Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This Phase 3, open-label extension, multicenter study will evaluate long-term safety, tolerability and efficacy of NNZ-2591 in pediatric participants with Phelan- McDermid Syndrome.
After providing informed consent/assent, pediatric participants with Phelan-McDermid syndrome who participated in previous studies (NEU-2591-PMS-301 and NEU-2591-PMS-001) will undergo assessments for eligibility, baseline characteristics and symptom severity. Once eligibility is confirmed, participants will receive orally administered NNZ-2591 during the 52-week Treatment Period. A 2-week safety follow-up period will occur immediately after the completion of the Treatment Period.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NNZ-2591 Arm | Experimental | The total duration of this study for each participant will be up to up to 56 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NNZ-2591 | Drug | The study drug will be administered twice daily orally. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Long-term safety and tolerability of NNZ-2591 as assessed by the incidence of adverse events across participants | Incidence of TEAEs, AESI and SAEs across participants | Baseline through Safety Follow-Up (Month 12) |
| Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. | Change from Baseline in ECG Heart Rate (bpm) | Baseline through Month 12 |
| Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. | Change from Baseline PR Interval (ms QRS interval (ms) | Baseline through Safety Follow-Up (Month 12) |
| Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. | Change from Baseline in QT interval (ms) | Baseline through Safety Follow-Up (Month 12) |
| Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. | Change from Baseline in QTcB interval (ms) | Baseline through Safety Follow-Up (Month 12) |
| Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. | Change from Baseline in QTcF interval (ms) | Baseline through Safety Follow-Up (Month 12) |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score | Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score. The PMSA-C scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse. | Months 3 and 12 |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Medical Information Lead | Contact | 231-203-8050 | medicalinformation@neurenpharma.com |
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neuren PMS-302 Site#111 | Recruiting | San Rafael | California | 94903 | United States |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| C536801 | Telomeric 22q13 Monosomy Syndrome |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| C540261 | cyclo-L-glycyl-L-2-allylproline |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. | Change from Baseline in RR interval (ms) | Baseline through Safety Follow-Up (Month 12) |
| Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. | Change from Baseline for heart rate (bpm) | Baseline through Month 12 |
| Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. | Change from Baseline for respiration rate (breaths per minute) | Baseline through Month 12 |
| Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. | Change from Baseline for Temperature (Celsius) | Baseline through Month 12 |
| Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. | Change from Baseline for Diastolic Blood Pressure (mm Hg) | Baseline through Month 12 |
| Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. | Change from Baseline for Systolic Blood Pressure (mm Hg) | Baseline through Month 12 |
| Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant clinical laboratory parameters events across participants. | Incidence of abnormal and clinically significant laboratory parameters | Baseline through Month 12 |
| Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant physical examination findings across participants. | Incidence of abnormal, clinically significant physical examination findings | Baseline through Month 12 |
| Efficacy of NNZ-2591 as measured by the change from baseline in the Vineland Adaptive Behavior Scales-3, Interview version (Vineland-3) receptive communication subdomain raw score. | Efficacy of NNZ-2591 as measured by the change from baseline in the Vineland Adaptive Behavior Scales-3, Interview version (Vineland-3) receptive communication subdomain raw score. A higher raw score for the receptive communication subdomain indicates better adaptive behavior. | Months 3 and 12 |
| Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores. | Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores. The PMSA-C domain scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse. | Months 3 and 12 |
| Efficacy of NNZ-2591 as measured by the Caregiver Impression of Change (CIC) domain scores. | Efficacy of NNZ-2591 as measured by the Caregiver Impression of Change (CIC) domain scores. The CIC scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse. | Months 3 and 12 |
| Efficacy of NNZ-2591 as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) domain scores. | Efficacy of NNZ-2591 as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) domain scores. The PMSA-S scores range from 1 to 7 with 1 indicating typical for age, not at all impaired and 7 among the most severely impaired. | Months 3 and 12 |
| Efficacy of NNZ-2591 as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) overall score. | Efficacy of NNZ-2591 as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) overall score. The PMSA-S scores range from 1 to 7 with 1 indicating typical for age, not at all impaired and 7 among the most severely impaired. | Months 3 and 12 |
| Efficacy of NNZ-2591 as measured by the change from baseline in PMS Clinician Domain Specific Rating Scale (PMS-DSRS) scores. | Efficacy of NNZ-2591 as measured by the change from baseline in PMS Clinician Domain Specific Rating Scale (PMS-DSRS) scores. The PMS-DSRS scores range from 0 to 4 with 0 indicating Symptom Not Present and 4 indicating Very Severe. | Months 3 and 12 |