Long Term Study of Genotropin (Somatropin) for Short Children Born Small for Gestational Age (SGA)

• ClinicalTrials.gov Identifier: NCT01859949
• Recruitment Status: Completed
• First Posted: May 22, 2013
• Results First Posted: March 15, 2016
• Last Update Posted: May 3, 2016
• Sponsor: Pfizer
• Information provided by (Responsible Party): Pfizer

Tracking Information

• First Submitted Date: April 17, 2013
• First Posted Date: May 22, 2013
• Results First Submitted Date: February 16, 2016
• Results First Posted Date: March 15, 2016
• Last Update Posted Date: May 3, 2016
• Study Start Date: October 2002
• Actual Primary Completion Date: August 2015 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: February 16, 2016): Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Month 12 (at the end of previous study) to 156 ]

Original Primary Outcome Measures (submitted: May 20, 2013)

• Percentage of Participants With Treatment-Related Adverse Events (AEs) or Serious Adverse Events (SAEs) [ Time Frame: 6 months ]
• Number of Participants With Laboratory Test Values of Potential Clinical Importance [ Time Frame: 6 months ]

Current Secondary Outcome Measures (submitted: February 16, 2016)

• Height Velocity Standard Deviation Score (SDS) for Chronological Age [ Time Frame: Month 12 (at the end of previous study) to 156 ]
  Height velocity is the yearly height gain. Height velocity SDS is calculated as following formula; Height velocity SDS = (height velocity - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values of the participants age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender.
• Height Velocity [ Time Frame: Month 12 (at the end of previous study) to 156 ]
  Height velocity is the yearly height gain
• Height SDS for Chronological Age [ Time Frame: Month 12 (at the end of previous study) to 156 ]
  Height SDS is calculated as following formula; Height SDS = (height - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values on the participant age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender.
• Height Velocity SDS for Bone Age [ Time Frame: Month 12 (at the end of previous study) to 156 ]
  To measure bone age, X-ray images of the left hand were centrally assessed by an independent specialist using the Tanner-Whitehouse 2 (RUS) method standardized for Japanese children. Height velocity is the yearly height gain. Height velocity SDS for bone age is calculated as following formula; Height velocity SDS = (height velocity - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values corresponding to bone age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender.
• Height SDS for Bone Age [ Time Frame: Month 12 (at the end of previous study) to 156 ]
  To measure bone age, X-ray images of the left hand were centrally assessed by an independent specialist using the Tanner-Whitehouse 2 (RUS) method standardized for Japanese children. Height SDS for bone age is calculated as following formula; Height SDS = (height - mean) / standard deviation, where mean and standard deviation were based on standard Japanese values corresponding to bone age and gender. The scores were centred around zero. Negative score indicated a participant was smaller for their age/gender.

Original Secondary Outcome Measures (submitted: May 20, 2013)

• Height velocity. [ Time Frame: 6 months ]
• Height velocity standard deviation scores (SDS) for chronological age. [ Time Frame: 6 months ]
• Height standard deviation scores (SDS) for chronological age. [ Time Frame: 6 months ]
• ∆ height standard deviation scores (SDS) for chronological age. [ Time Frame: 6 months ]
• Questionnaire for satisfaction of the treatment. [ Time Frame: 12 months ]
• Height velocity standard deviation scores (SDS) for bone age. [ Time Frame: 12 months ]
• Height standard deviation scores (SDS) for bone age. [ Time Frame: 12 months ]
• ∆ height standard deviation scores (SDS) for bone age. [ Time Frame: 12 months ]
• ∆ height SDS for bone age. [ Time Frame: 12 months ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Long Term Study of Genotropin (Somatropin) for Short Children Born Small for Gestational Age (SGA)
• Official Title: Long Term Study Of Pnu-180307 For Short Children Born Small For Gestational Age (Sga) Without Epiphyseal Closure (Extension Of The Study 307-met-0021-002)
• Brief Summary: To assess the long-term safety of Genotropin(somatropin) on Small for Gestational Age (SGA) without epiphyseal closing.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 4
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Short Stature Born Small for Gestational Age (SGA)

Intervention

Drug: Genotropin (somatropin)

Genotropin(somatoropin) 12 mg, Genotropin(somatropin) will be given at a dose of 0.067 mg/kg/day or 0.033 mg/kg/day to all participating patients

Study Arms

Experimental: Genotropin (somatropin)

Intervention: Drug: Genotropin (somatropin)

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 20, 2013): 62
• Original Actual Enrollment: Same as current
• Actual Study Completion Date: August 2015
• Actual Primary Completion Date: August 2015 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

Children with short stature due to SGA who received treatment in the study GENASG-0021-002.

Exclusion Criteria:

• Children who have any chronic disease requiring treatment with steroid hormone that may affect growth promotion including estrogen, androgen, anabolic hormone, and corticosteroids (except those for external use), and have received the treatment.
• Children who have received radiotherapy or chemotherapy.
• Children who have serious cardiac disease, renal disease, or hepatic disease.
• Children who have diabetes mellitus with a manifestation of abnormal glucose metabolism.
• Children who have serious chronic disease.
• Children who have malignant tumor.
• Children who are allergic to m-cresol.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 4 Years to 8 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Japan

Administrative Information

• NCT Number: NCT01859949
• Other Study ID Numbers: GENASG-0021-007; A6281225 ( Other Identifier: Alias Study Number ); 2015-004552-21 ( EudraCT Number )
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Pfizer
• Original Responsible Party: Same as current
• Current Study Sponsor: Pfizer
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Pfizer CT.gov Call Center; Pfizer

• PRS Account: Pfizer
• Verification Date: March 2016