Clinical trial: PK and PD Study of LUM-201 in Children With Growth Hormone Deficiency (NCT04806854)

Eligibility 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.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 4 Years old.
Subject must be at most 9 Years

Patient description (medical language): A 9-year-old girl is brought to the office for evaluation of short stature and overweight body habitus. The patient's mother and father are 170 cm and 181 cm tall, respectively.  On physical examination, the patient's height is in the 5th percentile of her age.  Other findings include low-set ears, a high arched palate, a webbed neck, and cubitus valgus.  Chromosomal analysis reveals a 45, XO karyotype.

Patient description (patient's own words): I took my 9-year-old daughter to the doctor because my mother-in-law kept saying she seemed small and overweight. My husband and I are 170 cm and 181 cm tall, respectively. The physical health highlighted that she is in the 5th percentile of her age. The doctor said that she has low-set ears, a high-arched palate, a webbed neck, and cubitus valgus. She also did a chromosomal analysis, which revealed a 45, XO karyotype.

Does the patient match the trial criteria? Contradiction