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).

Обзор

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.

Подробное описание

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

Вмешательства

• Препарат 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

Первичные конечные точки

• Incidence of SAEs (including AESI of neoplasia) and all AEs, targeted AEs, deaths and withdrawals due to AEs. [Срок оценки: During the treatment period up to 30 days after the last dose.]

Вторичные конечные точки (12)

• Incidence of SAEs (including AESI of neoplasia), targeted AEs, all AEs, deaths, withdrawals due to AEs, special situations and concomitant medications [Срок оценки: Within 5 years post-treatment]
• Incidence of special situations and concomitant medications [Срок оценки: During the treatment period an average of 5 years and within 5 years post-treatment]
• Changes in height Standard Deviation Score (SDS) [Срок оценки: From baseline at least up to 5 years or until the final adult height is achieved.]
• Height velocity [Срок оценки: From baseline at least up to 5 years or until the final adult height is achieved.]
• Bone age development [Срок оценки: From baseline at least up to 5 years or until the final adult height is achieved]
• Body mass index (BMI) [Срок оценки: From baseline at least up to 5 years or until the final adult height is achieved.]
• Pubertal stage [Срок оценки: From baseline at least up to 5 years or until the final adult height is achieved.]
• Estimation of differences between predicted adult height (PAH) and final adult height (FAH) [Срок оценки: From baseline at least up to 5 years or until the final adult height is achieved.]
• Modelisation to identify predictive factors of height SDS change [Срок оценки: From baseline at least up to 5 years or until the final adult height is achieved.]
• Modelisation to identify predictive factors of Height velocity [Срок оценки: From baseline at least up to 5 years or until the final adult height is achieved]
• Modelisation to identify predictive factors of FAH [Срок оценки: From baseline at least up to 5 years or until the final adult height is achieved]
• Modelisation to identify predictive factors of pubertal (Tanner) stage [Срок оценки: From baseline at least up to 5 years or until the final adult height is achieved]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Только случаи

Центры проведения

Франция · 13 центров

• Hôpital Amiens-Picardie — Amiens
• Centre Hospitalier de Blois — Blois
• Hôpital Jean Verdier — Bondy
• Hôpital Femme Mère-Enfant — Bron
• Hôpital Estaing — Clermont-Ferrand
• Hôpital Timone Enfants — Marseille
• Hôpital Arnaud de Villeneuve — Montpellier
• GHR Mulhouse Sud-Alsace — Mulhouse
• … и ещё 5 центров

Италия · 10 центров

• Diabetologia Pediatrica Azienda Ospedaliero-Universitaria — Ancona
• Ospedale di Bolzano — Bolzano
• Spedali Civili di Brescia — Brescia
• Azienda ospedaliera universitaria Meyer — Florence
• I.R.C.C.S. Giannina Gaslini — Genova
• Azienda Ospedaliera Universitaria II — Naples
• Azienda Ospedaliera-Universitaria di Parma — Parma
• U.O. Pediatria e Neonatologia Ospedale di Macerata — Province of Macerata
• … и ещё 2 центра

США · 7 центров

• Children's Hospital of Orange County — Orange
• University of Miami Leonard M Miller — Miami
• University Of Miami Leonard M. Miller — Miami
• D&H National Research Centers — Miami
• Cincinnati Children's Hospital Medical Center — Cincinnati
• UT Southwestern Medical Center — Dallas
• Children's Health Specialty Center West Plano — Plano

Польша · 6 центров

• Samodzielny Publiczny Dzieciecy Szpital Kliniczny — Bialystok
• Uniwersyteckie Centrum Kliniczne — Gdansk
• Uniwersytecki Szpital Dziecięcy w Lublinie — Lublin
• Szpital kliniczny im. Karola Jonschnera — Poznan
• Kliniczny Szpital Wojewódzki — Rzeszów
• Pomeranian Medical University — Szczecin

Испания · 6 центров

• Hospital Univ Vall d'Hebrón — Barcelona
• Hospital Parc Taulí de Sabadell — Barcelona
• Hospital Sant Joan de Déu — Barcelona
• Hospital Univ. de Cruces — Bilbao
• Hospital Universitari Sant Joan de Reus — Reus
• Hospital Universitario y Politécnico La Fe — Valencia

Великобритания · 6 центров

• Royal Belfast Hospital for Sick Children — Belfast
• Birmingham Children's Hospital — Birmingham
• Leeds General Infirmary — Leeds
• The Royal London Hospital — London
• Great Ormond Street Hospital — London
• Royal Manchester Children's Hospital — Manchester

Германия · 5 центров

• Universitätsklinikum Erlangen Kinder- und Jugendklinik — Erlangen
• Universitätsklinikum Heidelberg Kinderheilkunde — Heidelberg
• Universitätskliniken des Saarlandes Kinderklinik — Homburg
• Klinikum der Otto von Guericke Universität — Magdeburg
• Klinikum Oldenburg — Oldenburg

Швеция · 2 центра

• Linköping University Hospital — Linköping
• Karolinska Universitetssjukhuset — Stockholm

Австрия · 1 центр

• Salzkammergut-Klinik Vöcklabruck — Vöcklabruck

French Guiana · 1 центр

• Hôpital de Cayenne — Cayenne

Martinique · 1 центр

• Hôpital Pierre Zobda Quitman — Fort-de-France

Публикации

• Ramon-Krauel M, Polak M, Maghnie M, Woelfle J, Sert C, Perrot V, Bang P. Near-Adult Height Outcomes in Patients Treated With rhIGF-1 for Severe Growth Failure: Real-World IGFD Registry Data. J Clin Endocrinol Metab. 2026 Jan 21;111(2):e500-e511. doi: 10.1210/clinem/dgaf390. PMID 40626687
• Bang P, Polak M, Bossowski A, Maghnie M, Argente J, Ramon-Krauel M, Sert C, Perrot V, Mazain S, Woelfle J. Frequency and Predictive Factors of Hypoglycemia in Patients Treated With rhIGF-1: Data From the Eu-IGFD Registry. J Clin Endocrinol Metab. 2023 Dec 21;109(1):46-56. doi: 10.1210/clinem/dgad479. PMID 37579214
• 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. PMID 35250870
• 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. PMID 33434161

Идентификаторы

NCT: NCT00903110 · 2-79-52800-002 · EUPAS7708

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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