Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial)

• Status: Recruiting
• Trial end date: 2024-03-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Lumos Pharma

Criteria

Inclusion Criteria:

• Have an established diagnosis of PGHD as determined by standard diagnostic criteria. Eligible subjects must be naïve-to-treatment and be prepubertal.

• Morning cortisol ≥ 7 µg/dL or stimulated cortisol ≥ 14 µg/dL.

• At Screening, be ≥ 3.0 years and ≤ 11.0 years for girls and ≤ 12.0 years for boys.

• Have HT-SDS ≤ -2.0 or the combination of a HT-SDS 2 SD below mean parental HT-SDS and a 6-month height minus velocity < 25th percentile for age and gender.

• Have a bone age delayed by ≥ 6 months with respect to chronological age for subjects ≤ 9.0 years old and by at least 1.0 year for subjects older than 9.0 years of age.

• Have prepubertal status as evidenced by Tanner Stage I breast development in girls and testicular volume < 4.0 mL in boys.

• 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 30 days prior to Day 1.

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. (Examples: diabetes, named syndromes).

• A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201 or rhGH.

• 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 (Example: glucocorticoids).

• Evidence or history of an intracranial mass (e.g., pituitary tumor, craniopharyngioma).

• Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3 ng/mL on two prior standard of care GH stimulation tests, or pituitary deficiencies beyond GH and thyroid function.

• Malnutrition as evidenced by medical history or a body weight < 5th percentile for current height.

• BMI > 95th percentile.

• Gestational age-adjusted birth weight < 5th percentile (small for gestational age).

• History of spinal, cranial, or total body irradiation.

• Treatment with methylphenidate or medications known to act as moderate or strong inhibitors or strong inducers of CYP3A/4, or with medications known to act as strong inhibitors of P-glycoprotein (P-gp) or potent substrates of P-gp or Multidrug and toxin extrusion protein 1 (MATE1).