Back to resultsA 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)
Primary Purpose
Growth Hormone Deficiency
Status
Enrolling by invitation
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
LUM-201
Sponsored by
About this trial
This is an interventional treatment trial for Growth Hormone Deficiency focused on measuring LUM-201, PGHD, GHD, Growth hormone secretagogue, Oral, Height
Eligibility Criteria
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.
Sites / Locations
- University of Iowa
- University of South Carolina
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
LUM-201 (1.6 mg/kg/day)
Arm Description
Outcomes
Primary Outcome Measures
Incidence of treatment-emergent adverse events
Number of events
Secondary Outcome Measures
Growth Hormone Concentrations in subjects
Serum GH concentration
Insulin-like growth factor 1 concentration
Serum concentrations of insulin-like growth factor 1
Height standard deviation score (SDS)
Change in HT-SDS
Change in Weight SDS
Change in Weight
Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201
(AHV) measured as standing height with stadiometer
Change in Bone Age (BA)
Change in Bone Age (BA) compared to Chronological Age
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05796440
Brief Title
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)
Acronym
OraGrowtH211
Official Title
An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children With Idiopathic Growth Hormone Deficiency
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
August 1, 2023 (Actual)
Primary Completion Date
March 2028 (Anticipated)
Study Completion Date
November 2028 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Lumos Pharma
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Growth Hormone Deficiency
Keywords
LUM-201, PGHD, GHD, Growth hormone secretagogue, Oral, Height
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Open Label
Masking
None (Open Label)
Allocation
N/A
Enrollment
60 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
LUM-201 (1.6 mg/kg/day)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
LUM-201
Intervention Description
Administered orally once daily
Primary Outcome Measure Information:
Title
Incidence of treatment-emergent adverse events
Description
Number of events
Time Frame
Day 1 to Month 36
Secondary Outcome Measure Information:
Title
Growth Hormone Concentrations in subjects
Description
Serum GH concentration
Time Frame
Day 1 to Month 36
Title
Insulin-like growth factor 1 concentration
Description
Serum concentrations of insulin-like growth factor 1
Time Frame
Day 1 to Month 36
Title
Height standard deviation score (SDS)
Description
Change in HT-SDS
Time Frame
Day 1 to Month 36
Title
Change in Weight SDS
Description
Change in Weight
Time Frame
Day 1 to Month 36
Title
Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201
Description
(AHV) measured as standing height with stadiometer
Time Frame
Day 1 to Month 36
Title
Change in Bone Age (BA)
Description
Change in Bone Age (BA) compared to Chronological Age
Time Frame
Day 1 to Month 36
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
12. IPD Sharing Statement
Learn more about this trial
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)
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