID 19 infection with last 12 months that required hospitalization.
  6. Unstable or changes Psychotropic treatment 2 weeks prior to screening
  7. Excluded concomitant treatments.
  8. Actively undergoing regression or loss of skills.
  9. Unstable seizure profile.
  10. Current clinically significant renal conditions and abnormalities
  11. Current clinically significant cardiovascular, hepatic, gastrointestinal, respiratory, endocrine disease, or clinically significant organ impairment.
  12. 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.
  13. Has planned surgery during the study.
  14. History of, or current, cerebrovascular disease or brain trauma.
  15. History of, or current catatonia or catatonia-like symptoms.
  16. History of, or current, malignancy.
  17. Current major or persistent depressive disorder (including bipolar depression).
  18. Significant, uncorrected visual or uncorrected hearing impairment.
  19. Allergy to strawberry.
  20. Positive pregnancy test
  21. Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05025332


Contacts
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Contact: James Shaw +61 427 299 669 jshaw@neurenpharma.com

Locations
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United States, Alabama
University of Alabama at Birmingham Recruiting
Birmingham, Alabama, United States, 35294
Contact: Cassandra Newsom    205-934-2352    Cassandranewsom@uabmc.edu   
United States, California
University of California at San Francisco Recruiting
San Francisco, California, United States, 94143
Contact: Alison Walmsley    408-334-5630    alison.walmsley@ucsf.edu   
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Eliana Obando    720-777-5378    eliana.obando@childrenscolorado.org   
United States, Illinois
Rush University Medical Center Recruiting
Chicago, Illinois, United States, 60612
Contact: Alison Bauman    312-942-2005    Allison_L_Baumann@rush.edu   
United States, Texas
UT Southwestern Recruiting
Dallas, Texas, United States, 75390
Contact: Jessica Clark    972-655-4847    Jessica.Clark@UTSouthwestern.edu   
Sponsors and Collaborators
Neuren Pharmaceuticals Limited
Investigators
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Study Director: James Shaw Neuren Pharmaceuticals
More Information
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Responsible Party: Neuren Pharmaceuticals Limited
ClinicalTrials.gov Identifier: NCT05025332    
Other Study ID Numbers: NEU-2591-PTHS-001
First Posted: August 27, 2021    Key Record Dates
Last Update Posted: March 15, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Neuren Pharmaceuticals Limited:
Pitt Hopkins Syndrome
Additional relevant MeSH terms:
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Hyperventilation
Intellectual Disability
Syndrome
Facies
Disease
Pathologic Processes
Respiration Disorders
Respiratory Tract Diseases
 Signs and Symptoms, Respiratory
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Neurodevelopmental Disorders
Mental Disorders
Disease Attributes