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

Lumos Pharma

Status and phase: Active, not recruiting; Phase 2

Conditions: Growth Hormone Deficiency

Treatments: Drug: LUM-201

Study type: Interventional

Funder types: Industry

Identifiers

NCT05250063

LUM-201-04

Details and patient eligibility

About

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.

Full 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.

Enrollment: 18 patients

Sex: All

Ages: 3 to 13 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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18 participants in 1 patient group

LUM-201 (3.2 mg/kg/day)

Experimental group

Treatment:

Drug: LUM-201