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Trial record 1 of 1 for:    NCT04806854
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PK and PD Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH212)

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ClinicalTrials.gov Identifier: NCT04806854
Recruitment Status : Not yet recruiting
First Posted : March 19, 2021
Last Update Posted : May 24, 2021
Sponsor:
Information provided by (Responsible Party):
Lumos Pharma

Brief 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 pediatric growth hormone deficiency (PGHD).

Condition or disease Intervention/treatment Phase
Growth Hormone Deficiency Drug: LUM-201 Phase 2

Detailed 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 6 months of treatment. After screening, subjects will return to the clinic monthly for visits. 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single-Center, 6-Month, Randomized, Open-Label, Parallel Arm Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children With Pediatric Growth Hormone Deficiency (PGHD)
Estimated Study Start Date : June 2021
Estimated Primary Completion Date : October 2022
Estimated Study Completion Date : November 2022


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. Evaluation of Augmented Growth Hormone (GH) Pulsatility [ Time Frame: Day 1 to Month 6 ]
    12-hour mean GH concentration

  2. Pharmacokinetics of LUM-201 and M8 [ Time Frame: Day 1 to Month 6 ]
    Plasma concentrations (Cmax/Steady State)


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

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



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 9 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Have a chronological age > 4.0 years and < 8.0 years for girls and < 10.0 years for boys.
  • Have a minimum body weight of 17 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
  • 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 < 4.5 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.
  • Gestational age-adjusted birth weight < 5th percentile (small for gestational age).
  • At birth, gestational age < 36.0 weeks.
  • Participation in any trial of investigational drug(s) within the prior 6 months.
  • History of spinal or total body irradiation.
  • A diagnosis of Attention Deficit Hyperactivity Disorder (ADHD) and/or use of medication to treat ADHD.

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): NCT04806854


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

Locations
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Chile
Institute of Maternal and Child Research, University of Chile
Santiago, Chile
Contact: Fernando Cassorla, MD    56 22 977 0850    fcassorl@med.uchile.cl   
Sponsors and Collaborators
Lumos Pharma
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Responsible Party: Lumos Pharma
ClinicalTrials.gov Identifier: NCT04806854    
Other Study ID Numbers: LUM-201-03
First Posted: March 19, 2021    Key Record Dates
Last Update Posted: May 24, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Lumos Pharma:
GHD, PGHD, LUM-201, growth hormone secretagogue, height, catch-up growth, pulsatility, pharmacokinetics, pharmacodynamics, PK, PD
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