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

Launched by LUMOS PHARMA · Apr 21, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called LUM-201 for children with Growth Hormone Deficiency (GHD), which means their bodies don't produce enough growth hormone to help them grow normally. The trial aims to see how effective LUM-201 is for children who have not received any treatment before and to find ways to identify which children are most likely to benefit from this therapy.

To be eligible for the trial, children must be between 3 and 10 years old for girls and up to 11 years old for boys, be prepubertal, and show specific signs of GHD, such as a low growth hormone response in tests and being significantly shorter than their peers. They also need to have normal thyroid function and must not have certain medical conditions that could affect their growth. Participants can expect to be part of a study that will help researchers understand how well LUM-201 works and help improve treatment for kids with this condition in the future. The trial is not yet recruiting participants, so interested families will need to wait for the study to begin.

Gender

ALL

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