PK and PD Study of LUM-201 in Children With Growth Hormone Deficiency
PK and PD Study of LUM-201 in Children With Growth Hormone Deficiency
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).
This trial will have a single screening visit 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 return to the clinic for a 12-hour pulsatility study. On Day Two of the
 trial, subjects will be randomized to receive one of two oral daily doses of LUM-201 and PK
 samples will be collected. All subjects will have an equal chance of being placed in either
 of the two groups.

 The trial consists of 6 months of treatment. After screening, subjects will return to the
 clinic monthly for visits. At most clinic visits, subjects will have a physical exam, blood
 and urine collections. At the Month 6 visit subjects will repeat the 12-hour pulsatility and
 PK studies.
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.