Prader-Willi Syndrome
Conditions
Interventions
Trade Name: Genotropin
Product Name: Genotropin GoQuick
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Genotropin
Current Sponsor code: Somatropin
Other descri
Sponsors
Pfizer, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion 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. Are the trial subjects under 18? yes Number of subjects for this age range: 33 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 11 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: To evaluate the effects of somatropin on other body composition parameters. To evaluate the safety and tolerability of somatropin.;Primary end point(s): Change from baseline to Month 12 in lean body mass (%) measured by DEXA.;Timepoint(s) of evaluation of this end point: Month 12;Main Objective: To evaluate the efficacy of somatropin in participants with PWS. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 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) Baseline up to 50 months Number of Participants With Treatment Emergent Treatment-Related Serious Adverse Events (SAEs) Baseline up to 50 months Number of Participants With Clinically Significant Change From Baseline in Laboratory Abnormalities Baseline up to 48 months;Timepoint(s) of evaluation of this end point: Month 12 Month 6 50 months 48 months | — |
Countries
Japan
Contacts
Public ContactClinical Trials.gov Call Center
Pfizer, Inc.
Outcome results
None listed