Clinical trial: Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial) (NCT04614337)

Eligibility 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 ≥ 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.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 3 Years old.
Subject must be at most 10 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? 0