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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05025241
Other study ID # NEU-2591-PMS-001
Secondary ID
Status Active, not recruiting
Phase Phase 2
First received
Last updated
Start date August 8, 2022
Est. completion date December 31, 2023

Study information

Verified date August 2023
Source Neuren Pharmaceuticals Limited
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Phelan-McDermid Syndrome.


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 Phelan-McDermid 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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 20
Est. completion date December 31, 2023
Est. primary completion date December 31, 2023
Accepts healthy volunteers No
Gender All
Age group 3 Years to 12 Years
Eligibility Inclusion Criteria: 1. Clinical diagnosis of PMS with a documented disease-causing genetic abnormality of SHANK3. 2. Males or females aged 3-12 years. 3. Body weight of 12 kg 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, renal, 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

Study Design


Intervention

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

Locations

Country Name City State
United States Boston Children's Hospital Boston Massachusetts
United States Massachusetts General Hospital Boston Massachusetts
United States Rush University Medical Center Chicago Illinois
United States Texas Children's Hospital Houston Texas

Sponsors (1)

Lead Sponsor Collaborator
Neuren Pharmaceuticals Limited

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Safety and Tolerability To examine the incidence, severity and frequency of adverse events (AEs), including serious adverse events (SAEs) during treatment with NNZ-2591. 13 weeks
Primary Pharmacokinetic - Measurement of Cmax Maximum observed concentration (Cmax) of NNZ-2591 13 weeks
Primary Pharmacokinetic - Measurement of AUC Area under the concentration-time curve of NNZ-2591 13 weeks
Primary Pharmacokinetic - Measurement of time to Cmax Time to Cmax of NNZ-2591 13 weeks
Primary Pharmacokinetic - Measurement of t1/2 Apparent terminal elimination half-life of NNZ-2591 13 weeks
Secondary Exploratory efficacy measurement Assessed by Phelan-McDermid syndrome-specific Clinical Global Impression Scale-Overall Improvement (CGI-I) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Caregiver Impression of Improvement 13 weeks
Secondary Exploratory efficacy measurement Assessed by Phelan-McDermid syndrome-specific Clinical Global Impression Scales-Domain Improvement 13 weeks
Secondary Exploratory efficacy measurement Assessed by Phelan-McDermid syndrome-specific Clinical Global Impression Scale-Severity (CGI-S)-Overall and Domain 13 weeks
Secondary Exploratory efficacy measurement Assessed by Caregiver Top 3 Concerns Likert Scale 13 weeks
Secondary Exploratory efficacy measurement Assessed by MacArthur-Bates Communicative Development Inventory (MB-CDI) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Observer-Reported Communication Ability (ORCA) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Aberrant Behavior Checklist-2 (ABC-2) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Child Sleep Habits Questionnaire (CSHQ) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Gastrointestinal Health Questionnaire (GIHQ) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Vineland Adaptive Behavior Scales-3, Interview version 13 weeks
Secondary Exploratory efficacy measurement Caregiver Diaries 13 weeks
Secondary Exploratory efficacy measurement Assessed by Quality of Life Inventory-Disability (QI-Disability) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Impact of Childhood Neurological Disability (ICND)-Overall quality of life rating 13 weeks
See also
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Completed NCT03493607 - AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy Phase 2
Completed NCT03426059 - Mapping the Phenotype in Adults With Phelan-McDermid Syndrome
Recruiting NCT02461420 - Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome
Completed NCT02000167 - Mitochondrial Dysfunction in Phelan-McDermid Syndrome
Enrolling by invitation NCT04312152 - Q10 Ubiquinol in Autism Spectrum Disorder and in Phelan-McDermid Syndrome. N/A
Completed NCT05105685 - Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS Phase 1/Phase 2
Enrolling by invitation NCT05187377 - A Controlled Trial of Growth Hormone in Phelan-McDermid Syndrome and Idiopathic Autism Phase 2
Completed NCT01525901 - Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome) Phase 2
Completed NCT02710084 - Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome Phase 2