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

NCT ID: NCT05198362

Last Updated: 2022-12-13

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-12-28
• Study Completion Date: 2022-12-09

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.

Conditions

Prader-Willi Syndrome

Keywords

hyperphagia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Inactive comparator

Tesomet

Intervention Type: DRUG

Fixed-dose combination

Tesomet Low Dose

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

Group Type: EXPERIMENTAL

Tesomet

Intervention Type: DRUG

Fixed-dose combination

Tesomet Medium Dose

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

Group Type: EXPERIMENTAL

Tesomet

Intervention Type: DRUG

Fixed-dose combination

Tesomet High Dose

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

Group Type: EXPERIMENTAL

Tesomet

Intervention Type: DRUG

Fixed-dose combination

Interventions

Placebo

Inactive comparator

Intervention Type: OTHER

Tesomet

Fixed-dose combination

Intervention Type: DRUG

Other Intervention Names

tesofensine; metoprolol

Eligibility Criteria

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

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

• Minimum Eligible Age: 13 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Saniona

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Sophie Guillaume, MS

Role: STUDY_DIRECTOR

Saniona

Locations

Sparrow Clinical Research Institute

Lansing, Michigan, United States

Countries

United States

Other Identifiers

TM006

Identifier Type: -

Identifier Source: org_study_id