Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
Phase 3
Not yet recruiting
- Conditions
- Growth Hormone Deficiency (GHD)
- Interventions
- Other: Matched Placebo (Capsules)
- Registration Number
- NCT06948214
- Lead Sponsor
- Lumos Pharma
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 150
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description LUM-201 LUM-201 - Placebo Matched Placebo (Capsules) -
- Primary Outcome Measures
Name Time Method AHV after 12 months on LUM-201 compared to placebo Day 1 to Month 12 Annualized height velocity (AHV) measured as standing height with stadiometer
- Secondary Outcome Measures
Name Time Method