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Clinical Trials/NCT04697381
NCT04697381CompletedPhase 3

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)

Pfizer5 sites in 1 country33 target enrollmentStarted: February 9, 2021Last updated:
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Conditions
Interventions

Trial Snapshot

Phase
Phase 3
Status
Completed
Sponsor
Pfizer
Enrollment
33
Locations
5
Primary Endpoint
Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

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.

Study Design

Study Type
Interventional
Allocation
Non Randomized
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
0 Years to — (Child, Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

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.

Arms & Interventions

somatropin - GH naïve pediatric cohort

Experimental

All participants will receive somatropin.

Intervention: somatropin - GH naïve pediatric cohort (Biological)

somatropin - GH treated pediatric cohort

Experimental

All participants will receive somatropin

Intervention: somatropin - GH treated cohort (Biological)

somatropin - adult cohort

Experimental

All participants will receive somatropin

Intervention: somatropin - adult cohort (Biological)

Outcomes

Primary Outcomes

Change From Baseline to Month 12 in Lean Body Mass Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort

Time Frame: Baseline, Month 12

Lean body mass, a measurement of body composition, was assessed by DEXA scan, and calculated as lean body mass (%) = lean body mass (kg) / (lean body mass \[kg\] + fat mass \[kg\])\*100.

Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort

Time Frame: Baseline, Month 12

Lean body mass, a measurement of body composition, was assessed by DEXA scan, and calculated as lean body mass (%) = lean body mass kilogram (kg) / (lean body mass \[kg\] + fat mass \[kg\]) \*100.

Secondary Outcomes

  • Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: GH Naive Pediatric and GH Treated Pediatric Cohort(Baseline, Month 12)
  • Change From Baseline to Month 12 in Lean Body Mass Measured by Bioelectrical Impedance Analysis (BIA)-Adult Cohort(Baseline, Month 12)
  • Change From Baseline to Month 6 in Lean Body Mass Measured by DEXA: Adult Cohort Only(Baseline, Month 6)
  • Change From Baseline to Month 12 in Lean Body Mass Measured by BIA-GH Naive Pediatric and GH Treated Pediatric Cohort(Baseline, Month 12)
  • Change From Baseline to Month 12 in Body Fat (Percentage) Measured by DEXA: Adult Cohort(Baseline, Month 12)
  • Change From Baseline to Month 12 in Adipose Tissue Distribution Measured by Abdominal Computed Tomography (CT)(Baseline, Month 12)
  • Bone Maturation(12 months)
  • Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events(From day 1 up to 28 days after end of study treatment (maximum duration up to 38 months))
  • Number of Participants With Laboratory Test Abnormalities(From Day 1 up to 28 days after end of study treatment (maximum duration up to 38 months))

Investigators

Sponsor
Pfizer
Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (5)

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