Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial)
- Conditions
- Growth Hormone Deficiency
- Interventions
- Drug: LUM-201Drug: rhGH Norditropin® pen (34 µg/kg)
- Registration Number
- NCT04614337
- Lead Sponsor
- Lumos Pharma
- Brief Summary
This is a multi-national trial. The goals of the trial are to study LUM-201 as a possible treatment for Pediatric Growth Hormone Deficiency (PGHD) and investigate a predictive enrichment marker (PEM) strategy to select subjects likely to respond to therapy with LUM-201.
- Detailed Description
This trial will have one screening visit with tests 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 be randomized to receive one of three oral daily doses of LUM-201 or daily injections of recombinant human growth hormone (rhGH). All subjects will have an equal chance of being placed in any of the four groups.
The trial consists of up to 24 months of treatment. After screening, subjects will return to the clinic for 6 (subjects placed in rhGH group) or 10 visits (subjects placed in LUM-201 group). During several of these clinic visits, subjects will have a physical exam, blood, and urine collections. There will also be 3 phone calls with study staff that will take place between the clinic visits.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 82
- Have an established diagnosis of idiopathic PGHD as determined by standard diagnostic criteria. Eligible subjects must be naïve-to-treatment and be prepubertal.
- Morning cortisol ≥ 7 µg/dL or stimulated cortisol ≥ 14 µg/dL.
- At Screening, be ≥ 3.0 years and ≤ 11.0 years for girls and ≤ 12.0 years for boys.
- Have HT-SDS ≤ -2.0 or HT-SDS ≥ 2 SD below mean parental HT-SDS.
- Have a baseline height velocity < 5.5 cm/year based on at least 6 months of growth.
- Have a bone age delayed by ≥ 6 months with respect to chronological age.
- Have prepubertal status as evidenced by Tanner Stage I breast development in girls and testicular volume < 4.0 mL in boys.
- 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 Day 1.
- 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. (Examples: diabetes, idiopathic short stature).
- A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201 or rhGH.
- 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 (Example: glucocorticoids).
- Evidence or history of an intracranial mass (e.g., pituitary tumor, craniopharyngioma).
- Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3 ng/mL on two prior standard of care GH stimulation tests, or pituitary deficiencies beyond GH and thyroid function.
- Malnutrition as evidenced by medical history or a body weight < 3rdth percentile for current height.
- BMI > 95th percentile.
- Gestational age-adjusted birth weight < 5th percentile (small for gestational age).
- History of spinal, cranial, or total body irradiation.
- Treatment with medications known to act as moderate or strong inhibitors or strong inducers of CYP3A/4, or with medications known to act as strong inhibitors of P-glycoprotein (P-gp) or potent substrates of P-gp or Multidrug and toxin extrusion protein 1 (MATE1).
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description LUM-201 (1.6 mg/kg/day) LUM-201 - rhGH (34 µg/kg/day) rhGH Norditropin® pen (34 µg/kg) - LUM-201 (0.8 mg/kg/day) LUM-201 - LUM-201 (3.2 mg/kg/day) LUM-201 -
- Primary Outcome Measures
Name Time Method AHV after 6 months on LUM-201 compared to rhGH Day 1 to Month 6 Annualized height velocity to be measured
Percentage of subjects selected by PEM strategy who meet target growth Day 1 to Month 6 Annualized height velocity (AHV) measured as standing height with stadiometer
- Secondary Outcome Measures
Name Time Method Incidence of adverse events in children with GHD Day 1 to Month 24 Number of events
Bone Age Day 1 to Month 6 and Month 18 Change in bone age, measured by X-ray of left hand and wrist using Greulich \& Pyle atlas
Height standard deviation score (SDS) Day 1 to Month 6 and Month 12 Change in HT-SDS
