Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial)

NCT04614337 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: LUM-201-01
• Study Type: interventional
• Target: 82
• Locations: 6 countries
• Sites: 43

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.

Conditions

Growth Hormone Deficiency

Keywords

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

Outcome Measures

Primary Outcomes (2)

• Percentage of subjects selected by PEM strategy who meet target growth

  Annualized height velocity (AHV) measured as standing height with stadiometer

  Day 1 to Month 6

• AHV after 6 months on LUM-201 compared to rhGH

  Annualized height velocity to be measured

  Day 1 to Month 6

Secondary Outcomes (12)

• Degree of concordance between the first and second assessment with the PEM strategy.

  Screening to Day 1

• Incidence of adverse events in children with GHD

  Day 1 to Month 24

• Height standard deviation score (SDS)

  Day 1 to Month 6 and Month 12

• Height velocity standard deviation score (HV-SDS)

  Day 1 to Month 6, and Month 12

• Change in Weight

  Day 1 to Month 6, and Month 12

Study Arms (4)

LUM-201 (0.8 mg/kg/day)

EXPERIMENTAL

Drug: LUM-201

LUM-201 (1.6 mg/kg/day)

EXPERIMENTAL

Drug: LUM-201

LUM-201 (3.2 mg/kg/day)

EXPERIMENTAL

Drug: LUM-201

rhGH (34 µg/kg/day)

ACTIVE COMPARATOR

Drug: rhGH Norditropin® pen (34 µg/kg)

Interventions

LUM-201 DRUG

Administered orally once daily

LUM-201 (0.8 mg/kg/day); LUM-201 (1.6 mg/kg/day); LUM-201 (3.2 mg/kg/day)

rhGH Norditropin® pen (34 µg/kg) DRUG

Administered subcutaneously (s.c., under the skin) once daily.

rhGH (34 µg/kg/day)

Eligibility Criteria

• Age: 3 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Lumos Pharma: lead

Study Sites (43)

Center of Excellence in Diabetes and Endocrinology

Sacramento, California, 95821, United States

Location

Rady Children's Hospital

San Diego, California, 92123, United States

Location

Pediatric Endocrine Associates

Greenwood Village, Colorado, 80111, United States

Location

Children's National Hospital

Washington D.C., District of Columbia, 20010, United States

Location

Atlanta Diabetes Associates

Atlanta, Georgia, 30318, United States

Location

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Novak Center For Childrens Health

Louisville, Kentucky, 40202, United States

Location

UMass Memorial Medical Center

Worcester, Massachusetts, 01655, United States

Location

M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic

Minneapolis, Minnesota, 55454, United States

Location

Children's Minnesota

Saint Paul, Minnesota, 55102, United States

Location

The Children's Mercy Hospital

Kansas City, Missouri, 64111, United States

Location

UBMD Pediatrics

Buffalo, New York, 14203, United States

Location

The Mount Sinai Hospital

Mount Sinai, New York, 30093, United States

Location

NYU Grossman School of Medicine

New York, New York, 10016, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

University of Oklahoma Health Sciences Center, Pediatric Diabetes and Endocrinology

Oklahoma City, Oklahoma, 73104, United States

Location

Penn State College of Medicine

Hershey, Pennsylvania, 17033, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Texas Tech University Health Sciences Center

Amarillo, Texas, 79106, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Diabetes & Glandular Disease Clinic, P.A.

San Antonio, Texas, 78229, United States

Location

University of Virginia Health System

Charlottesville, Virginia, 22908, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

MultiCare Institute for Research and Innovation

Tacoma, Washington, 98405, United States

Location

Canberra Hospital

Garran, Australian Capital Territory, 2605, Australia

Location

Department of Pediatrics and Endocrinology- Monash Health

Clayton, Victoria, 3168, Australia

Location

Royal Children's Hospital

Melbourne, Victoria, 3052, Australia

Location

Queensland Children's Hospital

South Brisbane, Australia

Location

Schneider Children's Medical Center Institute for Endocrinology and Diabetes National Center

Petah Tikva, Tiqwa, 4920235, Israel

Location

Wellington Regional Hospital CCDHB

Newtown, Wellington Region, 6021, New Zealand

Location

Liggins Institute, University of Auckland

Auckland, New Zealand

Location

Klinika Pediatrii, Endokrynologii, Diabetologii z Pododdziałem Kardiologii, Uniwersytecki Dziecięcy Szpital Kliniczny im.Ludwika Zamenhofa w Białymstoku

Bialystok, Poland

Location

Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki

Lodz, Poland

Location

Klinika Pediatrii, Diabetologii i Endokrynologii Gdansk

Pomorskie, Poland

Location

klinika Pediatrii, Endokrynologii i Diabetologii Dziecięcej

Rzeszów, Poland

Location

Sonomed - Centrum Medyczne

Szczecin, Poland

Location

Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka

Warsaw, Poland

Location

SP Dziecięcy Szpital Kliniczny w Warszawie

Warsaw, Poland

Location

Klinika Endokrynologii i Diabetologii Wieku Rozwojowego UM

Wroclaw, Poland

Location

State Institution 'V. P. Komissarenko Institute of Endocrinology and Metabolism of the National academy of medical science of Ukraine

Kyiv, 04114, Ukraine

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: October 26, 2020
• First Posted: November 4, 2020
• Study Start: December 31, 2020
• Primary Completion: September 4, 2024
• Study Completion: September 4, 2024
• Last Updated: October 15, 2024

Record last verified: 2024-10

Locations

UA (1); PL (8); AU (4); NZ (2); IL (1); US (27)