Clinical trial NCT06948214: Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
Official 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)
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: Growth Hormone Deficiency (GHD)
Keywords: GHD
Pediatric Growth Hormone Deficiency
LUM-201
Growth hormone secretagogue
Height
Catch-up growth
PEM
Oral
Predictive Enrichment Marker
Lead sponsor: Lumos Pharma
Sponsor class: INDUSTRY
First posted: 2025-04-29
Enrollment: 150
Sex: ALL
Minimum age (years): 3
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a multi-national trial. The goals of the trial are to study LUM-201 as a treatment for Pediatric Growth Hormone Deficiency (PGHD) in naive to treatment children and validate a predictive enrichment marker (PEM) strategy to select subjects likely to respond to therapy with LUM-201.


Eligibility criteria: Inclusion Criteria:

* Subjects must be naïve to treatment and prepubertal
* Subjects must have a maximal GH response of \< 10 ng/mL from 2 prior GH stimulation tests conducted within the preceding 12 months
* Impaired height defined as ≥ 2.0 standard deviations (SDs) below the mean height for chronological age and sex
* Morning or random cortisol level of ≥ 7.0 μg/dL
* ≥ 3.0 years and age ≤ 10.0 years for girls and ≤ 11.0 years for boys
* Baseline height velocity (HV) based on ≥ 6 months of growth assessments \< 25th percentile for age and sex
* Bone Age delay of ≥ 12 months compared to the chronological age
* In girls, have genetic testing results to rule out Turner syndrome. If SHOX genetic testing results are available, they need to be negative.
* Have normal thyroid function. Subjects diagnosed with hypothyroidism must have documented successful treatment for at least 3 months prior to Day 1
* Baseline IGF-1 standard deviation score (SDS) ≤ -1.0

Exclusion Criteria:

* Any medical or genetic condition which, in the opinion of the Investigator or Medical Monitor (MM), can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment.
* Arm span to height ratio \> 2 SDs below the mean for age and sex
* A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201
* Use of any medication that, in the opinion of the Investigator and/or MM, can independently cause short stature or limit the response to exogenous growth factors
* Current inflammatory diseases requiring systemic corticosteroid treatment for \> 2 consecutive weeks within the last 3 months prior to the Screening Visit
* Use of hormone replacement therapy for any hormone deficiency other than thyroid deficiency
* Any ECG at the Screening Visit noted to have a clinically significant abnormality, as confirmed by the MM
* Any subjects suspected of having past or present intracranial tumor growth as confirmed by brain imaging prior to the Screening or Day 1 Visit
* Any subject suspected of having intracranial hypertension (IH) as confirmed by fundoscopy and other assessments
* Any subject with serum alanine transaminase (ALT), aspartate transaminase (AST), or total bilirubin \> upper limit of normal (ULN)
* Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3.0 ng/mL on any prior standard of care GH stimulation test completed within 12 months
* Body weight ≤ 14.0 kg
* BMI \< -2 or \> +2 SDs for age and sex based on WHO standards
* Birth weight for gestational age \< 3rd percentile based on WHO standards
* Treatment with medications known to be moderate or strong inhibitors or strong inducers of cytochrome P450 (CYP) 3A/4
* History of spinal, cranial, or total body irradiation
* Attention deficit hyperactivity disorder (ADHD) diagnosis

Primary outcomes:
measure: AHV after 12 months on LUM-201 compared to placebo
timeFrame: Day 1 to Month 12
Secondary outcomes:
Other outcomes:
measure: Percentage of subjects selected by PEM strategy who meet target growth
timeFrame: Day 1 to Month 12
Locations:
Study officials:
References: