The goals of this single site trial are to study the pharmacokinetics (PK) and pharmacodynamics of LUM-201 and effects of LUM-201 administration on growth hormone release over time in children with idiopathic pediatric growth hormone deficiency (PGHD).
This trial will have a single screening visit to assess if subjects are eligible to start study therapy. Once subjects have completed screening, and if they are determined to be eligible, they will return to the clinic for a 12-hour pulsatility study. On Day Two of the trial, subjects will be randomized to receive one of two oral daily doses of LUM-201 and PK samples will be collected. All subjects will have an equal chance of being placed in either of the two groups. The trial consists of treatment until subjects achieve near adult height. After screening, subjects will return to the clinic monthly for the first 6 visits and every three months thereafter. At most clinic visits, subjects will have a physical exam, blood and urine collections. At the Month 6 visit subjects will repeat the 12-hour pulsatility and PK studies.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
24
Administered orally once daily
Institute of Maternal and Child Research, University of Chile
Santiago, Chile
Evaluation of Augmented Growth Hormone (GH) Pulsatility
12-hour mean GH concentration
Time frame: Day 1 to Month 6
Pharmacokinetics of LUM-201 and M8
Plasma concentrations (Cmax/Steady State)
Time frame: Day 1 to Month 6
Incidence of adverse events in children with idiopathic GHD
Number of events
Time frame: up to 8 years
Height standard deviation score (SDS)
Change in HT-SDS from Baseline to Month 6 and Month 12
Time frame: Day 1 to Month 12
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