Prader-Willi Syndrome
Conditions
Keywords
hyperphagia
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.
Interventions
OTHERPlacebo
Inactive comparator
DRUGTesomet
Fixed-dose combination
Sponsors
Saniona
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
13 Years to 65 Years
Healthy volunteers
No
Inclusion 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Hyperphagia | Baseline to Week 16 | Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Body Weight | Baseline to Week 16 | Percentage change in body weight |
| Hyperphagia Severity (Caregiver) | Baseline to Week 16 | Change in caregiver rating of hyperphagia severity |
| Hyperphagia Change (Caregiver) | Week 16 | Proportion of caregiver responses for change in subject's hyperphagia |
| PWS Severity (Clinician) | Baseline to Week 16 | Change in clinician rating of the subject's PWS severity |
| Overall Status Change (Clinician) | Week 16 | Proportion of clinician responses for change in subject's overall clinical status |
Countries
United States
Outcome results
None listed