Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial)
Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial)
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.
This trial will have two screening visits over about a month with tests to assess if subjects
 are eligible to start study therapy. Once subjects have completed screening, and if they are
 determined to be eligible, they will be randomized to receive one of three oral daily doses
 of LUM-201 or daily injections of recombinant human growth hormone (rhGH). All subjects will
 have an equal chance of being placed in any of the four groups.

 The trial consists of 6 months of treatment. After screening, subjects will return to clinic
 for 5 visits. At each clinic visit subjects will have a physical exam, blood and urine
 collections. There will also be 3 phone calls with study staff that will take place between
 the clinic visits.
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 ≥ 8 mcg/dL or stimulated cortisol ≥ 16 µg/dL.

 -  At Screening, be ≥ 3.0 years and < 10.0 years for girls and < 11.0 years for boys.

 -  Have HT-SDS < -2.0.

 -  Have a bone age < 9.0 years for girls and < 10.0 years for boys.

 -  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.

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