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

NCT ID: NCT04614337

Last Updated: 2024-10-15

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 82 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-12-31
• Study Completion Date: 2024-09-04

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.

Conditions

Growth Hormone Deficiency

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

LUM-201 (0.8 mg/kg/day)

Group Type: EXPERIMENTAL

LUM-201

Intervention Type: DRUG

Administered orally once daily

LUM-201 (1.6 mg/kg/day)

Group Type: EXPERIMENTAL

LUM-201

Intervention Type: DRUG

Administered orally once daily

LUM-201 (3.2 mg/kg/day)

Group Type: EXPERIMENTAL

LUM-201

Intervention Type: DRUG

Administered orally once daily

rhGH (34 µg/kg/day)

Group Type: ACTIVE_COMPARATOR

rhGH Norditropin® pen (34 µg/kg)

Intervention Type: DRUG

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

Interventions

LUM-201

Administered orally once daily

Intervention Type: DRUG

rhGH Norditropin® pen (34 µg/kg)

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

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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.

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. (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).

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Lumos Pharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Center of Excellence in Diabetes and Endocrinology

Sacramento, California, United States

Rady Children's Hospital

San Diego, California, United States

Pediatric Endocrine Associates

Greenwood Village, Colorado, United States

Children's National Hospital

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

Atlanta Diabetes Associates

Atlanta, Georgia, United States

Indiana University School of Medicine

Indianapolis, Indiana, United States

University of Iowa

Iowa City, Iowa, United States

Novak Center For Childrens Health

Louisville, Kentucky, United States

UMass Memorial Medical Center

Worcester, Massachusetts, United States

M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic

Minneapolis, Minnesota, United States

Children's Minnesota

Saint Paul, Minnesota, United States

The Children's Mercy Hospital

Kansas City, Missouri, United States

UBMD Pediatrics

Buffalo, New York, United States

The Mount Sinai Hospital

Mount Sinai, New York, United States

NYU Grossman School of Medicine

New York, New York, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

University of Oklahoma Health Sciences Center, Pediatric Diabetes and Endocrinology

Oklahoma City, Oklahoma, United States

Penn State College of Medicine

Hershey, Pennsylvania, United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Texas Tech University Health Sciences Center

Amarillo, Texas, United States

Cook Children's Medical Center

Fort Worth, Texas, United States

Diabetes & Glandular Disease Clinic, P.A.

San Antonio, Texas, United States

University of Virginia Health System

Charlottesville, Virginia, United States

Seattle Children's Hospital

Seattle, Washington, United States

MultiCare Institute for Research and Innovation

Tacoma, Washington, 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

Queensland Children's Hospital

South Brisbane, , Australia

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

Petah Tikva, Tiqwa, Israel

Wellington Regional Hospital CCDHB

Newtown, Wellington Region, New Zealand

Liggins Institute, University of Auckland

Auckland, , New Zealand

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

Bialystok, , Poland

Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki

Lodz, , Poland

Klinika Pediatrii, Diabetologii i Endokrynologii Gdansk

Pomorskie, , Poland

klinika Pediatrii, Endokrynologii i Diabetologii Dziecięcej

Rzeszów, , Poland

Sonomed - Centrum Medyczne

Szczecin, , Poland

Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka

Warsaw, , Poland

SP Dziecięcy Szpital Kliniczny w Warszawie

Warsaw, , Poland

Klinika Endokrynologii i Diabetologii Wieku Rozwojowego UM

Wroclaw, , Poland

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

Kyiv, , Ukraine

Countries

United States; Australia; Israel; New Zealand; Poland; Ukraine

Other Identifiers

LUM-201-01

Identifier Type: -

Identifier Source: org_study_id