Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213)

Growth Hormone Deficiency Clinical Trial

— OraGrowtH213  

Official title:

A Multicenter, Open-Label, Phase 2 Study to Evaluate Growth and Safety of LUM-201 Following 12 Months of Daily rhGH Treatment in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed the LUM-201-01 Trial

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05250063
• Other study ID #: LUM-201-04
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: February 18, 2022
• Est. completion date: February 2025

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 goals of the trial are to study the growth response to LUM-201 administration in children with idiopathic growth hormone deficiency (GHD) previously treated with daily rhGH for 12 months in the LUM-201-01 trial.

Description:

This trial will have one screening/baseline visit with tests to assess if subjects are eligible to start study therapy. The screening/baseline visit can coincide with the Month 12 visit in the LUM-201-01 trial. Subjects will receive the same dose of LUM-201 as a daily oral dose each morning. LUM-201 will be adjusted for body weight increments at each clinic visit, as applicable. The trial consists of 12 months of treatment. After screening, subjects will return to the clinic for 5 visits. During four of these clinic visits, safety laboratory samples will be collected.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 18
• Est. completion date: February 2025
• Est. primary completion date: February 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 13 Years

Eligibility

Inclusion Criteria:

• Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable.
• Must have previously completed 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial.
• Is eligible for study participation as confirmed by the principal investigator (PI)

Exclusion Criteria:

• Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201.
• Uses any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors.
• 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

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Growth Hormone Deficiency

Intervention

Drug:
• LUM-201
Administered orally once daily

Locations

Country	Name	City	State
Poland	Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki	Lodz
Poland	Sonomed - Centrum Medyczne	Szczecin
Poland	Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka	Warsaw
United States	University of Virginia Health System	Charlottesville	Virginia
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Indiana University School of Medicine	Indianapolis	Indiana
United States	The Children's Mercy Hospital	Kansas City	Missouri
United States	M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic	Minneapolis	Minnesota
United States	Rady Children's Hospital	San Diego	California
United States	Children's National Hospital	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Lumos Pharma

Countries where clinical trial is conducted

United States,  Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annualized height velocity (AHV) measured as standing height with stadiometer	Annualized Height Velocity (AHV) measured as standing height with stadiometer	Day 1 to Month 12
Secondary	Incidence of adverse events in children with GHD	Number of events	Day 1 to Month 12
Secondary	GH Concentration on maintenance treatment	Serum GH concentration	Day 1 to Month 12
Secondary	Insulin-like growth factor 1	Serum concentrations of insulin-like growth factor 1	Day 1 to Month 12
Secondary	Insulin-like growth factor binding protein 3	Serum concentrations of insulin-like growth factor binding protein 3	Day 1 to Month 12
Secondary	Height standard deviation score (SDS)	Change in HT-SDS	Day 1 to Month 12
Secondary	Change in Weight	Change in Weight	Day 1 to Month 12
Secondary	Change in Weight-SDS	Change in Weight-SDS	Day 1 to Month 12
Secondary	Change in BMI	Change in BMI	Day 1 to Month 12
Secondary	Change in BMI SDS	Change in BMI SDS	Day 1 to Month 12
Secondary	Change in Bone Age	Change in bone age, measured by X-ray of left hand and wrist using Greulich & Pyle atlas	Month 6
Secondary	Insulin-like growth factor 1 standard deviation score (SDS)	Change in IGF-1 SDS	Day 1 to Month 12