Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213)

Study Overview
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Brief Summary
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This is a multi-national trial. The goals of the trial are to study the growth response to LUM-201 administration in children with idiopathic growth hormone deficiency (GHD) previously treated with daily rhGH for 12 months in the LUM-201-01 trial.

Detailed Description
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This trial will have one screening/baseline visit with tests to assess if subjects are eligible to start study therapy. The screening/baseline visit can coincide with the Month 12 visit in the LUM-201-01 trial. Subjects will receive the same dose of LUM-201 as a daily oral dose each morning. LUM-201 will be adjusted for body weight increments at each clinic visit, as applicable. The trial consists of 12 months of treatment. After screening, subjects will return to the clinic for 5 visits. During four of these clinic visits, safety laboratory samples will be collected.

Official Title
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A Multicenter, Open-Label, Phase 2 Study to Evaluate Growth and Safety of LUM-201 Following 12 Months of Daily rhGH Treatment in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed the LUM-201-01 Trial

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 previously completed 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial. Is eligible for study participation as confirmed by the principal investigator (PI) Exclusion Criteria: Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201. Uses any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors. 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

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 13 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
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: LUM-201 (3.2 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 |
| --- | --- | --- |
| Annualized height velocity (AHV) measured as standing height with stadiometer | Annualized Height Velocity (AHV) measured as standing height with stadiometer | Day 1 to Month 12 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of adverse events in children with GHD | Number of events | Day 1 to Month 12 | 
| GH Concentration on maintenance treatment | Serum GH concentration | Day 1 to Month 12 | 
| Insulin-like growth factor 1 | Serum concentrations of insulin-like growth factor 1 | Day 1 to Month 12 | 
| Insulin-like growth factor binding protein 3 | Serum concentrations of insulin-like growth factor binding protein 3 | Day 1 to Month 12 | 
| Height standard deviation score (SDS) | Change in HT-SDS | Day 1 to Month 12 | 
| Change in Weight | Change in Weight | Day 1 to Month 12 | 
| Change in Weight-SDS | Change in Weight-SDS | Day 1 to Month 12 | 
| Change in BMI | Change in BMI | Day 1 to Month 12 | 
| Change in BMI SDS | Change in BMI SDS | Day 1 to Month 12 | 
| Change in Bone Age | Change in bone age, measured by X-ray of left hand and wrist using Greulich & Pyle atlas | Month 6 | 
| Insulin-like growth factor 1 standard deviation score (SDS) | Change in IGF-1 SDS | Day 1 to Month 12 | 

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