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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT00903110
Other study ID # 2-79-52800-002
Secondary ID EUPAS7708
Status Recruiting
Phase
First received
Last updated
Start date December 9, 2008
Est. completion date December 31, 2027

Study information

Verified date May 2024
Source Ipsen
Contact Ipsen Recruitment Enquiries
Email clinical.trials@ipsen.com
Is FDA regulated No
Health authority
Study type Observational [Patient Registry]

Clinical Trial 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.


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


Recruitment information / eligibility

Status Recruiting
Enrollment 500
Est. completion date December 31, 2027
Est. primary completion date December 31, 2027
Accepts healthy volunteers No
Gender All
Age group 2 Years to 18 Years
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Increlex®
Increlex® (mecasermin [rDNA origin] injection), 10 mg/ml solution for injection, 40-120mcg/kg BID or 0,04 to 0,12 mg/kg BID, as prescribed by physician

Locations

Country Name City State
Austria Salzkammergut-Klinik Vöcklabruck Vöcklabruck
France Hôpital Amiens-Picardie Amiens
France Centre Hospitalier de Blois Blois
France Hôpital Jean Verdier Bondy
France Hôpital Femme Mère-Enfant Bron
France Hôpital Estaing Clermont-Ferrand
France Hôpital Timone Enfants Marseille
France Hôpital Arnaud de Villeneuve Montpellier
France GHR Mulhouse Sud-Alsace Mulhouse
France CHU Lenval Nice
France Hôpital Kremlin Bicetre Paris
France Hôpital Necker Enfants Malades Paris
France Hôpital des Enfants Toulouse
French Guiana Hôpital de Cayenne Cayenne
Germany Universitätsklinikum Erlangen Kinder- und Jugendklinik Erlangen
Germany Universitätsklinikum Heidelberg Kinderheilkunde Heidelberg
Germany Universitätskliniken des Saarlandes Kinderklinik Homburg
Germany Klinikum der Otto von Guericke Universität Magdeburg
Germany Klinikum Oldenburg Oldenburg
Italy Diabetologia Pediatrica Azienda Ospedaliero-Universitaria Ancona
Italy Ospedale di Bolzano Bolzano
Italy Spedali Civili di Brescia Brescia
Italy Azienda ospedaliera universitaria Meyer Firenze
Italy I.R.C.C.S. Giannina Gaslini Genova
Italy U.O. Pediatria e Neonatologia Ospedale di Macerata Macerata
Italy Azienda Ospedaliera Universitaria II Naples
Italy Azienda Ospedaliera-Universitaria di Parma Parma
Italy Azienda USL-IRCCS Reggio Emilia
Italy Ospedale Pediatrico Bambino Gesù Roma
Martinique Hôpital Pierre Zobda Quitman Fort-de-France
Poland Samodzielny Publiczny Dzieciecy Szpital Kliniczny Bialystok
Poland Uniwersyteckie Centrum Kliniczne Gdansk
Poland Uniwersytecki Szpital Dzieciecy w Lublinie Lublin
Poland Szpital kliniczny im. Karola Jonschnera Poznan
Poland Kliniczny Szpital Wojewódzki Rzeszów
Poland Pomeranian Medical University Szczecin
Spain Hospital Parc Taulí de Sabadell Barcelona
Spain Hospital Sant Joan de Déu Barcelona
Spain Hospital Univ Vall d'Hebrón Barcelona
Spain Hospital Univ. de Cruces Bilbao
Spain Hospital Universitari Sant Joan de Reus Reus
Spain Hospital Universitario y Politécnico La Fe Valencia
Sweden Linköping University Hospital Linkoping
Sweden Karolinska Universitetssjukhuset Stockholm
United Kingdom Royal Belfast Hospital for Sick Children Belfast
United Kingdom Birmingham Children's Hospital Birmingham
United Kingdom Leeds General Infirmary Leeds
United Kingdom Great Ormond Street Hospital London
United Kingdom The Royal London Hospital London
United Kingdom Royal Manchester Children's Hospital Manchester
United States Cincinnati Children's Hospital Medical Center Cincinnati Ohio
United States UT Southwestern Medical Center Dallas Texas
United States D&H National Research Centers Miami Florida
United States University of Miami Leonard M Miller Miami Florida
United States University Of Miami Leonard M. Miller Miami Florida
United States Children's Hospital of Orange County Orange California
United States Children's Health Specialty Center West Plano Plano Texas

