An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children with Idiopathic Growth Hormone Deficiency
- Conditions
- Idiopathic Growth Hormone DeficiencyTherapeutic area: Diseases [C] - Hormonal diseases [C19]
- Registration Number
- CTIS2023-504020-25-00
- Lead Sponsor
- umos Pharma Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 69
1. Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable., 2. Must have successfully participated in a pediatric LUM-201 GHD study through at least the 12-month visit, and be eligible for continuation of treatment, pending all other enrollment criteria are met. Specific enrollment criteria for subjects in each LUM-201 trial are listed below. Subjects transitioning from the LUM-201-01 trial will be considered eligible for enrollment pending successful participation in the LUM-201-01 trial, having met an AHV of = 6.7 cm/year after 12 months of LUM-201 therapy. Subjects transitioning from the LUM-201-04 trial will be considered eligible for enrollment pending successful completion of 12 months of therapy with rhGH on the LUM-201-01 trial, followed by 12 months of treatment with LUM-201 in the LUM-201-04 trial and having met an AHV of = 80% of first year growth on rhGH., 3. Subjects who are sexually active must use an acceptable form of contraception., 4. Is eligible for a Day 1 visit as confirmed by the principal investigator (PI).
1. Has a medical or genetic condition that, in the opinion of the PI and/or MMs, adds unwarranted risk to use of LUM-201., 2. Has planned or is receiving current long-term treatment with medications known to act as substrates, inducers, or inhibitors of the cytochrome system CYP3A4 that metabolizes LUM-201 (see Appendix 1 of protocol for list of example medications). Subjects receiving shorter-term (two weeks or less) treatment with these medications should be evaluated on case-by-case basis by the PI in consultation with the MMs.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Assess the long-term safety of LUM-201 administration in children with idiopathic growth hormone deficiency (GHD);Secondary Objective: Assess pharmacodynamic (PD) markers of LUM-201 treatment, Assess long-term growth outcomes in response to LUM-201 treatment;Primary end point(s): 1. Occurrence of AEs, SAEs; laboratory results, and physical examination findings.
- Secondary Outcome Measures
Name Time Method Secondary end point(s):1. GH values: actual and change over time (every 6 months).;Secondary end point(s):2. IGF-1 values: actual and change over time (every 6 months).;Secondary end point(s):3. IGFBP-3 values: actual and change over time (every 6 months).;Secondary end point(s):4. Change in height standard deviation score (HT-SDS) every 6 months.;Secondary end point(s):5. Change in weight and weight SDS every 6 months.;Secondary end point(s):6. Change in body mass index (BMI) and BMI SDS every 6 months.;Secondary end point(s):7. Change in Bone Age (BA) compared to Chronological Age (CA) (e.g., BA/CA) ratio assessed annually.