A pharmacokinetic and pharmacodynamic study of recombinant human IGF-I (rhIGF-I) in three boys with ALS deficiency, and insulin sensitivity and bone density in patients and heterozygous first-degree relatives. - ALS deficiency: insulin resistance, bone strength & response to rhIGF1
- Conditions
- ALS (acid label subunit) deficiencyMedDRA version: 12.1Level: LLTClassification code 10056438Term: Growth Hormone Deficiency
- Registration Number
- EUCTR2010-019296-30-GB
- Lead Sponsor
- niversity Hospital Birmingham NHS Foundation Trust
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria
PK/PD study arm: Patients with confirmed IGFALS gene mutations, able to give informed consent
Cross-sectional study arm: Heterozygous first-degree relatives, able to give informed consent.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion Criteria
PK/PD study arm: Patients currently receiving IncrelexTM treatment, patients allergic to Increlex, patients with cancer
Cross-sectional study arm: Individuals suffering from cancer or chronic inflammatory conditions (e.g. chronic rheumatoid arthritis, inflammatory bowels disease).
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method