Clinical trial NCT00903110: Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).
Official title: Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: IGF1 Deficiency
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2009-05-15
Enrollment: 500
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children and adolescents with Growth Failure due to Severe Primary IGF-1 Deficiency

Brief summary: The Increlex® Global Registry is a descriptive, multicenter, observational, prospective, open-ended, non interventional, post-authorisation surveillance registry.

The main purpose of this global registry is to collect, analyse and report safety data during and up to at least 5 years after the end of treatment in children and adolescents receiving Increlex® therapy for SPIGFD according to the locally approved product information.

Detailed description: This registry is a Post-Authorisation Safety Study called the Increlex® Global Registry which is intended primarily to monitor the safety of Increlex® therapy in children and adolescents with Severe Primary IGF-1 Deficiency and secondly to follow the effectiveness of this treatment. Patients who have already started Increlex® therapy before entering this registry may be included and data will be collected retrospectively.

The countries participating in this registry are Austria, France, Germany, Italy, Poland, Spain, Sweden, United Kingdom and the USA

Eligibility criteria: Inclusion Criteria:

* For US : patients starting or planning to start or currently receiving treatment with Increlex® therapy for severe primary IGF-1 deficiency as defined by the US Increlex® prescribing information or for growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH.For EU : patients starting or planning to start or currently receiving treatment with Increlex® therapy according to the locally approved product information.
* Parents or legally authorized representatives if applicable must give signed informed consent before any registry-related activities are conducted. Assent from the subject should also be obtained as appropriate

Exclusion Criteria:

* Subject currently participating in an Increlex® clinical trial
* Subject currently participating in any clinical trial for growth retardation
* Patient with any contraindication to Increlex® or any condition subject to special warning as per the locally approved label
* For US patients, these include patients with hypersensitivity to the active substance or any of the excipients, patients with active or suspected neoplasia and patients with closed epiphyses.
* For EU patients: these include patients with hypersensitivity to the active substance or any of the excipients, patients with active or suspected neoplasia or any condition or medical history which increases the risk of benign or malignant neoplasia and patients with closed epiphyses

