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

NCT04697381

Last updated date
Study Location
Kanagawa Children's Medical Center
Yokohama, Kanagawa, 232-8555, Japan
Contact
1-800-718-1021

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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Prader-Willi Syndrome
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
0 +
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


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.

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[email protected]

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Prader-Willi SyndromeStudy of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
NCT04697381
  1. Yokohama, Kanagawa
  2. Izumi, Osaka
  3. Koshigaya, Saitama
  4. Hamamatsu, Shizuoka
  5. Setagaya-ku, Tokyo
ALL GENDERS
0+
years
MULTIPLE SITES
Prader-Willi SyndromeGlobal Growth Hormone Study in Adults With Prader-Willi Syndrome
NCT04484051
  1. Camperdown,
  2. Rotterdam, Zuid-Holland
ALL GENDERS
30 Years+
years
MULTIPLE SITES
Advanced Information
Descriptive Information
Brief Title  ICMJE Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
Official Title  ICMJE 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)
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Prader-Willi Syndrome
Intervention  ICMJE
  • Biological: somatropin - GH naïve pediatric cohort
    somatropin 0.245 mg/kg/week
  • Biological: somatropin - GH treated cohort
    somatropin 0.084 mg/kg/week
  • Biological: somatropin - adult cohort
    somatropin 0.084 mg/kg/week
Study Arms  ICMJE
  • Experimental: somatropin - GH naïve pediatric cohort
    All participants will receive somatropin.
    Intervention: Biological: somatropin - GH naïve pediatric cohort
  • Experimental: somatropin - GH treated pediatric cohort
    All participants will receive somatropin
    Intervention: Biological: somatropin - GH treated cohort
  • Experimental: somatropin - adult cohort
    All participants will receive somatropin
    Intervention: Biological: somatropin - adult cohort
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 4, 2021)
30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 1, 2024
Estimated Primary Completion Date December 22, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study:All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Pfizer CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries  ICMJE Japan
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04697381
Other Study ID Numbers  ICMJE A6281323
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD:Yes
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/d….
URL:https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d…
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director:Pfizer CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP