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

Primary Purpose

Prader-Willi Syndrome

Status
Withdrawn
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Placebo
Tesomet
Sponsored by
Saniona
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Prader-Willi Syndrome focused on measuring hyperphagia

Eligibility Criteria

13 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Sparrow Clinical Research Institute

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Placebo Comparator

Experimental

Experimental

Experimental

Arm Label

Placebo

Tesomet Low Dose

Tesomet Medium Dose

Tesomet High Dose

Arm Description

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

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

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

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Change in Body Weight
Percentage change in body weight
Hyperphagia Severity (Caregiver)
Change in caregiver rating of hyperphagia severity
Hyperphagia Change (Caregiver)
Proportion of caregiver responses for change in subject's hyperphagia
PWS Severity (Clinician)
Change in clinician rating of the subject's PWS severity
Overall Status Change (Clinician)
Proportion of clinician responses for change in subject's overall clinical status

Full Information

First Posted
January 5, 2022
Last Updated
December 9, 2022
Sponsor
Saniona
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1. Study Identification

Unique Protocol Identification Number
NCT05198362
Brief Title
Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome
Acronym
PWS
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

2. Study Status

Record Verification Date
December 2022
Overall Recruitment Status
Withdrawn
Why Stopped
Due to financial considerations Sponsor is unable to complete the trial and assess the planned objectives/endpoints. No subjects have been randomized to treatment in the clinical trial and the decision therefore has no safety concern for patients
Study Start Date
December 28, 2021 (Actual)
Primary Completion Date
December 9, 2022 (Actual)
Study Completion Date
December 9, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Saniona

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome
Keywords
hyperphagia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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
Arm Title
Tesomet Low Dose
Arm Type
Experimental
Arm Description
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
Arm Title
Tesomet Medium Dose
Arm Type
Experimental
Arm Description
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
Arm Title
Tesomet High Dose
Arm Type
Experimental
Arm Description
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
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Inactive comparator
Intervention Type
Drug
Intervention Name(s)
Tesomet
Other Intervention Name(s)
tesofensine, metoprolol
Intervention Description
Fixed-dose combination
Primary Outcome Measure Information:
Title
Hyperphagia
Description
Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score
Time Frame
Baseline to Week 16
Secondary Outcome Measure Information:
Title
Change in Body Weight
Description
Percentage change in body weight
Time Frame
Baseline to Week 16
Title
Hyperphagia Severity (Caregiver)
Description
Change in caregiver rating of hyperphagia severity
Time Frame
Baseline to Week 16
Title
Hyperphagia Change (Caregiver)
Description
Proportion of caregiver responses for change in subject's hyperphagia
Time Frame
Week 16
Title
PWS Severity (Clinician)
Description
Change in clinician rating of the subject's PWS severity
Time Frame
Baseline to Week 16
Title
Overall Status Change (Clinician)
Description
Proportion of clinician responses for change in subject's overall clinical status
Time Frame
Week 16

10. Eligibility

Sex
All
Minimum Age & Unit of Time
13 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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: 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 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: 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sophie Guillaume, MS
Organizational Affiliation
Saniona
Official's Role
Study Director
Facility Information:
Facility Name
Sparrow Clinical Research Institute
City
Lansing
State/Province
Michigan
ZIP/Postal Code
48912
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
The Sponsor will consider requests from qualified researchers for access to TM006 study materials
IPD Sharing Time Frame
Following completion of Tesomet clinical development

Learn more about this trial

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

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