Primary outcomes:
measure: Incidence of SAEs (including AESI of neoplasia) and all AEs, targeted AEs, deaths and withdrawals due to AEs.
timeFrame: During the treatment period up to 30 days after the last dose.
Secondary outcomes:
measure: Incidence of SAEs (including AESI of neoplasia), targeted AEs, all AEs, deaths, withdrawals due to AEs, special situations and concomitant medications
timeFrame: Within 5 years post-treatment
measure: Incidence of special situations and concomitant medications
timeFrame: During the treatment period an average of 5 years and within 5 years post-treatment
measure: Changes in height Standard Deviation Score (SDS)
timeFrame: From baseline at least up to 5 years or until the final adult height is achieved.
measure: Height velocity
timeFrame: From baseline at least up to 5 years or until the final adult height is achieved.
measure: Bone age development
timeFrame: From baseline at least up to 5 years or until the final adult height is achieved
measure: Body mass index (BMI)
timeFrame: From baseline at least up to 5 years or until the final adult height is achieved.
measure: Pubertal stage
timeFrame: From baseline at least up to 5 years or until the final adult height is achieved.
measure: Estimation of differences between predicted adult height (PAH) and final adult height (FAH)
timeFrame: From baseline at least up to 5 years or until the final adult height is achieved.
measure: Modelisation to identify predictive factors of height SDS change
timeFrame: From baseline at least up to 5 years or until the final adult height is achieved.
measure: Modelisation to identify predictive factors of Height velocity
timeFrame: From baseline at least up to 5 years or until the final adult height is achieved
measure: Modelisation to identify predictive factors of FAH
timeFrame: From baseline at least up to 5 years or until the final adult height is achieved
measure: Modelisation to identify predictive factors of pubertal (Tanner) stage
timeFrame: From baseline at least up to 5 years or until the final adult height is achieved
measure: Modelisation to identify predictive factors of bone age development
timeFrame: From baseline at least up to 5 years or until the final adult height is achieved
measure: Dose of Increlex® administrated
timeFrame: Periodically assessed during the study until treatment stop at least up to 5 years.
measure: Duration of Increlex exposure
timeFrame: Periodically assessed during the study until treatment stop at least up to 5 years.
measure: Description of effectiveness parameters height SDS according to average dose received and according to dose ranges (e.g. 4 dose ranges (≤50, ]50-80], ]80-110], > 110 μg/kg BID)).
timeFrame: Periodically assessed during the study until treatment stop at least up to 5 years.
measure: Description of effectiveness parameters height velocity according to average dose received and according to dose ranges (e.g. 4 dose ranges (≤50, ]50-80], ]80-110], > 110 μg/kg BID)).
timeFrame: Periodically assessed during the study until treatment stop at least up to 5 years.
measure: Biological assessment : baseline GH concentrations, IGF-1 levels, IGFBP-3 levels and binding proteins.
timeFrame: Throughout study at least up to 5 years.
measure: Presence or absence of gene deletion/mutation
timeFrame: Throughout study at least up to 5 years.
measure: Changes in QoL assessment using EQ-5D in participant aged 4 and over.
timeFrame: At baseline, at year one, at least up to 5 years, at Final Adult Height.
measure: Description of neoplasia (benign and malignant) and hypoglycaemia
timeFrame: Within the first 3 years after treatment start, between 3 and 5 years and over 5 years.
Other outcomes:
Locations:
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: Miami
country: United States
facility: University of Miami Leonard M Miller
state: Florida
city: Miami
country: United States
facility: University Of Miami Leonard M. Miller
state: Florida
city: Miami
country: United States
facility: D&H National Research Centers
state: Florida
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Plano
country: United States
facility: Children's Health Specialty Center West Plano
state: Texas
city: Vöcklabruck
country: Austria
facility: Salzkammergut-Klinik Vöcklabruck
city: Amiens
country: France
facility: Hôpital Amiens-Picardie
city: Blois
country: France
facility: Centre Hospitalier de Blois
city: Bondy
country: France
facility: Hôpital Jean Verdier
city: Bron
country: France
facility: Hôpital Femme Mère-Enfant
city: Clermont-Ferrand
country: France
facility: Hôpital Estaing
city: Marseille
country: France
facility: Hôpital Timone Enfants
city: Montpellier
country: France
facility: Hôpital Arnaud de Villeneuve
city: Mulhouse
country: France
facility: GHR Mulhouse Sud-Alsace
city: Mulhouse
country: France
facility: Pole medical Bel'Air
city: Nice
country: France
facility: CHU Lenval
city: Paris
country: France
facility: Hôpital Necker Enfants Malades
city: Paris
country: France
facility: Hôpital Kremlin Bicetre
city: Toulouse
country: France
facility: Hôpital des Enfants
city: Cayenne
country: French Guiana
facility: Hôpital de Cayenne
city: Erlangen
country: Germany
facility: Universitätsklinikum Erlangen Kinder- und Jugendklinik
city: Heidelberg
country: Germany
facility: Universitätsklinikum Heidelberg Kinderheilkunde
city: Homburg
country: Germany
facility: Universitätskliniken des Saarlandes Kinderklinik
city: Magdeburg
country: Germany
facility: Klinikum der Otto von Guericke Universität
city: Oldenburg
country: Germany
facility: Klinikum Oldenburg
city: Ancona
country: Italy
facility: Diabetologia Pediatrica Azienda Ospedaliero-Universitaria
city: Bolzano
country: Italy
facility: Ospedale di Bolzano
city: Brescia
country: Italy
facility: Spedali Civili di Brescia
city: Firenze
country: Italy
facility: Azienda ospedaliera universitaria Meyer
city: Genova
country: Italy
facility: I.R.C.C.S. Giannina Gaslini
city: Macerata
country: Italy
facility: U.O. Pediatria e Neonatologia Ospedale di Macerata
city: Naples
country: Italy
facility: Azienda Ospedaliera Universitaria II
city: Parma
country: Italy
facility: Azienda Ospedaliera-Universitaria di Parma
city: Reggio Emilia
country: Italy
facility: Azienda USL-IRCCS
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesù
city: Fort-de-France
country: Martinique
facility: Hôpital Pierre Zobda Quitman
city: Białystok
country: Poland
facility: Samodzielny Publiczny Dzieciecy Szpital Kliniczny
city: Gdańsk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
city: Lublin
country: Poland
facility: Uniwersytecki Szpital Dziecięcy w Lublinie
city: Poznań
country: Poland
facility: Szpital kliniczny im. Karola Jonschnera
city: Rzeszów
country: Poland
facility: Kliniczny Szpital Wojewódzki
city: Szczecin
country: Poland
facility: Pomeranian Medical University
city: Barcelona
country: Spain
facility: Hospital Univ Vall d'Hebrón
city: Barcelona
country: Spain
facility: Hospital Parc Taulí de Sabadell
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Déu
city: Bilbao
country: Spain
facility: Hospital Univ. de Cruces
city: Reus
country: Spain
facility: Hospital Universitari Sant Joan de Reus
city: Valencia
country: Spain
facility: Hospital Universitario y Politécnico La Fe
city: Linkoping
country: Sweden
facility: Linköping University Hospital
city: Stockholm
country: Sweden
facility: Karolinska Universitetssjukhuset
city: Belfast
country: United Kingdom
facility: Royal Belfast Hospital for Sick Children
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Leeds
country: United Kingdom
facility: Leeds General Infirmary
city: London
country: United Kingdom
facility: The Royal London Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References:
citation: Bang P, Polak M, Perrot V, Sert C, Shaikh H, Woelfle J. Pubertal Timing and Growth Dynamics in Children With Severe Primary IGF-1 Deficiency: Results From the European Increlex(R) Growth Forum Database Registry. Front Endocrinol (Lausanne). 2022 Feb 18;13:812568. doi: 10.3389/fendo.2022.812568. eCollection 2022.
citation: Bang P, Woelfle J, Perrot V, Sert C, Polak M. Effectiveness and safety of rhIGF1 therapy in patients with or without Laron syndrome. Eur J Endocrinol. 2021 Feb;184(2):267-276. doi: 10.1530/EJE-20-0325.