A Long-Term Safety Trial of LUM-201 in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed a LUM-201 Clinical Trial (OraGrowtH211)
- Registration Number
- NCT05796440
- Lead Sponsor
- Lumos Pharma
- 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.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 60
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description LUM-201 (1.6 mg/kg/day) LUM-201 -
- Primary Outcome Measures
Name Time Method Incidence of treatment-emergent adverse events Day 1 to Month 36 Number of events
- Secondary Outcome Measures
Name Time Method 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
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
Height standard deviation score (SDS) Day 1 to Month 36 Change in HT-SDS
Trial Locations
- Locations (7)
Canberra Hospital
🇦🇺Garran, Australian Capital Territory, Australia
University of Iowa
🇺🇸Iowa City, Iowa, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
UMass Memorial Medical Center
🇺🇸Worcester, Massachusetts, United States
University of South Carolina
🇺🇸Charleston, South Carolina, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Texas Tech University Health Sciences Center
🇺🇸Amarillo, Texas, United States