Clinical trial NCT04697381: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Prader-Willi Syndrome
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2021-01-06
Enrollment: 33
Sex: ALL
Minimum age (years): 0
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort
timeFrame: Baseline, Month 12
measure: Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort
timeFrame: Baseline, Month 12
Secondary outcomes:
measure: Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort
timeFrame: Baseline, Month 12
measure: Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort
timeFrame: Baseline, Month 12
measure: Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort
timeFrame: Baseline, Month 12
measure: Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort
timeFrame: Baseline, Month 12
measure: Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT)
timeFrame: Baseline, Month 12
measure: Change From Baseline to Month 6 in Lean Body Mass Measured by DEXA: Adult Cohort Only
timeFrame: Baseline, Month 6
measure: Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
timeFrame: From dose 1 up to 28 days after end of study treatment (maximum treatment period of 12 months; maximum duration for AEs collection of 13 months)
measure: Number of Participants With Laboratory Test Abnormalities
timeFrame: 12 months
measure: Bone Maturation
timeFrame: 12 months
Other outcomes:
Locations:
city: Yokohama
country: Japan
facility: Kanagawa Children's Medical Center
state: Kanagawa
city: Izumi
country: Japan
facility: Osaka Women's and Children's Hospital
state: Osaka
city: Koshigaya
country: Japan
facility: Dokkyo Medical University Saitama Medical Center
state: Saitama
city: Hamamatsu
country: Japan
facility: Hamamatsu University Hospital
state: Shizuoka
city: Setagaya-ku
country: Japan
facility: National Center for Child Health and Development
state: Tokyo
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: