Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome

Phase 2

Withdrawn

• Conditions: Prader-Willi Syndrome
• Interventions: Other: Placebo; Drug: Tesomet

• Registration Number: NCT05198362
• Lead Sponsor: Saniona

Brief Summary

This study will evaluate the safety and efficacy of Tesomet (tesofensine + metoprolol) in subjects with PWS.

Detailed Description

For the double-blind portion of the study, dosing will be initiated in a subgroup of adults who are 18-65 years of age. Following independent Data Monitoring Board review of subgroup safety data, and review and confirmation to proceed by FDA, enrollment of subjects \<18 years of age will commence.

Study Timeline

• Study Start: 2021-12-28
• Study First Submitted: 2022-01-05
• Study First Submitted QC: 2022-01-05
• Study First Posted: 2022-01-20
• Status Verified: 2022-12
• Primary Completion: 2022-12-09
• Study Completion: 2022-12-09
• Last Update Submitted: 2022-12-09
• Last Update Posted: 2022-12-13

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• Subject and their legally authorized representative must be willing to provide informed consent

• Confirmed genetic diagnosis of PWS

• Body mass index (BMI) within the following range at Screening:

  1. Female and male subjects 18 to 65 years of age: 27 to 60 kg/m2; or
  2. Female and male subjects 13 to 17 years of age with BMI that is at least 85th percentile for age and sex;

• Female subjects must be of non-child-bearing potential

• Documented stable body weight

• Moderate hyperphagia at Screening and at Baseline

• Participants must have a reliable and stable caregiver who should be able to spend an adequate amount of time with the participants to be able to address behaviors, activities and symptoms

• Male subjects who are sexually active must be surgically sterile

Key

Exclusion Criteria

• Females who are pregnant, breastfeeding, or actively intending to become pregnant during the study

• Sitting BP that meets the following criteria after 5 minutes of rest at Screening:

  1. Adult subjects with systolic BP >/=145 mmHg or <100 mmHg; or
  2. Adult subjects with diastolic BP >/=95 mmHg or <70 mmHg; or
  3. Adolescent subjects with a systolic or diastolic BP that is 95th percentile or greater for age and sex

• Type 1 diabetes mellitus

• History of dementia (eg, Alzheimer's disease, Parkinson's disease)

• History of bulimia or anorexia nervosa

• History of major depressive disorder within 2 years prior to Screening, or any history of other severe psychiatric disorder (eg, schizophrenia, bipolar disorder), or symptoms of delusions, hallucinations, or mania/hypomania within 90 days prior to Screening, as described by the Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-5)

• Uncontrolled endocrine disorders (eg, Cushing syndrome, Addison's, hypothyroidism, hyperthyroidism)

• Medical condition or recent systemic infection that, in the opinion of the Investigator, could impact the safety of the subject

• Use of prohibited medications, including current use of SSRIs/SNRIs

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Once-daily PO for 16 weeks during the double-blind period; then if eligible for OLE, once-daily dosing for 36 weeks of the highest tolerated Tesomet dose from the double-blind period
Placebo	Tesomet	Once-daily PO for 16 weeks during the double-blind period; then if eligible for OLE, once-daily dosing for 36 weeks of the highest tolerated Tesomet dose from the double-blind period
Tesomet Medium Dose	Tesomet	Once-daily PO for 16 weeks during the double-blind period; then if eligible for OLE, once-daily dosing for 36 weeks of the highest tolerated dose from the double-blind period
Tesomet High Dose	Tesomet	Once-daily PO for 16 weeks during the double-blind period; then if eligible for OLE, once-daily dosing for 36 weeks of the highest tolerated dose from the double-blind period
Tesomet Low Dose	Tesomet	Once-daily PO for 16 weeks during the double-blind period; then if eligible for OLE, once-daily dosing for 36 weeks of the highest tolerated dose from the double-blind period

Primary Outcome Measures

Name	Time	Method
Hyperphagia	Baseline to Week 16	Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score

Secondary Outcome Measures

Name	Time	Method
Overall Status Change (Clinician)	Week 16	Proportion of clinician responses for change in subject's overall clinical status
Change in Body Weight	Baseline to Week 16	Percentage change in body weight
Hyperphagia Change (Caregiver)	Week 16	Proportion of caregiver responses for change in subject's hyperphagia
Hyperphagia Severity (Caregiver)	Baseline to Week 16	Change in caregiver rating of hyperphagia severity
PWS Severity (Clinician)	Baseline to Week 16	Change in clinician rating of the subject's PWS severity

Trial Locations

Locations (1)

Sparrow Clinical Research Institute; 🇺🇸 Lansing, Michigan, United States