IGF1 Deficiency Clinical Trial
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).
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.
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
Ipsen |
United States, Austria, France, French Guiana, Germany, Italy, Martinique, Poland, Spain, Sweden, United Kingdom,
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. |
Status | Clinical Trial | Phase | |
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