Phase 3 Efficacy and Safety Study of Daily LUM-201 in Children with (GHD) (OraGrowth)
Research type
Research Study
Full title
A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children with Growth Hormone Deficiency (GHD)
IRAS ID
1011916
Contact name
Susan Thornton
Contact email
Sponsor organisation
Lumos Pharma LLC
Clinicaltrials.gov Identifier
Research summary
The study is to see if a test medication not yet approved for market, named LUM-201, will help in the treatment of paediatric Growth Hormone Deficiency (GHD) and how safe it is to use in children.
The investigational medication LUM-201 is mini tablets inside a capsule for oral use taken daily.
Participant will have a 2:1 chance of receiving the LUM-201 vs placebo, referred to as ‘study medication’.
There will be a Predictive Enrichment Marker (PEM) test, which is a blood test to see if participants are eligible to enter the treatment period. It will be made by taking a blood sample before and approximately one hour after receiving a single oral administration of the study medication.
Participant may receive placebo which looks like the LUM-201 but contains no medicine and is not expected to have any effect to allow the Sponsor to see if LUM-201 works better than the placebo.
In this study neither the participant or your study doctor will know what treatment is being received.
Participation in the study will last approximately 15 months, including Screening (up to 60 days = 2 months), treatment with the study medication (12 months), and follow up by your study doctor (1 month) who will continue to monitor participant after they complete the treatment if participant choose to not enter the LUM-201 three-year extension trial.
Participant will complete the 12-month visit they may be eligible to join an extension study. In the extension study all participants will receive LUM-201 study medication for up to three years.
The study is planned to involve approximately 170 children who have paediatric GHD.REC name
East of Scotland Research Ethics Service REC 2
REC reference
25/ES/0098
Date of REC Opinion
23 Dec 2025
REC opinion
Further Information Favourable Opinion