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) (OraGrowtH211)

February 14, 2024 updated by: Lumos Pharma

An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children With Idiopathic Growth Hormone Deficiency

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.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

60

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Australian Capital Territory
      • Garran, Australian Capital Territory, Australia, 2605
        • Canberra Hospital
  • United States
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Massachusetts
      • Worcester, Massachusetts, United States, 01655
        • UMass Memorial Medical Center
    • Missouri
      • Kansas City, Missouri, United States, 64111
        • Children's Mercy Hospital
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • University of South Carolina
    • Texas
      • Amarillo, Texas, United States, 79106
        • Texas Tech University Health Sciences Center
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 14 years (Child)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: LUM-201 (1.6 mg/kg/day)
Drug: LUM-201
Administered orally once daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of treatment-emergent adverse events
Time Frame: Day 1 to Month 36
Number of events
Day 1 to Month 36

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Lumos Pharma

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 1, 2023

Primary Completion (Estimated)

March 1, 2028

Study Completion (Estimated)

April 1, 2028

Study Registration Dates

First Submitted

March 19, 2023

First Submitted That Met QC Criteria

March 30, 2023

First Posted (Actual)

April 3, 2023

Study Record Updates

Last Update Posted (Actual)

February 15, 2024

Last Update Submitted That Met QC Criteria

February 14, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • LUM-201-02

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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