Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq

NCT00234533 | Completed Phase 3 Results

• 2 other identifiers: 2-79-58035-7002004-000356-17
• Study Type: interventional
• Target: 251
• Locations: 14 countries
• Sites: 45

Brief Summary

The main purpose of this study is to establish an optimal monitoring regimen in NutropinAq treated children, using newly developed capillary blood spot IGF-1 measurement technology.

Conditions

Turner Syndrome; Renal Insufficiency, Chronic; Pituitary Diseases; Dwarfism

Keywords

growth; child development; growth hormone; inadequate growth hormone secretion; growth failure

Outcome Measures

Primary Outcomes (1)

• Insulin-Like Growth Factor I (IGF-I) Levels Measured Using the Timed Capillary Blood Spot Samples

  Fingertip capillary blood was collected using filter paper cards for the assay of capillary blood spot IGF-I in line with the monitoring recommendations of the Lawson Wilkins Paediatric Endocrine Society (LWPES) for treatment with recombinant GH therapy in children. Capillary IGF-I assays were performed by the patient at home one day per week during Weeks 21, 22 and 23 only (same week day). The samples were scheduled in the evening prior to the injection of NutropinAq and between 7:00 and 9:00 the following morning. An extended window from 6:00 to 12:00 was allowed for defining protocol deviations. The number of capillary blood spot IGF-I measurements and the optimal timing of samples to assess the IGF-I status of NutropinAq treated patients was assessed. IGF-I measurements for the morning and evening sampling are presented.

  At Weeks 21, 22 and 23

Secondary Outcomes (15)

• Assessment of IGF-I Levels: Categorised by Weekly Timing (Weeks 21-23) and Daily Timing (Morning and Evening)

  At Weeks 21, 22 and 23

• Assessment of IGF-I Levels: Categorised by Sex and Prepubertal Status

  At Weeks 21, 22 and 23

• Multivariate Linear Regression Analyses to Assess Factors Affecting the Variability of IGF-I Levels: Categorised by Disease Condition and Location

  Up to Week 24

• Multivariate Linear Regression Analyses to Assess Factors Affecting the Variability of IGF-I Levels: Categorised by Time of Year, Calculated Age at Enrolment and Disease Condition

  Up to Week 24

• Change From Baseline at Week 24 in the IGF-I Levels as Measured by Capillary Blood Spot Method and Serum IGF-I Assay

  Baseline to Week 24

Study Arms (1)

NutropinAq 10 mg/2 mL (30 IU)

EXPERIMENTAL

Patients received daily subcutaneous (s.c.) injections of NutropinAq 10 milligrams (mg)/2 milliliters (mL) for 6 months. The therapeutic daily doses administered were as follows: * GHD patients: 0.025 - 0.035 mg/ kilogram (kg) bodyweight * TS patients: up to 0.05 mg/kg bodyweight * CRI patients: up to 0.05 mg/kg bodyweight Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23. The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Drug: Somatropin (rDNA origin)

Interventions

Somatropin (rDNA origin) DRUG

Daily subcutaneous injections, 0,025 - 0,05 mg/kg/day for 6 months.

NutropinAq 10 mg/2 mL (30 IU)

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Children under 18 with growth failure associated with inadequate growth hormone secretion, or Turner syndrome or chronic renal insufficiency.

You may not qualify if:

• Children with closed epiphyses
• Children with active neoplasm
• Children with acute critical illness

Sponsors & Collaborators

• Ipsen: lead

Study Sites (45)

