Clinical trial NCT05198362: Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome
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
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Prader-Willi Syndrome
Keywords: hyperphagia
Lead sponsor: Saniona
Sponsor class: INDUSTRY
First posted: 2022-01-20
Enrollment: 0
Sex: ALL
Minimum age (years): 13
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Hyperphagia
timeFrame: Baseline to Week 16
Secondary outcomes:
measure: Change in Body Weight
timeFrame: Baseline to Week 16
measure: Hyperphagia Severity (Caregiver)
timeFrame: Baseline to Week 16
measure: Hyperphagia Change (Caregiver)
timeFrame: Week 16
measure: PWS Severity (Clinician)
timeFrame: Baseline to Week 16
measure: Overall Status Change (Clinician)
timeFrame: Week 16
Other outcomes:
Locations:
city: Lansing
country: United States
facility: Sparrow Clinical Research Institute
state: Michigan
Study officials:
affiliation: Saniona
name: Sophie Guillaume, MS
role: STUDY_DIRECTOR
References: