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Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS

Primary Purpose

Prader-Willi Syndrome

Status
Active
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
somatropin - GH naïve pediatric cohort
somatropin - GH treated cohort
somatropin - adult cohort
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Prader-Willi Syndrome

Eligibility Criteria

0 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Male or female participants with documentation of genetically confirmed diagnosis of PWS.
  2. No plan to initiate a new treatment that may affect the body composition, such as gonadal hormone replacement therapy.
  3. Currently on appropriate diet and exercise programs and willing to continue throughout the study period at the discretion of the investigator.
  4. Participants, and if required by local/site regulations their parent(s)/legal guardian(s) must be willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures.
  5. Evidence of a personally signed and dated ICD (and written assent where applicable based on age and country regulation) indicating that the participant or a legally acceptable representative/parent(s)/legal guardian has been informed of all pertinent aspects of the study. Refer to Appendix 1 for the detailed process of obtaining consent.

    For inclusion of GH naïve pediatric cohort, participants must meet criteria 6 to 8:

  6. 18 years or younger.
  7. Naïve to GH treatment.
  8. Tanner stage 1 (for testes in males, for breasts in females).

    For inclusion of GH treated pediatric cohort, participants must meet criteria 9 and 10:

  9. Continued GH treatment for at least 2 years with stable dose for the last 6 months and being on GH at time of inclusion. The recent dose should be higher than 0.084 mg/kg/week.
  10. Participants who are about to complete GH treatment for his/her short stature (eg, due to meeting the treatment stopping criteria defined as a height SDS more than -2.5 for Japanese adult standards).

    For inclusion of adult cohort, participants must meet criteria 11 to 13:

  11. 18 years of chronological age or older at Day 1 visit.
  12. Off from GH treatment for at least 1 year.
  13. Serum IGF-I level within +2 SDS, adjusted for age and sex.

Exclusion Criteria:

  1. Participants with uncontrolled diabetes at the discretion of the investigator.
  2. Participants with malignant tumors.
  3. Participants with severe obesity or serious respiratory impairment.
  4. Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study.
  5. Previous administration with an investigational drug within 30 days (or as determined by the local requirement) or 5 half- lives preceding the first dose of study intervention used in this study (whichever is longer).
  6. Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family members.

Sites / Locations

  • Kanagawa Children's Medical Center
  • Osaka Women's and Children's Hospital
  • Dokkyo Medical University Saitama Medical Center
  • Hamamatsu University Hospital
  • National Center for Child Health and Development

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

somatropin - GH naïve pediatric cohort

somatropin - GH treated pediatric cohort

somatropin - adult cohort

Arm Description

All participants will receive somatropin.

All participants will receive somatropin

All participants will receive somatropin

Outcomes

Primary Outcome Measures

Change from baseline to Month 12 in lean body mass (%) measured by DEXA

Secondary Outcome Measures

Change from baseline to Month 12 in lean body mass (%) measured by BIA
Change from baseline to Month 12 in body fat (%) measured by DEXA
Change from baseline to Month 12 in adipose tissue distribution measured by abdominal CT
Change from baseline to Month 6 in lean body mass (%) measured by DEXA (adult cohort only)
Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs)
Number of Participants With Treatment Emergent Treatment-Related Serious Adverse Events (SAEs)
Number of Participants With Clinically Significant Change From Baseline in Laboratory Abnormalities
Bone maturation

Full Information

First Posted
January 4, 2021
Last Updated
July 5, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT04697381
Brief Title
Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
Official Title
A PHASE 3 MULTICENTER, OPEN LABEL, MULTI COHORT STUDY TO EVALUATE THE EFFICACY AND SAFETY OF SOMATROPIN IN JAPANESE PARTICIPANTS WITH PRADER-WILLI SYNDROME (PWS)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
February 6, 2021 (Actual)
Primary Completion Date
December 6, 2022 (Actual)
Study Completion Date
May 16, 2024 (Anticipated)

3. Sponsor/Collaborators

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

4. Oversight

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

5. Study Description

Brief Summary
This is a multicenter, open label, multi cohort study to evaluate the efficacy and safety of somatropin in a cohort of Japanese participants with PWS.

6. Conditions and Keywords

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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
33 (Actual)

8. Arms, Groups, and Interventions

Arm Title
somatropin - GH naïve pediatric cohort
Arm Type
Experimental
Arm Description
All participants will receive somatropin.
Arm Title
somatropin - GH treated pediatric cohort
Arm Type
Experimental
Arm Description
All participants will receive somatropin
Arm Title
somatropin - adult cohort
Arm Type
Experimental
Arm Description
All participants will receive somatropin
Intervention Type
Biological
Intervention Name(s)
somatropin - GH naïve pediatric cohort
Intervention Description
somatropin 0.245 mg/kg/week
Intervention Type
Biological
Intervention Name(s)
somatropin - GH treated cohort
Intervention Description
somatropin 0.084 mg/kg/week
Intervention Type
Biological
Intervention Name(s)
somatropin - adult cohort
Intervention Description
somatropin 0.084 mg/kg/week
Primary Outcome Measure Information:
Title
Change from baseline to Month 12 in lean body mass (%) measured by DEXA
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
Change from baseline to Month 12 in lean body mass (%) measured by BIA
Time Frame
52 weeks
Title
Change from baseline to Month 12 in body fat (%) measured by DEXA
Time Frame
52 weeks
Title
Change from baseline to Month 12 in adipose tissue distribution measured by abdominal CT
Time Frame
52 weeks
Title
Change from baseline to Month 6 in lean body mass (%) measured by DEXA (adult cohort only)
Time Frame
26 weeks
Title
Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs)
Time Frame
Baseline up to 50 months
Title
Number of Participants With Treatment Emergent Treatment-Related Serious Adverse Events (SAEs)
Time Frame
Baseline up to 50 months
Title
Number of Participants With Clinically Significant Change From Baseline in Laboratory Abnormalities
Time Frame
Baseline up to 48 months
Title
Bone maturation
Time Frame
52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
0 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female participants with documentation of genetically confirmed diagnosis of PWS. No plan to initiate a new treatment that may affect the body composition, such as gonadal hormone replacement therapy. Currently on appropriate diet and exercise programs and willing to continue throughout the study period at the discretion of the investigator. Participants, and if required by local/site regulations their parent(s)/legal guardian(s) must be willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures. Evidence of a personally signed and dated ICD (and written assent where applicable based on age and country regulation) indicating that the participant or a legally acceptable representative/parent(s)/legal guardian has been informed of all pertinent aspects of the study. Refer to Appendix 1 for the detailed process of obtaining consent. For inclusion of GH naïve pediatric cohort, participants must meet criteria 6 to 8: 18 years or younger. Naïve to GH treatment. Tanner stage 1 (for testes in males, for breasts in females). For inclusion of GH treated pediatric cohort, participants must meet criteria 9 and 10: Continued GH treatment for at least 2 years with stable dose for the last 6 months and being on GH at time of inclusion. The recent dose should be higher than 0.084 mg/kg/week. Participants who are about to complete GH treatment for his/her short stature (eg, due to meeting the treatment stopping criteria defined as a height SDS more than -2.5 for Japanese adult standards). For inclusion of adult cohort, participants must meet criteria 11 to 13: 18 years of chronological age or older at Day 1 visit. Off from GH treatment for at least 1 year. Serum IGF-I level within +2 SDS, adjusted for age and sex. Exclusion Criteria: Participants with uncontrolled diabetes at the discretion of the investigator. Participants with malignant tumors. Participants with severe obesity or serious respiratory impairment. Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study. Previous administration with an investigational drug within 30 days (or as determined by the local requirement) or 5 half- lives preceding the first dose of study intervention used in this study (whichever is longer). Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family members.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
Kanagawa Children's Medical Center
City
Yokohama
State/Province
Kanagawa
ZIP/Postal Code
232-8555
Country
Japan
Facility Name
Osaka Women's and Children's Hospital
City
Izumi
State/Province
Osaka
ZIP/Postal Code
594-1101
Country
Japan
Facility Name
Dokkyo Medical University Saitama Medical Center
City
Koshigaya
State/Province
Saitama
ZIP/Postal Code
343-8555
Country
Japan
Facility Name
Hamamatsu University Hospital
City
Hamamatsu
State/Province
Shizuoka
ZIP/Postal Code
431-3192
Country
Japan
Facility Name
National Center for Child Health and Development
City
Setagaya-ku
State/Province
Tokyo
ZIP/Postal Code
157-8535
Country
Japan

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
IPD Sharing URL
https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=A6281323
Description
To obtain contact information for a study center near you, click here.

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Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS

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