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).

NCT00903110 | Recruiting DMC

• 2 other identifiers: 2-79-52800-002EUPAS7708
• Study Type: observational
• Target: 500
• Locations: 10 countries
• Sites: 45

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.

Conditions

IGF1 Deficiency

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (21)

• Incidence of SAEs (including AESI of neoplasia), targeted AEs, all AEs, deaths, withdrawals due to AEs, special situations and concomitant medications

  Within 5 years post-treatment

• Incidence of special situations and concomitant medications

  During the treatment period an average of 5 years and within 5 years post-treatment

• Changes in height Standard Deviation Score (SDS)

  From baseline at least up to 5 years or until the final adult height is achieved.

• Height velocity

  From baseline at least up to 5 years or until the final adult height is achieved.

• Bone age development

  From baseline at least up to 5 years or until the final adult height is achieved

Interventions

Increlex® DRUG

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

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Children and adolescents with Growth Failure due to Severe Primary IGF-1 Deficiency

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Esteve Pharmaceuticals, S.A.: lead

Study Sites (58)

Children's Hospital of Orange County

Orange, California, 92868, United States

RECRUITING

University of Miami Leonard M Miller

Miami, Florida, 33136, United States

RECRUITING

University Of Miami Leonard M. Miller

Miami, Florida, 33136, United States

RECRUITING

D&H National Research Centers

Miami, Florida, 33155, United States

WITHDRAWN

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

NOT YET RECRUITING

Children's Health Specialty Center West Plano

Plano, Texas, 75093, United States

WITHDRAWN

Salzkammergut-Klinik Vöcklabruck

Vöcklabruck, A-4840, Austria

RECRUITING

Hôpital Amiens-Picardie

Amiens, 80080, France

TERMINATED

Centre Hospitalier de Blois

Blois, 41000, France

RECRUITING

Hôpital Jean Verdier

Bondy, 93140, France

RECRUITING

Hôpital Femme Mère-Enfant

Bron, 69500, France

RECRUITING

Hôpital Estaing

Clermont-Ferrand, 63100, France

RECRUITING

Hôpital Timone Enfants

Marseille, 13005, France

RECRUITING

Hôpital Arnaud de Villeneuve

Montpellier, 34090, France

RECRUITING

GHR Mulhouse Sud-Alsace

Mulhouse, France

TERMINATED

Pole medical Bel'Air

Mulhouse, France

RECRUITING

CHU Lenval

Nice, 06200, France

WITHDRAWN

Hôpital Necker Enfants Malades

Paris, 75015, France

RECRUITING

Hôpital Kremlin Bicetre

Paris, 97270, France

RECRUITING

Hôpital des Enfants

Toulouse, 31300, France

RECRUITING

Hôpital de Cayenne

Cayenne, 97306, French Guiana

WITHDRAWN

Universitätsklinikum Erlangen Kinder- und Jugendklinik

Erlangen, 91054, Germany

RECRUITING

Universitätsklinikum Heidelberg Kinderheilkunde

Heidelberg, 69120, Germany

RECRUITING

Universitätskliniken des Saarlandes Kinderklinik

Homburg, 66424, Germany

RECRUITING

Klinikum der Otto von Guericke Universität

Magdeburg, 39120, Germany

TERMINATED

Klinikum Oldenburg

Oldenburg, 26133, Germany

TERMINATED

Diabetologia Pediatrica Azienda Ospedaliero-Universitaria

Ancona, Italy

NOT YET RECRUITING

Ospedale di Bolzano

Bolzano, 39100, Italy

RECRUITING

Spedali Civili di Brescia

Brescia, 25100, Italy

TERMINATED

Azienda ospedaliera universitaria Meyer

Florence, 50139, Italy

NOT YET RECRUITING

I.R.C.C.S. Giannina Gaslini

Genova, 16147, Italy

RECRUITING

U.O. Pediatria e Neonatologia Ospedale di Macerata

Macerata, Italy

TERMINATED

Azienda Ospedaliera Universitaria II

Naples, 80138, Italy

NOT YET RECRUITING

Azienda Ospedaliera-Universitaria di Parma

Parma, Italy

NOT YET RECRUITING

Azienda USL-IRCCS

Reggio Emilia, Italy

RECRUITING

Ospedale Pediatrico Bambino Gesù

Roma, 00165, Italy

RECRUITING

Hôpital Pierre Zobda Quitman

Fort-de-France, 97200, Martinique

RECRUITING

Samodzielny Publiczny Dzieciecy Szpital Kliniczny

Bialystok, 15-274, Poland

RECRUITING

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-294, Poland

RECRUITING

Uniwersytecki Szpital Dziecięcy w Lublinie

Lublin, 20-093, Poland

RECRUITING

Szpital kliniczny im. Karola Jonschnera

Poznan, 60-572, Poland

RECRUITING

Kliniczny Szpital Wojewódzki

Rzeszów, 35-301, Poland

RECRUITING

Pomeranian Medical University

Szczecin, Poland

RECRUITING

Hospital Univ Vall d'Hebrón

Barcelona, 08035, Spain

TERMINATED

Hospital Parc Taulí de Sabadell

Barcelona, 08208, Spain

RECRUITING

Hospital Sant Joan de Déu

Barcelona, 08950, Spain

RECRUITING

Hospital Univ. de Cruces

Bilbao, 48903, Spain

RECRUITING

Hospital Universitari Sant Joan de Reus

Reus, 43204, Spain

RECRUITING

Hospital Universitario y Politécnico La Fe

Valencia, 46026, Spain

RECRUITING

Linköping University Hospital

Linköping, 57850, Sweden

RECRUITING

Karolinska Universitetssjukhuset

Stockholm, 17176, Sweden

RECRUITING

Royal Belfast Hospital for Sick Children

Belfast, BT12 6BA, United Kingdom

RECRUITING

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

TERMINATED

Leeds General Infirmary

Leeds, LS1 3EX, United Kingdom

RECRUITING

The Royal London Hospital

London, E1 1FR, United Kingdom

RECRUITING

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

RECRUITING

Royal Manchester Children's Hospital

Manchester, M13 0JH, United Kingdom

TERMINATED

Related Publications (4)

• 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 DERIVED

• 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 DERIVED

• 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 DERIVED

• 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 DERIVED

Biospecimen

Retention: NONE RETAINED

Study Design Time Perspective: Retrospective and Prospective

MeSH Terms

Conditions

Insulin-Like Growth Factor I Deficiency

Interventions

mecasermin

Study Officials

• Global Program Clinical Lead

  Esteve

  STUDY DIRECTOR

Central Study Contacts

Esteve Global Clinical Development

CONTACT

increlexregistry@esteve.com

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: OTHER
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 30, 2009
• First Posted: May 15, 2009
• Study Start: December 9, 2008
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: February 6, 2026

Record last verified: 2026-02

Locations

FR (1); GB (6); PL (6); ES (6); DE (5); AU (1); MA (1); SE (2); IT (10); US (7)