Clinical trial NCT05250063: Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Growth Hormone Deficiency
Keywords: LUM-201
PGHD
Height
Oral
Lead sponsor: Lumos Pharma
Sponsor class: INDUSTRY
First posted: 2022-02-22
Enrollment: 18
Sex: ALL
Minimum age (years): 3
Maximum age (years): 13
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Annualized height velocity (AHV) measured as standing height with stadiometer
timeFrame: Day 1 to Month 12
Secondary outcomes:
measure: Incidence of adverse events in children with GHD
timeFrame: Day 1 to Month 12
measure: GH Concentration on maintenance treatment
timeFrame: Day 1 to Month 12
measure: Insulin-like growth factor 1
timeFrame: Day 1 to Month 12
measure: Insulin-like growth factor binding protein 3
timeFrame: Day 1 to Month 12
measure: Height standard deviation score (SDS)
timeFrame: Day 1 to Month 12
measure: Change in Weight
timeFrame: Day 1 to Month 12
measure: Change in Weight-SDS
timeFrame: Day 1 to Month 12
measure: Change in BMI
timeFrame: Day 1 to Month 12
measure: Change in BMI SDS
timeFrame: Day 1 to Month 12
measure: Change in Bone Age
timeFrame: Month 6
measure: Insulin-like growth factor 1 standard deviation score (SDS)
timeFrame: Day 1 to Month 12
Other outcomes:
Locations:
city: San Diego
country: United States
facility: Rady Children's Hospital
state: California
city: Washington
country: United States
facility: Children's National Hospital
state: District of Columbia
city: Indianapolis
country: United States
facility: Indiana University School of Medicine
state: Indiana
city: Minneapolis
country: United States
facility: M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic
state: Minnesota
city: Kansas City
country: United States
facility: The Children's Mercy Hospital
state: Missouri
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Charlottesville
country: United States
facility: University of Virginia Health System
state: Virginia
city: Lodz
country: Poland
facility: Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki
city: Szczecin
country: Poland
facility: Sonomed - Centrum Medyczne
city: Warsaw
country: Poland
facility: Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka
Study officials:
References: