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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
16
Administered orally once daily
Rady Children's Hospital
San Diego, California, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
Indiana University School of Medicine
Indianapolis, Indiana, United States
Annualized Height Velocity (AHV) Measured as Standing Height With Stadiometer
Mean change from day 1 to month 12, Annualized Height Velocity (AHV) Height is measured in triplicate, by a calibrated stadiometer and taking the mean value AVH equation is (h2-h1/t2-t1)\*365.25; h1 = height measured at day 1 h2 = height measured at month 12 t1 = day 1 t2 = month 12
Time frame: Day 1 to Month 12
Incidence of Adverse Events in Children With GHD
Number of events
Time frame: Day 1 to Month 12
Growth Hormone (GH) Values
Mean GH Values change from Day 1 to month 12 (Pre-Dose and 1 hour Post-Dose)
Time frame: Day 1 to Month 12
Insulin-Like Growth Factor 1 (IGF-1) Values
Mean change from Day 1 to month 12 (IGF-1 Values Pre-Dose)
Time frame: Day 1 to Month 12
Insulin-Like Growth Factor Binding Protein 3 (IGFBP-3) Values
Mean change from day 1 to month 12 (IGFBP-3 Values Pre-Dose)
Time frame: Day 1 to Month 12
Height Standard Deviation Score (SDS)
Mean change from day 1 to month 12 in HT-SDS
Time frame: Day 1 to Month 12
Change in Weight
Mean change in Weight from day 1 to month 12
Time frame: Day 1 to Month 12
Change in Weight - Standard Deviation Score (SDS)
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M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic
Minneapolis, Minnesota, United States
The Children's Mercy Hospital
Kansas City, Missouri, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
University of Virginia Health System
Charlottesville, Virginia, United States
Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki
Lodz, Poland
Sonomed - Centrum Medyczne
Szczecin, Poland
Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka
Warsaw, Poland
Mean change in Weight-SDS from day 1 to month 12
Time frame: Day 1 to Month 12
Change in Body Mass Index (BMI)
Mean change from day 1 to month 12 in BMI
Time frame: Day 1 to Month 12
Change in Body Mass Index (BMI) - Standard Deviation Score (SDS)
Mean change from Day 1 to month 12 in BMI-SDS
Time frame: Day 1 to Month 12
Change in Bone Age
Mean change from day 1 to month 6 in bone age, measured by X-ray of left hand and wrist using Greulich \& Pyle atlas
Time frame: Month 6
Insulin-Like Growth Factor 1 (IGF-1) - Standard Deviation Score (SDS)
Mean Change from day 1 to month 12 for IGF-1 - SDS Pre-dose Values
Time frame: Day 1 to Month 12