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 2

• Conditions: Growth Hormone Deficiency
• Interventions: Drug: LUM-201

• Registration Number: NCT05796440
• Lead Sponsor: Lumos Pharma

Brief Summary

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.

Detailed Description

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 Timeline

• Study First Submitted: 2023-03-19
• Study First Submitted QC: 2023-03-30
• Study First Posted: 2023-04-03
• Study Start: 2023-08-01
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-14
• Last Update Posted: 2024-02-15
• Primary Completion: 2028-03
• Study Completion: 2028-04

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 60

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
LUM-201 (1.6 mg/kg/day)	LUM-201	-

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events	Day 1 to Month 36	Number of events

Secondary Outcome Measures

Name	Time	Method
Change in Weight SDS	Day 1 to Month 36	Change in Weight
Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201	Day 1 to Month 36	(AHV) measured as standing height with stadiometer
Change in Bone Age (BA)	Day 1 to Month 36	Change in Bone Age (BA) compared to Chronological Age
Growth Hormone Concentrations in subjects	Day 1 to Month 36	Serum GH concentration
Insulin-like growth factor 1 concentration	Day 1 to Month 36	Serum concentrations of insulin-like growth factor 1
Height standard deviation score (SDS)	Day 1 to Month 36	Change in HT-SDS

Trial Locations

Locations (7)

Canberra Hospital; 🇦🇺 Garran, Australian Capital Territory, Australia

University of Iowa; 🇺🇸 Iowa City, Iowa, United States

Seattle Children's Hospital; 🇺🇸 Seattle, Washington, United States

UMass Memorial Medical Center; 🇺🇸 Worcester, Massachusetts, United States

University of South Carolina; 🇺🇸 Charleston, South Carolina, United States

Children's Mercy Hospital; 🇺🇸 Kansas City, Missouri, United States

Texas Tech University Health Sciences Center; 🇺🇸 Amarillo, Texas, United States