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

Lumos Pharma18 enrolled

Overview

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

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Growth Hormone Deficiency

Interventions

LUM-201DRUG

Administered orally once daily

Eligibility

Sex: ALLMin age: 3 YearsMax age: 13 Years

Medical Language ↔ Plain English

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

Locations (10)

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

Outcomes

Primary Outcomes

Annualized height velocity (AHV) measured as standing height with stadiometer

Annualized Height Velocity (AHV) measured as standing height with stadiometer

Time frame: Day 1 to Month 12

Secondary Outcomes

Incidence of adverse events in children with GHD

Number of events

Time frame: Day 1 to Month 12

GH Concentration on maintenance treatment

Serum GH concentration

Time frame: Day 1 to Month 12

Insulin-like growth factor 1

Serum concentrations of insulin-like growth factor 1

Time frame: Day 1 to Month 12

Insulin-like growth factor binding protein 3

Serum concentrations of insulin-like growth factor binding protein 3

Time frame: Day 1 to Month 12

Height standard deviation score (SDS)

Change in HT-SDS

Time frame: Day 1 to Month 12

Change in Weight

Time frame: Day 1 to Month 12

Change in Weight-SDS

Time frame: Day 1 to Month 12

Change in BMI

Time frame: Day 1 to Month 12

Data from ClinicalTrials.gov

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