Special Investigation Of Genotropin Long Follow-Up For Patients With A Small For Gestational Age "Scoped for Japanese"

AN INVESTIGATION INTENDED TO EVALUATE THE LONG-TERM EFFICACY AND SAFETY OF GENOTROPIN IN CHILDREN WITH SGA DWARFISM WITHOUT EPIPHYSEAL CLOSURE. (RETROSPECTIVE STUDY)

The objective of this surveillance is to collect Safety and Effectiveness information for Long term Use of Somatropin for Patients with a Small for Gestational Age (SGA).

Study Overview

• Status: Completed
• Conditions: Small for Gestational Age (SGA)
• Intervention / Treatment: Drug: Somatropin

Detailed Description

All the patients whom an investigator prescribes Genotropin should be registered consecutively until the number of subjects reaches target number in order to extract patients enrolled into the investigation at random.

• Study Type: Observational
• Enrollment (Actual): 482

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 20 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

• A patients who is diagnosed for SGA. (SGA is defined as birth weight and/or length at least 2 standard deviations (SDs) below the mean for gestational age (<or=-2 SD)).
• A patient who is SGA and older than 3 years.
• A patient who has persistent short stature (i.e., remaining at least 2.5 SD below the mean for chronologic age).

Description

Inclusion Criteria:

• A patient who was administered Somatropin (Genotropin).

Exclusion Criteria:

• N/A.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Somatropin; Patients administered Somatropin.	Drug: Somatropin; 0.23 mg to 0.48 mg/kg/week; Other Names: Genotropin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Adverse Drug Reaction (ADR)	An adverse drug reaction (ADR) was any untoward medical occurrence attributed to Genotropin in a participant who received Genotropin. A serious ADR (SADR) was an ADR resulting in any of the following out comes or deemed significant for any other reason: death; life-threatening experience (immediate risk of dying); initial or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Relatedness to Genotropin was assessed by the physician.	Approximately 11.58 years
Change in Height Velocity Standard Deviation (SD) Score for Chronological Age From Baseline At Year 1	Change in height Velocity SD score from baseline was calculated. Height Velocity SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 1
Change in Height Velocity Standard Deviation (SD) Score for Chronological Age From Baseline at Year 2	Change in height Velocity SD score from baseline was calculated. Height Velocity SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 2
Change in Height Velocity Standard Deviation (SD) Score for Chronological Age From Baseline at Year 3	Change in height Velocity SD score from baseline was calculated. Height Velocity SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 3
Change in Height Velocity Standard Deviation (SD) Score for Chronological Age From Baseline at Year 4	Change in height Velocity SD score from baseline was calculated. Height Velocity SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 4
Change in Height Velocity Standard Deviation (SD) Score for Chronological Age From Baseline at Year 5	Change in height Velocity SD score from baseline was calculated. Height Velocity SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 5
Change in Height Velocity Standard Deviation (SD) Score for Chronological Age From Baseline at Year 6	Change in height Velocity SD score from baseline was calculated. Height Velocity SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 6
Change in Height Velocity Standard Deviation (SD) Score for Chronological Age From Baseline at Year 7	Change in height Velocity SD score from baseline was calculated. Height Velocity SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 7
Change in Height Velocity Standard Deviation (SD) Score for Chronological Age From Baseline at Year 10	Change in height Velocity SD score from baseline was calculated. Height Velocity SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 10
Change in Height Standard Deviation (SD) Score for Chronological Age From Baseline at Year 1	Change in height SD score from baseline was calculated. Height SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 1
Change in Height Standard Deviation (SD) Score for Chronological Age From Baseline at Year 2	Change in height SD score from baseline was calculated. Height SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 2
Change in Height Standard Deviation (SD) Score for Chronological Age From Baseline at Year 3	Change in height SD score from baseline was calculated. Height SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 3
Change in Height Standard Deviation (SD) Score for Chronological Age From Baseline at Year 4	Change in height SD score from baseline was calculated. Height SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 4
Change in Height Standard Deviation (SD) Score for Chronological Age From Baseline at Year 5	Change in height SD score from baseline was calculated. Height SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 5
Change in Height Standard Deviation (SD) Score for Chronological Age From Baseline at Year 6	Change in height SD score from baseline was calculated. Height SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 6
Change in Height Standard Deviation (SD) Score for Chronological Age From Baseline at Year 7	Change in height SD score from baseline was calculated. Height SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 7
Change in Height Standard Deviation (SD) Score for Chronological Age From Baseline at Year 9	Change in height SD score from baseline was calculated. Height SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 9
Change in Height Standard Deviation (SD) Score for Chronological Age From Baseline at Year 10	Change in height SD score from baseline was calculated. Height SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population.	Baseline, Year 10

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Height Standard Deviation (SD) Score at Puberty and at Near Final Height in Participants Who Reached Near Final Height	Height SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population. Near final height was defined as the height at one of the following conditions: the annual height velocity became less than 2 cm/year after reaching the maximum height in puberty, bone age reached 17 years old and older for male and 15 years old and older for female, or the reason for discontinuation was that subject reached the near final height or had epiphyseal closure.	Baseline, At Puberty, At Near Final Height within the span of approximately maximum of 11.58 years
Change in Height Standard Deviation (SD) Score at Puberty and at Near Final Height in Participants Who Reached Near Final Height From Baseline	Change in height SD score from baseline was calculated. Height SD score = (Height - Standard height for chronological age of gender) / SD. An SD score indicates how far a participant's score deviates from the mean of the reference population; an SD score higher than the mean gives a positive SD score whereas an SD score lower than the mean gives a negative SD score. SD score indicates how similar participant was to reference population. Near final height was defined as the height at one of the following conditions: the annual height velocity became less than 2 cm/year after reaching the maximum height in puberty, bone age reached 17 years old and older for male and 15 years old and older for female, or the reason for discontinuation was that subject reached the near final height or had epiphyseal closure.	Baseline, At Puberty, At Near Final Height within the span of approximately maximum of 11.58 years

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): September 5, 2013
• Primary Completion (Actual): February 28, 2017
• Study Completion (Actual): June 7, 2017

Study Registration Dates

• First Submitted: July 9, 2013
• First Submitted That Met QC Criteria: July 9, 2013
• First Posted (Estimated): July 12, 2013

Study Record Updates

• Last Update Posted (Actual): September 25, 2023
• Last Update Submitted That Met QC Criteria: August 31, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Keywords: Genotropin; Somatropin; SGA; Small for Gestational Age; Long Term Follow-Up; Special Investigation
• Other Study ID Numbers: A6281314

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD 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/data_requests.