RECRUITING

Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency

Conditions

Growth Hormone Deficiency (GHD)GHD Pediatric Growth Hormone Deficiency LUM-201 Growth hormone secretagogue Height Catch-up growth PEM Oral Predictive Enrichment Marker

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children With Growth Hormone Deficiency (GHD)

Quick Facts

Study Start:2025-10

Study Completion:2028-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06948214

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:3 Years to 11 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
* Subjects must be naïve to treatment and prepubertal * Subjects must have a maximal GH response of \< 10 ng/mL from 2 prior GH stimulation tests conducted within the preceding 12 months * Impaired height defined as ≥ 2.0 standard deviations (SDs) below the mean height for chronological age and sex * Morning or random cortisol level of ≥ 7.0 μg/dL * ≥ 3.0 years and age ≤ 10.0 years for girls and ≤ 11.0 years for boys * Baseline height velocity (HV) based on ≥ 6 months of growth assessments \< 25th percentile for age and sex * Bone Age delay of ≥ 12 months compared to the chronological age * 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 3 months prior to Day 1 * Baseline IGF-1 standard deviation score (SDS) ≤ -1.0	* 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. * Arm span to height ratio \> 2 SDs below the mean for age and sex * A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201 * 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 * Current inflammatory diseases requiring systemic corticosteroid treatment for \> 2 consecutive weeks within the last 3 months prior to the Screening Visit * Use of hormone replacement therapy for any hormone deficiency other than thyroid deficiency * Any ECG at the Screening Visit noted to have a clinically significant abnormality, as confirmed by the MM * Any subjects suspected of having past or present intracranial tumor growth as confirmed by brain imaging prior to the Screening or Day 1 Visit * Any subject suspected of having intracranial hypertension (IH) as confirmed by fundoscopy and other assessments * Any subject with serum alanine transaminase (ALT), aspartate transaminase (AST), or total bilirubin \> upper limit of normal (ULN) * Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3.0 ng/mL on any prior standard of care GH stimulation test completed within 12 months * Body weight ≤ 14.0 kg * BMI \< -2 or \> +2 SDs for age and sex based on WHO standards * Birth weight for gestational age \< 3rd percentile based on WHO standards * Treatment with medications known to be moderate or strong inhibitors or strong inducers of cytochrome P450 (CYP) 3A/4 * History of spinal, cranial, or total body irradiation * Attention deficit hyperactivity disorder (ADHD) diagnosis

Inclusion Criteria

Exclusion Criteria

* Subjects must be naïve to treatment and prepubertal
* Subjects must have a maximal GH response of \< 10 ng/mL from 2 prior GH stimulation tests conducted within the preceding 12 months
* Impaired height defined as ≥ 2.0 standard deviations (SDs) below the mean height for chronological age and sex
* Morning or random cortisol level of ≥ 7.0 μg/dL
* ≥ 3.0 years and age ≤ 10.0 years for girls and ≤ 11.0 years for boys
* Baseline height velocity (HV) based on ≥ 6 months of growth assessments \< 25th percentile for age and sex
* Bone Age delay of ≥ 12 months compared to the chronological age
* 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 3 months prior to Day 1
* Baseline IGF-1 standard deviation score (SDS) ≤ -1.0

* 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.
* Arm span to height ratio \> 2 SDs below the mean for age and sex
* A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201
* 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
* Current inflammatory diseases requiring systemic corticosteroid treatment for \> 2 consecutive weeks within the last 3 months prior to the Screening Visit
* Use of hormone replacement therapy for any hormone deficiency other than thyroid deficiency
* Any ECG at the Screening Visit noted to have a clinically significant abnormality, as confirmed by the MM
* Any subjects suspected of having past or present intracranial tumor growth as confirmed by brain imaging prior to the Screening or Day 1 Visit
* Any subject suspected of having intracranial hypertension (IH) as confirmed by fundoscopy and other assessments
* Any subject with serum alanine transaminase (ALT), aspartate transaminase (AST), or total bilirubin \> upper limit of normal (ULN)
* Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3.0 ng/mL on any prior standard of care GH stimulation test completed within 12 months
* Body weight ≤ 14.0 kg
* BMI \< -2 or \> +2 SDs for age and sex based on WHO standards
* Birth weight for gestational age \< 3rd percentile based on WHO standards
* Treatment with medications known to be moderate or strong inhibitors or strong inducers of cytochrome P450 (CYP) 3A/4
* History of spinal, cranial, or total body irradiation
* Attention deficit hyperactivity disorder (ADHD) diagnosis

Contacts and Locations

Study Contact

Lumos Pharma

CONTACT

515-598-2921

clinical.trials@lumos-pharma.com

Study Locations (Sites)

Lumos Pharma Investigational Site

Greenwood Village, Colorado, 80111

United States

Collaborators and Investigators

Sponsor: Lumos Pharma

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-10

Study Completion Date2028-01

Study Record Updates

Study Start Date2025-10

Study Completion Date2028-01

Terms related to this study

Keywords Provided by Researchers

GHD
Pediatric Growth Hormone Deficiency
LUM-201
Growth hormone secretagogue
Height
Catch-up growth
PEM
Oral
Predictive Enrichment Marker

Additional Relevant MeSH Terms

Growth Hormone Deficiency (GHD)