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

NCT05198362 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: TM006
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Prader-Willi Syndrome

Keywords

hyperphagia

Outcome Measures

Primary Outcomes (1)

• Hyperphagia

  Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score

  Baseline to Week 16

Secondary Outcomes (5)

• Change in Body Weight

  Baseline to Week 16

• Hyperphagia Severity (Caregiver)

  Baseline to Week 16

• Hyperphagia Change (Caregiver)

  Week 16

• PWS Severity (Clinician)

  Baseline to Week 16

• Overall Status Change (Clinician)

  Week 16

Study Arms (4)

Placebo

PLACEBO COMPARATOR

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

Other: Placebo; Drug: Tesomet

Tesomet Low Dose

EXPERIMENTAL

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

Drug: Tesomet

Tesomet Medium Dose

EXPERIMENTAL

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

Drug: Tesomet

Tesomet High Dose

EXPERIMENTAL

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

Drug: Tesomet

Interventions

Placebo OTHER

Inactive comparator

Placebo

Tesomet DRUG

Fixed-dose combination

Also known as: tesofensine, metoprolol

Placebo; Tesomet High Dose; Tesomet Low Dose; Tesomet Medium Dose

Eligibility Criteria

• Age: 13 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Female and male subjects 18 to 65 years of age: 27 to 60 kg/m2; or
• 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

You may not qualify if:

• 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:
• Adult subjects with systolic BP \>/=145 mmHg or \<100 mmHg; or
• Adult subjects with diastolic BP \>/=95 mmHg or \<70 mmHg; or
• 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

Sponsors & Collaborators

• Saniona: lead

Study Sites (1)

Sparrow Clinical Research Institute

Lansing, Michigan, 48912, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome; Hyperphagia

Interventions

Tesofensine; Metoprolol

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Phenoxypropanolamines; Propanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Propanols; Amines

Study Officials

• Sophie Guillaume, MS

  Saniona

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 5, 2022
• First Posted: January 20, 2022
• Study Start: December 28, 2021
• Primary Completion: December 9, 2022
• Study Completion: December 9, 2022
• Last Updated: December 13, 2022

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Following completion of Tesomet clinical development

Locations

US (1)