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

  • STATUS
    Recruiting
  • End date
    Mar 13, 2024
  • participants needed
    80
  • sponsor
    Lumos Pharma
Updated on 13 November 2021

Summary

This is a multi-national trial. The goals of the trial are to study LUM-201 as a possible treatment for Pediatric Growth Hormone Deficiency (PGHD) and investigate a predictive enrichment marker (PEM) strategy to select subjects likely to respond to therapy with LUM-201.

Description

This trial will have one screening visit with tests 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 be randomized to receive one of three oral daily doses of LUM-201 or daily injections of recombinant human growth hormone (rhGH). All subjects will have an equal chance of being placed in any of the four groups.

The trial consists of 12 months of treatment. After screening, subjects will return to clinic for 8 visits. During seven of these clinic visits, subjects will have a physical exam, blood and urine collections. There will also be 3 phone calls with study staff that will take place between the clinic visits.

Details
Condition GH deficiency, Growth Hormone Deficiency
Treatment LUM-201, rhGH Norditropin® FlexPro® pen (34 µg/kg), rhGH Norditropin® pen (34 µg/kg)
Clinical Study IdentifierNCT04614337
SponsorLumos Pharma
Last Modified on13 November 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Have an established diagnosis of PGHD as determined by standard diagnostic criteria. Eligible subjects must be nave-to-treatment and be prepubertal
Morning cortisol 7 g/dL or stimulated cortisol 14 g/dL
At Screening, be 3.0 years and 11.0 years for girls and 12.0 years for boys
Have HT-SDS -2.0 or the combination of a HT-SDS 2 SD below mean parental HT-SDS and a 6-month height minus velocity < 25th percentile for age and gender
Have a bone age delayed by 6 months with respect to chronological age for subjects 9.0 years old and by at least 1.0 year for subjects older than 9.0 years of age
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
Treatment with methylphenidate or medications known to act as moderate or strong inhibitors or strong inducers of CYP3A/4, or with medications known to act as strong inhibitors of P-glycoprotein (P-gp) or potent substrates of P-gp or Multidrug and toxin extrusion protein 1 (MATE1)
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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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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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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