PK and PD Study of LUM-201 in Children With Growth Hormone Deficiency. (OraGrowtH212)

  • STATUS
    Recruiting
  • End date
    Jul 23, 2023
  • participants needed
    24
  • sponsor
    Lumos Pharma
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Updated on 23 March 2022
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Summary

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 idiopathic pediatric growth hormone deficiency (PGHD).

Description

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 12 months of treatment. After screening, subjects will return to the clinic monthly for the first 6 visits and every three months thereafter. 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.

Details
Condition Growth Hormone Deficiency
Treatment LUM-201
Clinical Study IdentifierNCT04806854
SponsorLumos Pharma
Last Modified on23 March 2022

Eligibility

 Yes No Not Sure

Inclusion Criteria

Have a chronological age ≥ 4.0 years and ≤ 10.0 years for girls and ≤ 12.0 years for boys
Have a minimum body weight of 16 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 or the combination of a HT-SDS 2 SD below mean parental HT-SDS and a 6-month height velocity < 25th percentile for age and gender
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 < 7.0 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
At birth, gestational age < 36.0 weeks
Participation in any therapeutic trial of investigational drug(s) within the prior 6 months
History of spinal, cranial or total body irradiation
Recent commencement of non-stimulant therapy to treat attention deficit hyperactivity disorder (ADHD) or use of methylphenidate to treat ADHD
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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