Growth Hormone Deficiency
Conditions
Keywords
LUM-201, PGHD, GHD, Growth hormone secretagogue, Oral, Height
Brief summary
This is a multi-national trial. The trial aims to study the long-term safety of LUM-201 in subjects with Idiopathic Pediatric Growth Hormone Deficiency (iPGHD). This study will also assess pharmacodynamics and efficacy response to therapy with LUM-201.
Detailed description
An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children with Idiopathic Growth Hormone Deficiency. This study will last for up to 36 months to allow collection of additional long-term safety and efficacy data related to LUM-201 treatment in the pediatric patient population. Subjects will enter into this trial after successful participation in a prior LUM-201 study. This study will consist of visits every 6 months through three years. There are a total of 7 in-person visits with a follow-up phone call between visits. At the clinic visits, subjects will have a physical exam and blood collection as well as efficacy assessments.
Interventions
DRUGLUM-201
Administered orally once daily
Sponsors
Lumos Pharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open Label
Eligibility
Sex/Gender
ALL
Age
5 Years to 14 Years
Healthy volunteers
No
Inclusion criteria
* Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable. * 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.
Exclusion criteria
* Medical or genetic condition that, in the opinion of the PI and/or MMs, adds unwarranted risk to the use of LUM-201 * 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. 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of treatment-emergent adverse events | Day 1 to Month 36 | Number of events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Growth Hormone Concentrations in subjects | Day 1 to Month 36 | Serum GH concentration |
| Insulin-like growth factor 1 concentration | Day 1 to Month 36 | Serum concentrations of insulin-like growth factor 1 |
| Change in Weight SDS | Day 1 to Month 36 | Change in Weight |
| Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201 | Day 1 to Month 36 | (AHV) measured as standing height with stadiometer |
| Change in Bone Age (BA) | Day 1 to Month 36 | Change in Bone Age (BA) compared to Chronological Age |
| Height standard deviation score (SDS) | Day 1 to Month 36 | Change in HT-SDS |
Countries
Australia, United States
Outcome results
None listed