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

Phase 2

Active, not recruiting

Brief Summary: 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: 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
LUM-201 (3.2 mg/kg/day)	LUM-201	-

Primary Outcome Measures

Name	Time	Method
Annualized height velocity (AHV) measured as standing height with stadiometer	Day 1 to Month 12	Annualized Height Velocity (AHV) measured as standing height with stadiometer

Secondary Outcome Measures

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

Locations (10): Nationwide Children's Hospital
🇺🇸
Columbus, Ohio, United States
The Children's Mercy Hospital
🇺🇸
Kansas City, Missouri, United States
Rady Children's Hospital
🇺🇸
San Diego, California, United States
Sonomed - Centrum Medyczne
🇵🇱
Szczecin, Poland
M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic
🇺🇸
Minneapolis, Minnesota, United States
University of Virginia Health System
🇺🇸
Charlottesville, Virginia, United States
Indiana University School of Medicine
🇺🇸
Indianapolis, Indiana, United States
Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki
🇵🇱
Lodz, Poland
Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka
🇵🇱
Warsaw, Poland
Children's National Hospital
🇺🇸
Washington, District of Columbia, United States