Skip to main content
OpenTrials
Recruiting

NCT Number: 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).

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

IGF1 Deficiency Insulin-Like Growth Factor I Deficiency

Age range

2 year–18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Salzkammergut-Klinik Vöcklabruck, Vöcklabruck, Austria

Loading trial locations.

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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

Primary outcomes

  1. Incidence of SAEs (including AESI of neoplasia) and all AEs, targeted AEs, deaths and withdrawals due to AEs.

    Time frame: During the treatment period up to 30 days after the last dose.

    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.

Secondary outcomes

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

    Time frame: Within 5 years post-treatment

    In the overall population, and in the subset of children and adolescents exposed to Increlex® for at least 3 cumulative years excluding interruptions.

  2. Incidence of special situations and concomitant medications

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

  3. Changes in height Standard Deviation Score (SDS)

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

  4. Height velocity

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

  5. Bone age development

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

  6. Body mass index (BMI)

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

  7. Pubertal stage

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

  8. Estimation of differences between predicted adult height (PAH) and final adult height (FAH)

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

  9. Modelisation to identify predictive factors of height SDS change

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

  10. Modelisation to identify predictive factors of Height velocity

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

  11. Modelisation to identify predictive factors of FAH

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

  12. Modelisation to identify predictive factors of pubertal (Tanner) stage

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

  13. Modelisation to identify predictive factors of bone age development

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

  14. Dose of Increlex® administrated

    Time frame: Periodically assessed during the study until treatment stop at least up to 5 years.

  15. Duration of Increlex exposure

    Time frame: Periodically assessed during the study until treatment stop at least up to 5 years.

  16. 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)).

    Time frame: Periodically assessed during the study until treatment stop at least up to 5 years.

    This analysis will support the description of the lowest effective dose

  17. 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)).

    Time frame: Periodically assessed during the study until treatment stop at least up to 5 years.

    This analysis will support the description of the lowest effective dose

  18. Biological assessment : baseline GH concentrations, IGF-1 levels, IGFBP-3 levels and binding proteins.

    Time frame: Throughout study at least up to 5 years.

  19. Presence or absence of gene deletion/mutation

    Time frame: Throughout study at least up to 5 years.

    including: GH gene, IGF-1 gene, FGF, PTPN11, GHR, D3-GHR, STAT5b, ALS, SHOX, PAPPA2 and any other genetic tests performed.

  20. Changes in QoL assessment using EQ-5D in participant aged 4 and over.

    Time frame: At baseline, at year one, at least up to 5 years, at Final Adult Height.

    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.

  21. Description of neoplasia (benign and malignant) and hypoglycaemia

    Time frame: Within the first 3 years after treatment start, between 3 and 5 years and over 5 years.

Study contacts

Contact information is provided by the study sponsor or research team.

Esteve Global Clinical Development

CONTACT

[email protected]

Sponsors and collaborators

Lead sponsor

Esteve Pharmaceuticals, S.A.

Industry

Registry information

Important dates

Study start
2008
Primary completion
2027
Study completion
2027
First posted
May 15, 2009
Registry last updated
Feb 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.