phase 3 study of lum-201 in children with growth hormone deficiency

Conditions: Growth Hormone Deficiency (GHD)

Summary: this is a multi-national trial. the goals of the trial are to study lum-201 as a treatment for pediatric growth hormone deficiency (pghd) in naive to treatment children and validate a predictive enrichment marker (pem) strategy to select subjects likely to respond to therapy with lum-201.


Eligibility criteria:
inclusion criteria: * subjects must be naïve to treatment and prepubertal * subjects must have a maximal gh response of \< 10 ng/ml from 2 prior gh stimulation tests conducted within the preceding 12 months * impaired height defined as ≥ 2.0 standard deviations (sds) below the mean height for chronological age and sex * morning or random cortisol level of ≥ 7.0 μg/dl * ≥ 3.0 years and age ≤ 10.0 years for girls and ≤ 11.0 years for boys * baseline height velocity (hv) based on ≥ 6 months of growth assessments \< 25th percentile for age and sex * bone age delay of ≥ 12 months compared to the chronological age * 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 3 months prior to day 1 * baseline igf-1 standard deviation score (sds) ≤ -1.0 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. * arm span to height ratio \> 2 sds below the mean for age and sex * a medical or genetic condition that, in the opinion of the investigator and/or mm, adds unwarranted risk to use of lum-201 * 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 * current inflammatory diseases requiring systemic corticosteroid treatment for \> 2 consecutive weeks within the last 3 months prior to the screening visit * use of hormone replacement therapy for any hormone deficiency other than thyroid deficiency * any ecg at the screening visit noted to have a clinically significant abnormality, as confirmed by the mm * any subjects suspected of having past or present intracranial tumor growth as confirmed by brain imaging prior to the screening or day 1 visit * any subject suspected of having intracranial hypertension (ih) as confirmed by fundoscopy and other assessments * any subject with serum alanine transaminase (alt), aspartate transaminase (ast), or total bilirubin \> upper limit of normal (uln) * suspicion of absent pituitary function as evidenced by a maximal stimulated gh ≤ 3.0 ng/ml on any prior standard of care gh stimulation test completed within 12 months * body weight ≤ 14.0 kg * bmi \ +2 sds for age and sex based on who standards * birth weight for gestational age \< 3rd percentile based on who standards * treatment with medications known to be moderate or strong inhibitors or strong inducers of cytochrome p450 (cyp) 3a/4 * history of spinal, cranial, or total body irradiation * attention deficit hyperactivity disorder (adhd) diagnosis

Keywords: ghd pediatric growth hormone deficiency lum-201 growth hormone secretagogue height catch-up growth pem oral predictive enrichment marker