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

NCT ID: NCT06948214

Last Updated: 2026-01-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

150 participants

Study Classification

INTERVENTIONAL

Study Start Date

2026-01-31

Study Completion Date

2028-01-31

Brief Summary

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The OraGrowtH Phase 3 Trial 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 the LUM-201 predictive enrichment marker (LUM-201 PEM) strategy to select subjects likely to respond to therapy with daily oral LUM-201.

Detailed Description

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Conditions

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Growth Hormone Deficiency (GHD)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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LUM-201

Group Type EXPERIMENTAL

LUM-201

Intervention Type DRUG

1.6 mg/kg/day, administered orally once daily

Placebo

Group Type PLACEBO_COMPARATOR

Matched Placebo (Capsules)

Intervention Type OTHER

Administered orally once daily

Interventions

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LUM-201

1.6 mg/kg/day, administered orally once daily

Intervention Type DRUG

Matched Placebo (Capsules)

Administered orally once daily

Intervention Type OTHER

Eligibility Criteria

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Inclusion 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

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.
* 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
Minimum Eligible Age

3 Years

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Lumos Pharma

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Lumos Pharma Investigational Site

Birmingham, Alabama, United States

Site Status RECRUITING

Lumos Pharma Investigational Site

Sacramento, California, United States

Site Status RECRUITING

Lumos Pharma Investigational Site

Centennial, Colorado, United States

Site Status RECRUITING

Lumos Pharma Investigational Site

Greenwood Village, Colorado, United States

Site Status RECRUITING

Lumos Pharma Investigational Site

Orlando, Florida, United States

Site Status RECRUITING

Lumos Pharma Investigational Site

Tallahassee, Florida, United States

Site Status RECRUITING

Lumos Pharma Investigational Site

New Orleans, Louisiana, United States

Site Status RECRUITING

Lumos Pharma Investigational Site

New Brunswick, New Jersey, United States

Site Status RECRUITING

Lumos Pharma Investigational Site

Staten Island, New York, United States

Site Status RECRUITING

Lumos Pharma Investigational Site

Columbia, South Carolina, United States

Site Status RECRUITING

Lumos Pharma Investigational Site

Corpus Christi, Texas, United States

Site Status RECRUITING

Lumos Pharma Investigational Site

Seattle, Washington, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Lumos Pharma

Role: CONTACT

515-598-2921

Other Identifiers

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LUM-201-10

Identifier Type: -

Identifier Source: org_study_id

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