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)

Study Overview
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Brief Summary
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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
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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.

Official Title
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An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children With Idiopathic Growth Hormone Deficiency

Conditions
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Growth Hormone Deficiency

Intervention / Treatment
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* Drug: LUM-201


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 14 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Open Label
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: LUM-201 (1.6 mg/kg/day)<br> | Drug: LUM-201<br>* Administered orally once daily<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent adverse events | Number of events | Day 1 to Month 36 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Growth Hormone Concentrations in subjects | Serum GH concentration | Day 1 to Month 36 | 
| Insulin-like growth factor 1 concentration | Serum concentrations of insulin-like growth factor 1 | Day 1 to Month 36 | 
| Height standard deviation score (SDS) | Change in HT-SDS | Day 1 to Month 36 | 
| Change in Weight SDS | Change in Weight | Day 1 to Month 36 | 
| Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201 | (AHV) measured as standing height with stadiometer | Day 1 to Month 36 | 
| Change in Bone Age (BA) | Change in Bone Age (BA) compared to Chronological Age | Day 1 to Month 36 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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LUM-201, PGHD, GHD, Growth hormone secretagogue, Oral, Height