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

• ClinicalTrials.gov Identifier: NCT05198362
• Recruitment Status: Withdrawn
• First Posted: January 20, 2022
• Last Update Posted: December 13, 2022
• Sponsor: Saniona
• Information provided by (Responsible Party): Saniona

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Prader-Willi Syndrome	Other: Placebo; Drug: Tesomet	Phase 2

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 0 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Phase 2b, Double-blind, Randomized, Placebo-controlled, Multi-center, 16-week Dose Finding, Safety and Efficacy Study With Open-label Extension (OLE) Period of Tesomet in Subjects With Prader-Willi Syndrome
• Actual Study Start Date: December 28, 2021
• Actual Primary Completion Date: December 9, 2022
• Actual Study Completion Date: December 9, 2022

Arms and Interventions

Arm	Intervention/treatment
Placebo Comparator: 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	Other: Placebo; Inactive comparator; Drug: Tesomet; Fixed-dose combination; Other Names: tesofensine; metoprolol
Experimental: 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	Drug: Tesomet; Fixed-dose combination; Other Names: tesofensine; metoprolol
Experimental: 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	Drug: Tesomet; Fixed-dose combination; Other Names: tesofensine; metoprolol
Experimental: 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	Drug: Tesomet; Fixed-dose combination; Other Names: tesofensine; metoprolol

Outcome Measures

Primary Outcome Measures :

1. Hyperphagia [ Time Frame: Baseline to Week 16 ]
   Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score

Secondary Outcome Measures :

1. Change in Body Weight [ Time Frame: Baseline to Week 16 ]
   Percentage change in body weight
2. Hyperphagia Severity (Caregiver) [ Time Frame: Baseline to Week 16 ]
   Change in caregiver rating of hyperphagia severity
3. Hyperphagia Change (Caregiver) [ Time Frame: Week 16 ]
   Proportion of caregiver responses for change in subject's hyperphagia
4. PWS Severity (Clinician) [ Time Frame: Baseline to Week 16 ]
   Change in clinician rating of the subject's PWS severity
5. Overall Status Change (Clinician) [ Time Frame: Week 16 ]
   Proportion of clinician responses for change in subject's overall clinical status

Eligibility Criteria

• Ages Eligible for Study: 13 Years to 65 Years (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Key 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

Contacts and Locations

Locations

United States, Michigan

Sparrow Clinical Research Institute

Lansing, Michigan, United States, 48912

Sponsors and Collaborators

Saniona

Investigators

• Study Director: Sophie Guillaume, MS; Saniona

More Information

• Responsible Party: Saniona
• ClinicalTrials.gov Identifier: NCT05198362
• Other Study ID Numbers: TM006
• First Posted: January 20, 2022
• Last Update Posted: December 13, 2022
• Last Verified: December 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: The Sponsor will consider requests from qualified researchers for access to TM006 study materials
• Supporting Materials: Study Protocol; Statistical Analysis Plan (SAP); Informed Consent Form (ICF); Clinical Study Report (CSR)
• Time Frame: Following completion of Tesomet clinical development

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Saniona:

hyperphagia

Additional relevant MeSH terms:

Prader-Willi Syndrome; Syndrome; Disease; Pathologic Processes; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Obesity; Overweight; Overnutrition; Nutrition Disorders; Metoprolol; Anti-Arrhythmia Agents; Antihypertensive Agents; Sympatholytics; Autonomic Agents; Peripheral Nervous System Agents; Physiological Effects of Drugs; Adrenergic beta-1 Receptor Antagonists; Adrenergic beta-Antagonists; Adrenergic Antagonists; Adrenergic Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action