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

NCT01491854 | Terminated Results

• 1 other identifier: CEP00-402
• Study Type: interventional
• Target: 130
• Locations: 6 countries
• Sites: 23

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).

Conditions

Short Children Born Small for Gestational Age (SGA)

Keywords

Long-term; safety; follow-up; growth hormone treatment; short children; Small for Gestational Age; Somatropin

Outcome Measures

Primary Outcomes (5)

• Evaluate the Long-term Effect of Growth Hormone Treatment on the Development of Diabetes After End of Therapy.

  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

  5 years

• To Evaluate the Long Term Effects of rhGH on Carbohydrate Metabolism Through Fasting Plasma Glucose (FPG) Levels

  Supportive to Primary Endpoint

  baseline, 6 months, 1 year, 5 years

• To Evaluate the Long Term Effects of rhGH on Carbohydrate Metabolism Through Fasting Insulin Levels

  Supportive to Primary Endpoint

  baseline, 6 months, 1 year, 5 years

• To Evaluate the Long Term Effects of rhGH on Carbohydrate Metabolism Through Glucose Glycolsylated Hemoglobin (HbA1c)

  Supportive to Primary Endpoint

  baseline, 6 months, 1 year, 5 years

• To Evaluate the Long Term Effects of rhGH on Carbohydrate Metabolism Through HOMA and QUICKI Scores

  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.

  baseline, 6 months, 1 year, 5 years

Secondary Outcomes (3)

• 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

• to Evaluate Final Height

  baseline, 6 months, 1 year, 5 years

Study Arms (1)

Monitoring of long-term safety

OTHER

Long-term safety follow-up after the end of treatment with Omnitrope (single arm)

Other: Bloodsampling

Interventions

Bloodsampling OTHER

Bloodsampling

Monitoring of long-term safety

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Sandoz: lead

Study Sites (23)

Novartis Investigative Site

Ústí nad Labem, Czech Republic, 400 11, Czechia

Location

Novartis Investigative Site

Hradec Králové, 500 05, Czechia

Location

Novartis Investigative Site

Prague, 150 06, Czechia

Location

Novartis Investigative Site

Tbilisi, 144, Georgia

Location

Novartis Investigative Site

Nordrhein Westfalen, Sankt Augustin, 53757, Germany

Location

Novartis Investigative Site

München, 80337, Germany

Location

Novartis Investigative Site

Miskolc, 3526, Hungary

Location

Novartis Investigative Site

Poznai, Greater Poland Voivodeship, 60-572, Poland

Location

Novartis Investigative Site

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85667, Poland

Location

Novartis Investigative Site

Wroclaw, Lower Silesian Voivodeship, 50-311, Poland

Location

Novartis Investigative Site

Wroclaw, Lower Silesian Voivodeship, 50-368, Poland

Location

Novartis Investigative Site

Rzeszów, Podkarpackie Voivodeship, 35-301, Poland

Location

Novartis Investigative Site

Katowice, Silesian Voivodeship, 40-752, Poland

Location

Novartis Investigative Site

Zabrze, Silesian Voivodeship, 41-800, Poland

Location

Novartis Investigative Site

Gdansk, 80 952, Poland

Location

Novartis Investigative Site

Krakow, 30-663, Poland

Location

Novartis Investigative Site

Lodz, 93-338, Poland

Location

Novartis Investigative Site

Szczecin, 71-252, Poland

Location

Novartis Investigative Site

Warsaw, 04 730, Poland

Location

Novartis Investigative Site

Kielce, Świętokrzyskie Voivodeship, 25734, Poland

Location

Novartis Investigative Site

Lasi, Iaşi, 700111, Romania

Location

Novartis Investigative Site

Bucharest, 20395, Romania

Location

Novartis Investigative Site

Cluj-Napoca, CLUJ, Romania

Location

Limitations and Caveats

The study was terminated prematurely in accordance with the request to close study EP00-402 "EMEA/H/C/000607/MEA 10.2" submitted to the EMA on 29-Mar-2018 and adopted by the EMA on 28-Jun-2018

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharma AG

novartis.email@novartis.com

862-778-8300

Study Officials

• Sandoz Biopharmaceuticals Sandoz

  Sandoz GmbH

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2011
• First Posted: December 14, 2011
• Study Start: July 20, 2009
• Primary Completion: October 31, 2018
• Study Completion: October 31, 2018
• Last Updated: August 14, 2019
• Results First Posted: August 14, 2019

Record last verified: 2019-07

Locations

HU (1); DE (2); PL (13); RO (3); CZ (3); GE (1)