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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 : October 7, 2022
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 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 up to 24 months of treatment. After screening, subjects will return to the clinic for 6 (subjects placed in rhGH group) or 10 visits (subjects placed in LUM-201 group). During several 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.

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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, 24-Month, Randomized, Open-Label, Active Control, Parallel Arm, Phase 2 Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children With Idiopathic Growth Hormone Deficiency (GHD)
Actual Study Start Date : December 31, 2020
Estimated Primary Completion Date : September 2023
Estimated Study Completion Date : April 2025


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

  2. AHV after 6 months on LUM-201 compared to rhGH [ Time Frame: Day 1 to Month 6 ]
    Annualized height velocity to be measured


Secondary Outcome Measures :
  1. 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

  2. Incidence of adverse events in children with GHD [ Time Frame: Day 1 to Month 24 ]
    Number of events

  3. Height standard deviation score (SDS) [ Time Frame: Day 1 to Month 6 and Month 12 ]
    Change in HT-SDS

  4. Height velocity standard deviation score (HV-SDS) [ Time Frame: Day 1 to Month 6, and Month 12 ]
    Change in HV-SDS

  5. Change in Weight [ Time Frame: Day 1 to Month 6, and Month 12 ]
    Change in Weight

  6. Change in Weight SDS [ Time Frame: Day 1 to Month 6 and Month 12 ]
    Change in Weight-SDS

  7. Change in BMI [ Time Frame: Day 1 to Month 6 and Month 12 ]
    Change in BMI

  8. Change in BMI SDS [ Time Frame: Day 1 to Month 6 and Month 12 ]
    Change in BMI SDS

  9. Bone Age [ Time Frame: Day 1 to Month 6 and Month 18 ]
    Change in bone age, measured by X-ray of left hand and wrist using Greulich & Pyle atlas

  10. Pharmacokinetics of LUM-201 [ Time Frame: Day 1 to Month 6 and 12 ]
    Serum concentrations (Cmax/Steady State)

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

  12. Insulin-like growth factor 1 SDS [ Time Frame: Day 1 to Month 6 and 12 ]
    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 idiopathic 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 HT-SDS ≥ 2 SD below mean parental HT-SDS.
  • Have a baseline height velocity < 5.5 cm/year based on at least 6 months of growth.
  • Have a bone age delayed by ≥ 6 months with respect to chronological 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, idiopathic short stature).
  • 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 < 3rdth 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 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).

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: October 7, 2022
Last Verified: October 2022
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