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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00368173




Registration number
NCT00368173
Ethics application status
Date submitted
23/08/2006
Date registered
24/08/2006
Date last updated
13/09/2012

Titles & IDs
Public title
IGF-I/IGFBP-3 Therapy in Children and Adolescents With Growth Hormone Insenitivity Syndrome (GHIS) Such as Laron Syndrome
Scientific title
Secondary ID [1] 0 0
INSM-110-303
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Growth Hormone Insensitivity Syndrome (GHIS) 0 0
Laron Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - rhIGF-I/rhIGFBP-3

Treatment: Drugs: rhIGF-I/rhIGFBP-3
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes

Eligibility
Key inclusion criteria
1. A diagnosis of GHIS such as Laron syndrome,

2. 2 - 18 years of age,

3. Height less than or equal to -3SD for age,

4. Pre-pubertal, defined as Tanner breast stage 1 or testis volume <4mL
Minimum age
2 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
Key exclusion criteria
1. Children in puberty,

2. Diagnosed malignancy,

3. A diagnosis of diabetes mellitus

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2/Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Dr. Bruce King - Newcastle
Recruitment postcode(s) [1] 0 0
- Newcastle
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
New York
Country [2] 0 0
Argentina
State/province [2] 0 0
Buenos Aires
Country [3] 0 0
Argentina
State/province [3] 0 0
Tucuman
Country [4] 0 0
Brazil
State/province [4] 0 0
Sao Paulo
Country [5] 0 0
China
State/province [5] 0 0
Hong Kong
Country [6] 0 0
Egypt
State/province [6] 0 0
Cairo
Country [7] 0 0
Germany
State/province [7] 0 0
Berlin
Country [8] 0 0
Israel
State/province [8] 0 0
Haifa
Country [9] 0 0
Italy
State/province [9] 0 0
Naples
Country [10] 0 0
Norway
State/province [10] 0 0
Oslo
Country [11] 0 0
Peru
State/province [11] 0 0
Lima
Country [12] 0 0
Slovakia
State/province [12] 0 0
Kosice
Country [13] 0 0
Turkey
State/province [13] 0 0
Ankara
Country [14] 0 0
United Kingdom
State/province [14] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Insmed Incorporated
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
STUDY OBJECTIVE

To evaluate the safety, tolerability, and efficacy, as growth velocity (statural growth), of
rhIGF-I/rhIGFBP-3 administered for 12 months in pre-pubertal children and adolescents with
GHIS.

STUDY DESIGN

This study is an open-label, multi-center clinical trial to evaluate the safety and
effectiveness of rhIGF-I/rhIGFBP-3 to increase rate of growth when administered once daily
for 12 months in children and adolescents with growth hormone insensitivity syndrome (GHIS)
such as Laron Syndrome. At the end of the initial twelve-month treatment period, additional
safety and long-term efficacy data will be assessed in a second 12 month treatment period.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00368173
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries