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)

Growth Hormone Deficiency Clinical Trial

— OraGrowtH211  

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05796440
• Other study ID #: LUM-201-02
• Status: Enrolling by invitation
• Phase: Phase 2
• Start date: August 1, 2023
• Est. completion date: April 2028

Study information

• Verified date: February 2024
• Source: Lumos Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 60
• Est. completion date: April 2028
• Est. primary completion date: March 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 14 Years

Eligibility

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.

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Hormone Deficiency

Intervention

Drug:
• LUM-201
Administered orally once daily

Locations

Country	Name	City	State
Australia	Canberra Hospital	Garran	Australian Capital Territory
United States	Texas Tech University Health Sciences Center	Amarillo	Texas
United States	University of South Carolina	Charleston	South Carolina
United States	University of Iowa	Iowa City	Iowa
United States	Children's Mercy Hospital	Kansas City	Missouri
United States	Seattle Children's Hospital	Seattle	Washington
United States	UMass Memorial Medical Center	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Lumos Pharma

Countries where clinical trial is conducted

United States,  Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-emergent adverse events	Number of events	Day 1 to Month 36
Secondary	Growth Hormone Concentrations in subjects	Serum GH concentration	Day 1 to Month 36
Secondary	Insulin-like growth factor 1 concentration	Serum concentrations of insulin-like growth factor 1	Day 1 to Month 36
Secondary	Height standard deviation score (SDS)	Change in HT-SDS	Day 1 to Month 36
Secondary	Change in Weight SDS	Change in Weight	Day 1 to Month 36
Secondary	Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201	(AHV) measured as standing height with stadiometer	Day 1 to Month 36
Secondary	Change in Bone Age (BA)	Change in Bone Age (BA) compared to Chronological Age	Day 1 to Month 36