Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency

Lumos Pharma

Status and phase

Enrolling

Phase 3

Conditions

Growth Hormone Deficiency (GHD)

Treatments

Drug: LUM-201

Other: Matched Placebo (Capsules)

Study type

Interventional

Funder types

Industry

Identifiers

NCT06948214

LUM-201-10

Details and patient eligibility

About

The OraGrowtH Phase 3 Trial 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 the LUM-201 predictive enrichment marker (LUM-201 PEM) strategy to select subjects likely to respond to therapy with daily oral LUM-201.

Enrollment

150 estimated patients

Sex

All

Ages

3 to 11 years old

Volunteers

No Healthy Volunteers

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