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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00903110




Registration number
NCT00903110
Ethics application status
Date submitted
30/04/2009
Date registered
15/05/2009
Date last updated
1/11/2024

Titles & IDs
Public 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).
Scientific 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).
Secondary ID [1] 0 0
EUPAS7708
Secondary ID [2] 0 0
2-79-52800-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
IGF1 Deficiency 0 0
Condition category
Condition code

Intervention/exposure
Study type
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Treatment: Drugs - Increlex®

Treatment: Drugs: 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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of SAEs (including AESI of neoplasia) and all AEs, targeted AEs, deaths and withdrawals due to AEs.
Timepoint [1] 0 0
During the treatment period up to 30 days after the last dose.
Secondary outcome [1] 0 0
Incidence of SAEs (including AESI of neoplasia), targeted AEs, all AEs, deaths, withdrawals due to AEs, special situations and concomitant medications
Timepoint [1] 0 0
Within 5 years post-treatment
Secondary outcome [2] 0 0
Incidence of special situations and concomitant medications
Timepoint [2] 0 0
During the treatment period an average of 5 years and within 5 years post-treatment
Secondary outcome [3] 0 0
Changes in height Standard Deviation Score (SDS)
Timepoint [3] 0 0
From baseline at least up to 5 years or until the final adult height is achieved.
Secondary outcome [4] 0 0
Height velocity
Timepoint [4] 0 0
From baseline at least up to 5 years or until the final adult height is achieved.
Secondary outcome [5] 0 0
Bone age development
Timepoint [5] 0 0
From baseline at least up to 5 years or until the final adult height is achieved
Secondary outcome [6] 0 0
Body mass index (BMI)
Timepoint [6] 0 0
From baseline at least up to 5 years or until the final adult height is achieved.
Secondary outcome [7] 0 0
Pubertal stage
Timepoint [7] 0 0
From baseline at least up to 5 years or until the final adult height is achieved.
Secondary outcome [8] 0 0
Estimation of differences between predicted adult height (PAH) and final adult height (FAH)
Timepoint [8] 0 0
From baseline at least up to 5 years or until the final adult height is achieved.
Secondary outcome [9] 0 0
Modelisation to identify predictive factors of height SDS change
Timepoint [9] 0 0
From baseline at least up to 5 years or until the final adult height is achieved.
Secondary outcome [10] 0 0
Modelisation to identify predictive factors of Height velocity
Timepoint [10] 0 0
From baseline at least up to 5 years or until the final adult height is achieved
Secondary outcome [11] 0 0
Modelisation to identify predictive factors of FAH
Timepoint [11] 0 0
From baseline at least up to 5 years or until the final adult height is achieved
Secondary outcome [12] 0 0
Modelisation to identify predictive factors of pubertal (Tanner) stage
Timepoint [12] 0 0
From baseline at least up to 5 years or until the final adult height is achieved
Secondary outcome [13] 0 0
Modelisation to identify predictive factors of bone age development
Timepoint [13] 0 0
From baseline at least up to 5 years or until the final adult height is achieved
Secondary outcome [14] 0 0
Dose of Increlex® administrated
Timepoint [14] 0 0
Periodically assessed during the study until treatment stop at least up to 5 years.
Secondary outcome [15] 0 0
Duration of Increlex exposure
Timepoint [15] 0 0
Periodically assessed during the study until treatment stop at least up to 5 years.
Secondary outcome [16] 0 0
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)).
Timepoint [16] 0 0
Periodically assessed during the study until treatment stop at least up to 5 years.
Secondary outcome [17] 0 0
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)).
Timepoint [17] 0 0
Periodically assessed during the study until treatment stop at least up to 5 years.
Secondary outcome [18] 0 0
Biological assessment : baseline GH concentrations, IGF-1 levels, IGFBP-3 levels and binding proteins.
Timepoint [18] 0 0
Throughout study at least up to 5 years.
Secondary outcome [19] 0 0
Presence or absence of gene deletion/mutation
Timepoint [19] 0 0
Throughout study at least up to 5 years.
Secondary outcome [20] 0 0
Changes in QoL assessment using EQ-5D in participant aged 4 and over.
Timepoint [20] 0 0
At baseline, at year one, at least up to 5 years, at Final Adult Height.
Secondary outcome [21] 0 0
Description of neoplasia (benign and malignant) and hypoglycaemia
Timepoint [21] 0 0
Within the first 3 years after treatment start, between 3 and 5 years and over 5 years.

Eligibility
Key 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
Minimum age
2 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
9/12/2008
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/12/2027
Actual
Sample size
Target
500
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Ohio
Country [4] 0 0
United States of America
State/province [4] 0 0
Texas
Country [5] 0 0
Austria
State/province [5] 0 0
Vöcklabruck
Country [6] 0 0
France
State/province [6] 0 0
Amiens
Country [7] 0 0
France
State/province [7] 0 0
Blois
Country [8] 0 0
France
State/province [8] 0 0
Bondy
Country [9] 0 0
France
State/province [9] 0 0
Bron
Country [10] 0 0
France
State/province [10] 0 0
Clermont-Ferrand
Country [11] 0 0
France
State/province [11] 0 0
Marseille
Country [12] 0 0
France
State/province [12] 0 0
Montpellier
Country [13] 0 0
France
State/province [13] 0 0
Mulhouse
Country [14] 0 0
France
State/province [14] 0 0
Nice
Country [15] 0 0
France
State/province [15] 0 0
Paris
Country [16] 0 0
France
State/province [16] 0 0
Toulouse
Country [17] 0 0
French Guiana
State/province [17] 0 0
Cayenne
Country [18] 0 0
Germany
State/province [18] 0 0
Erlangen
Country [19] 0 0
Germany
State/province [19] 0 0
Heidelberg
Country [20] 0 0
Germany
State/province [20] 0 0
Homburg
Country [21] 0 0
Germany
State/province [21] 0 0
Magdeburg
Country [22] 0 0
Germany
State/province [22] 0 0
Oldenburg
Country [23] 0 0
Italy
State/province [23] 0 0
Ancona
Country [24] 0 0
Italy
State/province [24] 0 0
Bolzano
Country [25] 0 0
Italy
State/province [25] 0 0
Brescia
Country [26] 0 0
Italy
State/province [26] 0 0
Firenze
Country [27] 0 0
Italy
State/province [27] 0 0
Genova
Country [28] 0 0
Italy
State/province [28] 0 0
Macerata
Country [29] 0 0
Italy
State/province [29] 0 0
Naples
Country [30] 0 0
Italy
State/province [30] 0 0
Parma
Country [31] 0 0
Italy
State/province [31] 0 0
Reggio Emilia
Country [32] 0 0
Italy
State/province [32] 0 0
Roma
Country [33] 0 0
Martinique
State/province [33] 0 0
Fort-de-France
Country [34] 0 0
Poland
State/province [34] 0 0
Bialystok
Country [35] 0 0
Poland
State/province [35] 0 0
Gdansk
Country [36] 0 0
Poland
State/province [36] 0 0
Lublin
Country [37] 0 0
Poland
State/province [37] 0 0
Poznan
Country [38] 0 0
Poland
State/province [38] 0 0
Rzeszów
Country [39] 0 0
Poland
State/province [39] 0 0
Szczecin
Country [40] 0 0
Spain
State/province [40] 0 0
Barcelona
Country [41] 0 0
Spain
State/province [41] 0 0
Bilbao
Country [42] 0 0
Spain
State/province [42] 0 0
Reus
Country [43] 0 0
Spain
State/province [43] 0 0
Valencia
Country [44] 0 0
Sweden
State/province [44] 0 0
Linkoping
Country [45] 0 0
Sweden
State/province [45] 0 0
Stockholm
Country [46] 0 0
United Kingdom
State/province [46] 0 0
Belfast
Country [47] 0 0
United Kingdom
State/province [47] 0 0
Birmingham
Country [48] 0 0
United Kingdom
State/province [48] 0 0
Leeds
Country [49] 0 0
United Kingdom
State/province [49] 0 0
London
Country [50] 0 0
United Kingdom
State/province [50] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ipsen
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT00903110
Trial related presentations / publications
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.
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.
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Ipsen Medical Director
Address 0 0
Ipsen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Ipsen Recruitment Enquiries
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT00903110