This is a multi-national trial. The goals of the trial are to study LUM-201 as a possible treatment for Pediatric Growth Hormone Deficiency (PGHD) and investigate a predictive enrichment marker (PEM) strategy to select subjects likely to respond to therapy with LUM-201.
This trial will have one screening visit with tests 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 be randomized to receive one of three oral daily doses of LUM-201 or daily injections of recombinant human growth hormone (rhGH). All subjects will have an equal chance of being placed in any of the four groups. The trial consists of up to 24 months of treatment. After screening, subjects will return to the clinic for 6 (subjects placed in rhGH group) or 10 visits (subjects placed in LUM-201 group). During several of these clinic visits, subjects will have a physical exam, blood, and urine collections. There will also be 3 phone calls with study staff that will take place between the clinic visits.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
104
Administered orally once daily
Administered subcutaneously (s.c., under the skin) once daily.
Percentage of Participants With a Positive Growth Response From Day 1 to Month 6 (AHV >= 6.85 cm/yr)
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 6 t1 = day 1 t2 = month 6
Time frame: Day 1 to Month 6
AHV After 6 Months on LUM-201 Compared to rhGH
Annualized height velocity to be measured.
Time frame: Day 1 to Month 6
Percentage of Participants With a Positive Result on Both PEM Tests (at Screening and on Day 1)
PEM Test Reproducibility Safety Population
Time frame: Screening to Day 1
Change in Height Standard Deviation Score (SDS)
Full Analysis Set Population month 6
Time frame: Day 1 to Month 6
Change in Height Standard Deviation Score (SDS)
Full Analysis Set Population month 12
Time frame: Day 1 to Month 12
Change in Weight From Baseline
Change in Weight from baseline
Time frame: Day 1 to Month 6
Change in Weight From Baseline
Change in Weight from baseline
Time frame: Day 1 to Month 12
Change in Weight SDS
Change in Weight-SDS (Standard Deviation Score)
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Center of Excellence in Diabetes and Endocrinology
Sacramento, California, United States
Rady Children's Hospital
San Diego, California, United States
Pediatric Endocrine Associates
Greenwood Village, Colorado, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
Atlanta Diabetes Associates
Atlanta, Georgia, United States
Indiana University School of Medicine
Indianapolis, Indiana, United States
University of Iowa
Iowa City, Iowa, United States
Novak Center For Childrens Health
Louisville, Kentucky, United States
UMass Memorial Medical Center
Worcester, Massachusetts, United States
M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic
Minneapolis, Minnesota, United States
...and 33 more locations
Time frame: Day 1 to Month 6
Change in Weight SDS
Change in Weight-SDS (Standard Deviation Score)
Time frame: Day 1 to Month 12
Change in Body Mass Index (BMI)
Change in BMI from baseline
Time frame: Day 1 to Month 6
Change in BMI
Change in BMI (Body Mass Index) from baseline
Time frame: Day 1 to Month 12
Change in BMI SDS
Change in BMI SDS
Time frame: Day 1 to Month 6
Change in BMI SDS
Change in BMI SDS This is not a Z-Score analysis. Standard deviation score of 0 is the population mean for the age/sex. Normal range is typically -2 to +2 SDS. A positive score is an improvement and a negative score a decrease.
Time frame: Day 1 to Month 12
Bone Age
Change in bone age, measured by X-ray of left hand and wrist using Greulich \& Pyle atlas
Time frame: Day 1 to Month 6
Bone Age
Change in bone age, measured by X-ray of left hand and wrist using Greulich \& Pyle atlas
Time frame: Day 1 to Month 18
Pharmacokinetics of LUM-201
Serum concentrations at 30 minutes (Cmax/Steady State)
Time frame: Day 1 to Month 6
Growth Hormone (GH) Concentration on Maintenance Treatment
Serum GH concentration
Time frame: Month 6
Growth Hormone (GH) Concentration on Maintenance Treatment
Serum GH concentration
Time frame: Month 12
Insulin-like Growth Factor 1 SDS
Serum concentrations of insulin-like growth factor 1 This is not a Z-Score analysis. Standard deviation score of 0 is the population mean for the age/sex. Normal range is typically -2 to +2 SDS. A positive score is an improvement and a negative score a decrease.
Time frame: Month 6
Insulin-like Growth Factor 1 SDS
Serum concentrations of insulin-like growth factor 1 This is not a Z-Score analysis. Standard deviation score of 0 is the population mean for the age/sex. Normal range is typically -2 to +2 SDS. A positive score is an improvement and a negative score a decrease.
Time frame: Month 12