Sponsors (1)

Lead Sponsor Collaborator
Ipsen

Countries where clinical trial is conducted

United States,  Austria,  France,  French Guiana,  Germany,  Italy,  Martinique,  Poland,  Spain,  Sweden,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Incidence of SAEs (including AESI of neoplasia) and all AEs, targeted AEs, deaths and withdrawals due to AEs. Targeted AE includes hypersensitivity; scoliosis; immunogenicity (presence of antibodies if available); slipped capital femoral epiphysis, headache, otitis media, papilloedema, hypoglycaemia (suspected or documented - documented means blood level glucose < 50 mg/dL or 2.78 mmol/L), acromegalic facial changes, gynaecomastia, hearing loss, intracranial hypertension, lipohypertrophy at injection sites, sleep apnoea, tonsillar hypertrophy, cardiomegaly, oedema and myalgia. During the treatment period up to 30 days after the last dose.
Secondary Incidence of SAEs (including AESI of neoplasia), targeted AEs, all AEs, deaths, withdrawals due to AEs, special situations and concomitant medications In the overall population, and in the subset of children and adolescents exposed to Increlex® for at least 3 cumulative years excluding interruptions. Within 5 years post-treatment
Secondary Incidence of special situations and concomitant medications During the treatment period an average of 5 years and within 5 years post-treatment
Secondary Changes in height Standard Deviation Score (SDS) From baseline at least up to 5 years or until the final adult height is achieved.
Secondary Height velocity From baseline at least up to 5 years or until the final adult height is achieved.
Secondary Bone age development From baseline at least up to 5 years or until the final adult height is achieved
Secondary Body mass index (BMI) From baseline at least up to 5 years or until the final adult height is achieved.
Secondary Pubertal stage From baseline at least up to 5 years or until the final adult height is achieved.
Secondary Estimation of differences between predicted adult height (PAH) and final adult height (FAH) From baseline at least up to 5 years or until the final adult height is achieved.
Secondary Modelisation to identify predictive factors of height SDS change From baseline at least up to 5 years or until the final adult height is achieved.
Secondary Modelisation to identify predictive factors of Height velocity From baseline at least up to 5 years or until the final adult height is achieved
Secondary Modelisation to identify predictive factors of FAH From baseline at least up to 5 years or until the final adult height is achieved
Secondary Modelisation to identify predictive factors of pubertal (Tanner) stage From baseline at least up to 5 years or until the final adult height is achieved
Secondary Modelisation to identify predictive factors of bone age development From baseline at least up to 5 years or until the final adult height is achieved
Secondary Dose of Increlex® administrated Periodically assessed during the study until treatment stop at least up to 5 years.
Secondary Duration of Increlex exposure Periodically assessed during the study until treatment stop at least up to 5 years.
Secondary 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)). This analysis will support the description of the lowest effective dose Periodically assessed during the study until treatment stop at least up to 5 years.
Secondary 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)). This analysis will support the description of the lowest effective dose Periodically assessed during the study until treatment stop at least up to 5 years.
Secondary Biological assessment : baseline GH concentrations, IGF-1 levels, IGFBP-3 levels and binding proteins. Throughout study at least up to 5 years.
Secondary Presence or absence of gene deletion/mutation including: GH gene, IGF-1 gene, FGF, PTPN11, GHR, D3-GHR, STAT5b, ALS, SHOX, PAPPA2 and any other genetic tests performed. Throughout study at least up to 5 years.
Secondary Changes in QoL assessment using EQ-5D in participant aged 4 and over. The QoL will be assessed using the EQ-5D-Y paediatric questionnaire. The 5 domains and VAS will be described at each timepoint as well as the evolution from baseline. At baseline, at year one, at least up to 5 years, at Final Adult Height.
Secondary Description of neoplasia (benign and malignant) and hypoglycaemia Within the first 3 years after treatment start, between 3 and 5 years and over 5 years.
See also
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