Height velocity standard deviation score (HV-SDS) Day 1 to Month 6, and Month 12 Change in HV-SDS
Change in Weight Day 1 to Month 6, and Month 12 Change in Weight
Insulin-like growth factor 1 SDS Day 1 to Month 6 and 12 Serum concentrations of insulin-like growth factor 1
Degree of concordance between the first and second assessment with the PEM strategy. Screening to Day 1 Peak serum concentration of GH in response to a single provocative dose of LUM-201
Change in Weight SDS Day 1 to Month 6 and Month 12 Change in Weight-SDS
Change in BMI Day 1 to Month 6 and Month 12 Change in BMI
Change in BMI SDS Day 1 to Month 6 and Month 12 Change in BMI SDS
GH Concentration on maintenance treatment Day 1 to Month 6 and 12 Serum GH concentration
Pharmacokinetics of LUM-201 Day 1 to Month 6 and 12 Serum concentrations (Cmax/Steady State)
Trial Locations
- Locations (43)
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Atlanta Diabetes Associates
🇺🇸Atlanta, Georgia, United States
Children's National Hospital
🇺🇸Washington, District of Columbia, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Children's Minnesota
🇺🇸Saint Paul, Minnesota, United States
Pediatric Endocrine Associates
🇺🇸Greenwood Village, Colorado, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
The Mount Sinai Hospital
🇺🇸Mount Sinai, New York, United States
UBMD Pediatrics
🇺🇸Buffalo, New York, United States
NYU Grossman School of Medicine
🇺🇸New York, New York, United States
Penn State College of Medicine
🇺🇸Hershey, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Texas Tech University Health Sciences Center
🇺🇸Amarillo, Texas, United States
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States
MultiCare Institute for Research and Innovation
🇺🇸Tacoma, Washington, United States
University of Virginia Health System
🇺🇸Charlottesville, Virginia, United States
Canberra Hospital
🇦🇺Garran, Australian Capital Territory, Australia
Department of Pediatrics and Endocrinology- Monash Health
🇦🇺Clayton, Victoria, Australia
Royal Children's Hospital
🇦🇺Melbourne, Victoria, Australia
Schneider Children's Medical Center Institute for Endocrinology and Diabetes National Center
🇮🇱Petah Tiqwa, Tiqwa, Israel
Liggins Institute, University of Auckland
🇳🇿Auckland, New Zealand
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
Wellington Regional Hospital CCDHB
🇳🇿Newtown, Wellington, New Zealand
Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki
🇵🇱Lodz, Poland
Klinika Pediatrii, Endokrynologii, Diabetologii z Pododdziałem Kardiologii, Uniwersytecki Dziecięcy Szpital Kliniczny im.Ludwika Zamenhofa w Białymstoku
🇵🇱Bialystok, Poland
Klinika Pediatrii, Diabetologii i Endokrynologii Gdansk
🇵🇱Pomorskie, Poland
Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka
🇵🇱Warsaw, Poland
klinika Pediatrii, Endokrynologii i Diabetologii Dziecięcej
🇵🇱Rzeszów, Poland
Sonomed - Centrum Medyczne
🇵🇱Szczecin, Poland
SP Dziecięcy Szpital Kliniczny w Warszawie
🇵🇱Warsaw, Poland
Klinika Endokrynologii i Diabetologii Wieku Rozwojowego UM
🇵🇱Wrocław, Poland
State Institution 'V. P. Komissarenko Institute of Endocrinology and Metabolism of the National academy of medical science of Ukraine
🇺🇦Kyiv, Ukraine
UMass Memorial Medical Center
🇺🇸Worcester, Massachusetts, United States
Indiana University School of Medicine
🇺🇸Indianapolis, Indiana, United States
Center of Excellence in Diabetes and Endocrinology
🇺🇸Sacramento, California, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Diabetes & Glandular Disease Clinic, P.A.
🇺🇸San Antonio, Texas, United States
Rady Children's Hospital
🇺🇸San Diego, California, United States
Novak Center For Childrens Health
🇺🇸Louisville, Kentucky, United States
M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic
🇺🇸Minneapolis, Minnesota, United States
The Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
University of Oklahoma Health Sciences Center, Pediatric Diabetes and Endocrinology
🇺🇸Oklahoma City, Oklahoma, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States