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

NCT04806854 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: LUM-201-03
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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 idiopathic pediatric growth hormone deficiency (PGHD).

Conditions

Growth Hormone Deficiency

Keywords

GHD, PGHD, LUM-201, growth hormone secretagogue, height, catch-up growth, pulsatility, pharmacokinetics, pharmacodynamics, PK, PD, idiopathic

Outcome Measures

Primary Outcomes (2)

• Evaluation of Augmented Growth Hormone (GH) Pulsatility

  12-hour mean GH concentration

  Day 1 to Month 6

• Pharmacokinetics of LUM-201 and M8

  Plasma concentrations (Cmax/Steady State)

  Day 1 to Month 6

Secondary Outcomes (2)

• Incidence of adverse events in children with idiopathic GHD

  up to 8 years

• Height standard deviation score (SDS)

  Day 1 to Month 12

Study Arms (2)

LUM-201 (1.6 mg/kg/day)

EXPERIMENTAL

Drug: LUM-201

LUM-201 (3.2 mg/kg/day)

EXPERIMENTAL

Drug: LUM-201

Interventions

LUM-201 DRUG

Administered orally once daily

LUM-201 (1.6 mg/kg/day); LUM-201 (3.2 mg/kg/day)

Eligibility Criteria

• Age: 4 Years - 9 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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 is delayed with respect to chronological age. Bone age should be based on the apparent BA of the proximal and distal phalanges, as opposed to the metacarpals, carpals and distal forearm. A central BA reader will make the final determination on BA eligibility.
• Have HT-SDS ≤ -2.0 or a HT-SDS ≥ 2 SD below mean parental HT-SDS. If the other data (slow HV, low IGF-1, peak GH \< 10, and 6-month BA delay) are all consistent with idiopathic GHD, the subject may be enrolled with a height SDS \> -2.0 after consultation with the MMs.
• Have an accurate baseline height velocity ≤ 5.5 cm/year based on at least 6-months of growth, if available
• Within the past 6 months have a maximal GH response \> 3 and \< 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 an arm span to height ratio \> 96.5%
• 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.

You may not qualify if:

• 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, , idiopathic short stature (ISS), SHOX-deficiency, skeletal dysplasia, constitutional growth delay, SGA, other).
• 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 or a diagnosis of Organic PGHD.
• 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).

Sponsors & Collaborators

• Lumos Pharma: lead

Study Sites (1)

Institute of Maternal and Child Research, University of Chile

Santiago, Chile

Location

MeSH Terms

Conditions

Dwarfism, Pituitary

Condition Hierarchy (Ancestors)

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; Endocrine System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 17, 2021
• First Posted: March 19, 2021
• Study Start: July 14, 2021
• Primary Completion (Estimated): April 1, 2030
• Study Completion (Estimated): July 1, 2030
• Last Updated: October 4, 2023

Record last verified: 2023-10

Data Sharing

• IPD Sharing: Will not share

Locations

CL (1)