PK and PD Study of LUM-201 in Children With Growth Hormone Deficiency

• Status: Recruiting
• Trial end date: 2022-11-01
• Target enrollment: 0
• Participant gender: All

Summary

The goals of this single site trial are to study the pharmacokinetics (PK) and pharmacodynamics of LUM-201 and effects of LUM-201 administration on growth hormone release over time in children with pediatric growth hormone deficiency (PGHD).

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

Details

• Lead Sponsor: Lumos Pharma

Criteria

Inclusion Criteria:

• Have a chronological age > 4.0 years and < 8.0 years for girls and < 10.0 years for boys.

• Have a minimum body weight of 17 kg at the time of screening.

• Have a bone age examination at screening or within the 6 months prior to screening that, in the interpretation of the PI, is delayed with respect to chronological age.

• Have a HT-SDS < -2.0

• Have a maximal GH response < 10 ng/mL from at least one prior GH stimulation test within the past 6 months

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

• Have height minus arm span difference < 4.5 cm.

• 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 the Baseline visit.

Exclusion Criteria:

• Any medical or genetic condition which, in the opinion of the PI or Medical Monitor (MM), can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment (e.g., diabetes).

• A medical or genetic condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201 (e.g., scoliosis).

• Use of any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors (e.g., glucocorticoids).

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

• Prior treatment with growth factors including, but not limited to, GH, IGF-1, and GH secretagogues. (These may be used for limited times as a diagnostic test.)

• Suspicion of absence of pituitary function as evidenced by a maximal stimulated GH ≤ 3 ng/mL or pituitary hormone deficiencies beyond GH and thyroid function.

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

• At birth, gestational age < 36.0 weeks.

• Participation in any trial of investigational drug(s) within the prior 6 months.

• History of spinal or total body irradiation.

• A diagnosis of Attention Deficit Hyperactivity Disorder (ADHD) and/or use of medication to treat ADHD.