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

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ClinicalTrials.gov Identifier: NCT04614337
Recruitment Status : Recruiting
First Posted : November 4, 2020
Last Update Posted : August 4, 2021
Sponsor:
Information provided by (Responsible Party):
Lumos Pharma

Brief 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.

Condition or disease Intervention/treatment Phase
Growth Hormone Deficiency Drug: LUM-201 Drug: rhGH Norditropin® pen (34 µg/kg) Phase 2

Detailed Description:

This trial will have two screening visits over about a month 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 6 months of treatment. After screening, subjects will return to clinic for 5 visits. At each clinic visit 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, 6-Month, Randomized, Open-Label, Active Control, Parallel Arm, Phase 2b Study of Daily Oral LUM-201 in Naïve to Treatment, Prepubertal Children With Growth Hormone Deficiency (GHD)
Actual Study Start Date : December 31, 2020
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : March 2022


Arm Intervention/treatment
Experimental: LUM-201 (0.8 mg/kg/day) Drug: LUM-201
Administered orally once daily

Experimental: LUM-201 (1.6 mg/kg/day) Drug: LUM-201
Administered orally once daily

Experimental: LUM-201 (3.2 mg/kg/day) Drug: LUM-201
Administered orally once daily

Active Comparator: rhGH (34 µg/kg/day) Drug: rhGH Norditropin® pen (34 µg/kg)
Administered subcutaneously (s.c., under the skin) once daily.




Primary Outcome Measures :
  1. Percentage of subjects selected by PEM strategy who meet target growth [ Time Frame: Day 1 to Month 6 ]
    Annualized height velocity (AHV) measured as standing height with stadiometer


Secondary Outcome Measures :
  1. Comparison of 6-month, AHVs achieved by daily rhGH and three dose levels of LUM-201 [ Time Frame: Day 1 to Month 6 ]
    6-month Annualized Height Velocity (AHV) measured as standing height with stadiometer

  2. Degree of concordance between the first and second assessment with the PEM strategy. [ Time Frame: Screening to Day 1 ]
    Peak serum concentration of GH in response to a single provocative dose of LUM-201

  3. Incidence of adverse events in children with GHD [ Time Frame: Day 1 to Month 6 ]
    Number of events

  4. Height standard deviation score (SDS) [ Time Frame: Day 1 to Month 6 ]
    Change in HT-SDS from Baseline to Month 6

  5. Height velocity standard deviation score (HV-SDS) [ Time Frame: Day 1 to Month 6 ]
    Change in HV-SDS from Baseline to Month 6

  6. Change in Weight [ Time Frame: Day 1 to Month 6 ]
    Change in Weight from Baseline to Month 6

  7. Change in Weight SDS [ Time Frame: Day 1 to Month 6 ]
    Change in Weight-SDS from Baseline to Month 6

  8. Change in BMI [ Time Frame: Day 1 to Month 6 ]
    Change in BMI from Baseline to Month 6

  9. Change in BMI SDS [ Time Frame: Day 1 to Month 6 ]
    Change in BMI SDS from Baseline to Month 6

  10. Bone Age [ Time Frame: Day 1 to Month 6 ]
    Change in bone age, measured by X-ray of left hand and wrist using Greulich & Pyle atlas

  11. Pharmacokinetics of LUM-201 [ Time Frame: Day 1 to Month 6 ]
    Serum concentrations (Cmax/Steady State)

  12. GH Concentration on maintenance treatment [ Time Frame: Day 1 to Month 6 ]
    Serum GH concentration

  13. Insulin-like growth factor 1 [ Time Frame: Day 1 to Month 6 ]
    Serum concentrations of insulin-like growth factor 1



Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 ≥ 7 µg/dL or stimulated cortisol ≥ 14 µg/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.
  • Recent commencement (within three months of the Screening Visit 1) of non-stimulant therapy to treat attention deficit hyperactivity disorder (ADHD).
  • Treatment with methylphenidate or 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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04614337


Contacts
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Contact: Lumos Pharma 515-598-2921 clinical.trials@lumos-pharma.com

Locations
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Sponsors and Collaborators
Lumos Pharma
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Responsible Party: Lumos Pharma
ClinicalTrials.gov Identifier: NCT04614337    
Other Study ID Numbers: LUM-201-01
First Posted: November 4, 2020    Key Record Dates
Last Update Posted: August 4, 2021
Last Verified: August 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Lumos Pharma:
GHD
PGHD
LUM-201
Growth hormone secretagogue
Height
Catch-up growth
PEM
Oral
Predictive Enrichment Marker
Additional relevant MeSH terms:
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Dwarfism, Pituitary
Endocrine System Diseases
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases