Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
Purpose
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.
Condition
- Growth Hormone Deficiency (GHD)
Eligibility
- Eligible Ages
- Between 3 Years and 11 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Care Provider, Investigator)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental LUM-201 |
|
|
|
Placebo Comparator Placebo |
|
Recruiting Locations
Lumos Pharma Investigational Site
Birmingham, Alabama 35233
Birmingham, Alabama 35233
Lumos Pharma Investigational Site
Madera, California 93636
Madera, California 93636
Lumos Pharma Investigational Site
Sacramento, California 95821
Sacramento, California 95821
Lumos Pharma Investigational Site
Centennial, Colorado 80112
Centennial, Colorado 80112
Lumos Pharma Investigational Site
Greenwood Village, Colorado 80111
Greenwood Village, Colorado 80111
Lumos Pharma Investigational Site
Washington D.C., District of Columbia 20010
Washington D.C., District of Columbia 20010
Lumos Pharma Investigational Site
Hollywood, Florida 33021
Hollywood, Florida 33021
Lumos Pharma Investigational Site
Miami, Florida 33155
Miami, Florida 33155
Lumos Pharma Investigational Site
Orlando, Florida 32806
Orlando, Florida 32806
Lumos Pharma Investigational Site
Tallahassee, Florida 32308
Tallahassee, Florida 32308
Lumos Pharma Investigational Site
Indianapolis, Indiana 46202
Indianapolis, Indiana 46202
Lumos Pharma Investigational Site
Iowa City, Iowa 52242
Iowa City, Iowa 52242
Lumos Pharma Investigational Site
New Orleans, Louisiana 70118
New Orleans, Louisiana 70118
Lumos Pharma Investigational Site
Minneapolis, Minnesota 55454
Minneapolis, Minnesota 55454
Lumos Pharma Investigational Site
Kansas City, Missouri 64108
Kansas City, Missouri 64108
Lumos Pharma Investigational Site
Las Vegas, Nevada 89113
Las Vegas, Nevada 89113
Lumos Pharma Investigational Site
New Brunswick, New Jersey 08901
New Brunswick, New Jersey 08901
Lumos Pharma Investigational Site
Staten Island, New York 10306
Staten Island, New York 10306
Lumos Pharma Investigational Site
Columbia, South Carolina 29203
Columbia, South Carolina 29203
Lumos Pharma Investigational Site
Amarillo, Texas 79106
Amarillo, Texas 79106
Lumos Pharma Investigational Site
Corpus Christi, Texas 78411
Corpus Christi, Texas 78411
Lumos Pharma Investigational Site
Fort Worth, Texas 76104
Fort Worth, Texas 76104
Lumos Pharma Investigational Site
Charlottesville, Virginia 22908
Charlottesville, Virginia 22908
Lumos Pharma Investigational Site
Seattle, Washington 98105
Seattle, Washington 98105
More Details
- Status
- Recruiting
- Sponsor
- Lumos Pharma