Long-term Safety Follow-up After Growth Hormone Treatment of Short Children Born Small for Gestational Age

Not Applicable

Terminated

• Conditions: Short Children Born Small for Gestational Age (SGA)
• Interventions: Other: Bloodsampling

• Registration Number: NCT01491854
• Lead Sponsor: Sandoz

Brief Summary

This study is performed as part of the Marketing Authorisation Holder's post-marketing pharmacovigilance plan to investigate the long-term safety, in particular the diabetogenic potential and immunogenicity of rhGH therapy in short children born small for gestational age (SGA).

Detailed Description

The purpose of this study is

1. to monitor short children born SGA who were treated with growth hormone in study EP00-401 for the development of diabetes for a further 10 years after termination of growth hormone treatment

and

2. to report the incidence of anti-rhGH antibodies and of E. coli host cell peptide (HCP) antibodies (ABs) for 6 months after termination of GH treatment.

Study Timeline

• Study Start: 2009-07-20
• Study First Submitted: 2011-12-12
• Study First Submitted QC: 2011-12-13
• Study First Posted: 2011-12-14
• Primary Completion: 2018-10-31
• Study Completion: 2018-10-31
• Results First Submitted: 2019-04-29
• Status Verified: 2019-07
• Results First Submitted QC: 2019-07-17
• Last Update Submitted: 2019-07-17
• Results First Posted: 2019-08-14
• Last Update Posted: 2019-08-14

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 130

Inclusion Criteria

• All patients who fulfilled the diagnosis SGA, participated in study EP00-401, and received at least one dose of study medication
• Written informed consent of patient (for children who can read and/ or understand) and/or parent or legal guardian

Exclusion Criteria

• Patients unwilling and/or parents/guardians who are not capable of ensuring compliance with the provisions of the study protocol

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Monitoring of long-term safety	Bloodsampling	Long-term safety follow-up after the end of treatment with Omnitrope (single arm)

Primary Outcome Measures

Name	Time	Method
Evaluate the Long-term Effect of Growth Hormone Treatment on the Development of Diabetes After End of Therapy.	5 years	Number of participants diagnosed with Diabetes mellitus type 2 during the study, defined as fullfilment of these 3 criteria: * FPG ≥ 126 mg/dl (7.0 mmol/L) during blood sampling and/or during Oral Glucose Tolerance Test (OGTT); * 2-h plasma glucose ≥ 200 mg/dl (11.1 mmol/L) during an OGTT; * Investigator documenting diagnosis of diabetes mellitus type 2 during OGTT
To Evaluate the Long Term Effects of rhGH on Carbohydrate Metabolism Through Fasting Plasma Glucose (FPG) Levels	baseline, 6 months, 1 year, 5 years	Supportive to Primary Endpoint
To Evaluate the Long Term Effects of rhGH on Carbohydrate Metabolism Through Fasting Insulin Levels	baseline, 6 months, 1 year, 5 years	Supportive to Primary Endpoint
To Evaluate the Long Term Effects of rhGH on Carbohydrate Metabolism Through Glucose Glycolsylated Hemoglobin (HbA1c)	baseline, 6 months, 1 year, 5 years	Supportive to Primary Endpoint
To Evaluate the Long Term Effects of rhGH on Carbohydrate Metabolism Through HOMA and QUICKI Scores	baseline, 6 months, 1 year, 5 years	Supportive to Primary Endpoint. HOMA = homeostasis model assessment for Insulin resistance: Healthy Range: 1.0 (0.5-1.4).; \< 1.0 means you are insulin-sensitive which is optimal. \>1.9 indicates early insulin resistance. \> 2.9 indicates significant insulin resistance. The quantitative insulin sensitivity check index (QUICKI) measures insulin sensitivity, which is the inverse of insulin resistance. The QUICKI calculation for insulin resistance in humans fall broadly within a range between 0.45 for unusually healthy individuals and 0.30 in diabetics. Lower numbers reflect greater insulin resistance.

Secondary Outcome Measures

Name	Time	Method
to Evaluate IGF-I and IGFBP-3 Levels After End of Growth Hormone Treatment	baseline, 6 months, 1 year , 5 years
To Evaluate the Incidence of Anti-rhGH Antibodies After Termination of Growth Hormone Treatment.	baseline, 6 months, 1 year, 5 years	number of participants with positive results for anti-drug antibody (ADA). Percentages indicated are calculated based on the total number of patients (118 participants).
to Evaluate Final Height	baseline, 6 months, 1 year, 5 years

Trial Locations

Locations (1)

Novartis Investigative Site; 🇷🇴 Cluj Napoca, Romania