Dienst Kindergeneeskunde

Brussels, B-1090, Belgium

Location

Dienst Kindergeneeskunde

Edegem, B-2650, Belgium

Location

Klinika Deti a Dorostu

Prague, 100 34, Czechia

Location

Aalborg Sygehus Nord, Borneafdelingen

Aalborg, 9100, Denmark

Location

Sygeh. i Ringkjobing Amt, Borneafdeling

Herning, 7400, Denmark

Location

Helsinki University Central Hospital

Helsinki, Finland

Location

CHU - Hôtel Dieu

Angers, 49033, France

Location

Cabinet Médical

Bordeaux, 33000, France

Location

CHU Grenoble

Grenoble, 38043, France

Location

Centre Hospitalier General

Le Havre, 76083, France

Location

CHU Timone Enfants

Marseille, 13385, France

Location

CHU de Montpellier

Montpellier, 34295, France

Location

Hôpital Archet 2

Nice, 06202, France

Location

Hôpital Saint-Vincent de Paul

Paris, 75674, France

Location

Groupe Hospitalier de Necker

Paris, 75743, France

Location

Hôpital Charles Nicolle

Rouen, 76031, France

Location

CHU Hautepierre

Strasbourg, 67100, France

Location

Centre Hospitalier de Bigorre

Tarbes, 65013, France

Location

Cabinet Médical

Toulouse, 31000, France

Location

Hôpital des Enfants

Toulouse, 31059, France

Location

Centre Pédiatrique Gatien de Clocheville

Tours, 37044, France

Location

Universitätsklinikum Leipzig AöR

Leipzig, 04317, Germany

Location

Universitätsklinikum Tübingen

Tübingen, 72076, Germany

Location

General State Hospital of Nikaia

Athens, 18454, Greece

Location

PA Kyriakou Children's Hospital

Athens, Greece

Location

Azienda Policlinico - Università di Catania

Catania, 78-95123, Italy

Location

Ospedale Policlinico

Chieti, 5-66013, Italy

Location

Clinica Pediatrica II

Florence, 13-50132, Italy

Location

Il Università degli Studi di Napoli

Napoli, 4-80138, Italy

Location

Clinica Pediatrica, Universita Federico II di Napoli

Napoli, Italy

Location

Clinica Pediatrica

Novara, 18-28100, Italy

Location

Clinica Pediatrica

Parma, 14-43100, Italy

Location

Institutul de Endocrinologie C.I. Parhon

Bucharest, Sector 1, Romania

Location

Endocrinology Research Centre RAMS, Institute of Pediatric Endocrinology

Moscow, Russia

Location

Tushino Pediatric Hospital, RMAPE Department of Endocrinology for Childhood and Adolescent Age

Moscow, Russia

Location

Il Detska Klinika

Bratislava, 833 40, Slovakia

Location

Hospital de Nens de Barcelona

Barcelona, 08009, Spain

Location

Hospital General Universitario

Elche, 03203, Spain

Location

Hospital Gregorio Marañón

Madrid, 28007, Spain

Location

Hospital Parc Taulí

Sabadell, 08208, Spain

Location

Hospital Clínico Universitario

Santiago de Compostela, 15706, Spain

Location

Scientific-Research Institute of Endocrinology, Academy of Medical Science of Ukraine

Kiev, Ukraine

Location

Ukrainian Scientific practical Centre of Endocrine surgery, Endocrine Organs and Tissues Transplantation

Kiev, Ukraine

Location

St George's Hospital

London, England, SW17 0QT, United Kingdom

Location

University Hospital Wales

Cardiff, Wales, 14 4XW, United Kingdom

Location

MeSH Terms

Conditions

Turner Syndrome; Renal Insufficiency, Chronic; Pituitary Diseases; Dwarfism; Failure to Thrive

Interventions

Growth Hormone

Condition Hierarchy (Ancestors)

Gonadal Dysgenesis; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Sex Chromosome Disorders of Sex Development; Male Urogenital Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Sex Chromosome Disorders; Chromosome Disorders; Genetic Diseases, Inborn; Gonadal Disorders; Endocrine System Diseases; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Medical Director, Pediatric Endocrinology
• Organization: Ipsen

clinical.trials@ipsen.com

Study Officials

• Ipsen Medical Director

  Ipsen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 5, 2005
• First Posted: October 7, 2005
• Study Start: June 1, 2004
• Primary Completion: July 1, 2008
• Study Completion: July 1, 2008
• Last Updated: December 9, 2019
• Results First Posted: August 9, 2018

Record last verified: 2019-11

Locations

BE (2); UA (2); RO (1); GB (2); RU (2); ES (5); FI (1); CZ (1); SL (1); DK (2); DE (2); FR (15); GR (2); IT (7)