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
82
Administered orally once daily
Administered subcutaneously (s.c., under the skin) once daily.
Percentage of subjects selected by PEM strategy who meet target growth
Annualized height velocity (AHV) measured as standing height with stadiometer
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
Degree of concordance between the first and second assessment with the PEM strategy.
Peak serum concentration of GH in response to a single provocative dose of LUM-201
Time frame: Screening to Day 1
Incidence of adverse events in children with GHD
Number of events
Time frame: Day 1 to Month 24
Height standard deviation score (SDS)
Change in HT-SDS
Time frame: Day 1 to Month 6 and Month 12
Height velocity standard deviation score (HV-SDS)
Change in HV-SDS
Time frame: Day 1 to Month 6, and Month 12
Change in Weight
Change in Weight
Time frame: Day 1 to Month 6, and Month 12
Change in Weight SDS
Change in Weight-SDS
Time frame: Day 1 to Month 6 and Month 12
Change in BMI
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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
Change in BMI
Time frame: Day 1 to Month 6 and Month 12
Change in BMI SDS
Change in BMI SDS
Time frame: Day 1 to Month 6 and 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 and Month 18
Pharmacokinetics of LUM-201
Serum concentrations (Cmax/Steady State)
Time frame: Day 1 to Month 6 and 12
GH Concentration on maintenance treatment
Serum GH concentration
Time frame: Day 1 to Month 6 and 12
Insulin-like growth factor 1 SDS
Serum concentrations of insulin-like growth factor 1
Time frame: Day 1 to Month 6 and 12