A Long-Term Safety Trial of LUM-201 in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed a LUM-201 Clinical Trial (OraGrowtH211)

Phase 2Enrolling By InvitationNCT05796440

Lumos Pharma60 enrolled

Overview

This is a multi-national trial. The trial aims to study the long-term safety of LUM-201 in subjects with Idiopathic Pediatric Growth Hormone Deficiency (iPGHD). This study will also assess pharmacodynamics and efficacy response to therapy with LUM-201.

An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children with Idiopathic Growth Hormone Deficiency. This study will last for up to 36 months to allow collection of additional long-term safety and efficacy data related to LUM-201 treatment in the pediatric patient population. Subjects will enter into this trial after successful participation in a prior LUM-201 study. This study will consist of visits every 6 months through three years. There are a total of 7 in-person visits with a follow-up phone call between visits. At the clinic visits, subjects will have a physical exam and blood collection as well as efficacy assessments.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Growth Hormone Deficiency

Interventions

LUM-201DRUG

Administered orally once daily

Eligibility

Sex: ALLMin age: 5 YearsMax age: 14 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 successfully participated in a pediatric LUM-201 GHD study through at least the 12-month visit, and be eligible for continuation of treatment, pending all other enrollment criteria are met. Exclusion Criteria: * Medical or genetic condition that, in the opinion of the PI and/or MMs, adds unwarranted risk to the use of LUM-201 * 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. Subjects receiving shorter-term (two weeks or less) treatment with these medications should be evaluated on case-by-case basis by the PI in consultation with the MMs.

Locations (7)

University of Iowa

Iowa City, Iowa, United States

UMass Memorial Medical Center

Worcester, Massachusetts, United States

Children's Mercy Hospital

Kansas City, Missouri, United States

University of South Carolina

Charleston, South Carolina, United States

Outcomes

Primary Outcomes

Incidence of treatment-emergent adverse events

Number of events

Time frame: Day 1 to Month 36

Secondary Outcomes

Growth Hormone Concentrations in subjects

Serum GH concentration

Time frame: Day 1 to Month 36

Insulin-like growth factor 1 concentration

Serum concentrations of insulin-like growth factor 1

Time frame: Day 1 to Month 36

Height standard deviation score (SDS)

Change in HT-SDS

Time frame: Day 1 to Month 36

Change in Weight SDS

Change in Weight

Time frame: Day 1 to Month 36

Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201

(AHV) measured as standing height with stadiometer

Time frame: Day 1 to Month 36

Change in Bone Age (BA)

Change in Bone Age (BA) compared to Chronological Age

Time frame: Day 1 to Month 36

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.