Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213) (OraGrowtH213)

A Multicenter, Open-Label, Phase 2 Study to Evaluate Growth and Safety of LUM-201 Following 12 Months of Daily rhGH Treatment in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed the LUM-201-01 Trial

This is a multi-national trial. The goals of the trial are to study the growth response to LUM-201 administration in children with idiopathic growth hormone deficiency (GHD) previously treated with daily rhGH for 12 months in the LUM-201-01 trial.

Study Overview

• Status: Completed
• Conditions: Growth Hormone Deficiency
• Intervention / Treatment: Drug: LUM-201

Detailed Description

This trial will have one screening/baseline visit with tests to assess if subjects are eligible to start study therapy. The screening/baseline visit can coincide with the Month 12 visit in the LUM-201-01 trial. Subjects will receive the same dose of LUM-201 as a daily oral dose each morning. LUM-201 will be adjusted for body weight increments at each clinic visit, as applicable.

The trial consists of 12 months of treatment. After screening, subjects will return to the clinic for 5 visits. During four of these clinic visits, safety laboratory samples will be collected.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 2

Contacts and Locations

Study Locations

• Poland
  • Lodz, Poland
    • Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki
  • Szczecin, Poland
    • Sonomed - Centrum Medyczne
  • Warsaw, Poland
    • Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka
• United States
  • California
    • San Diego, California, United States, 92123
      • Rady Children's Hospital
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • Children's National Hospital
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University School of Medicine
  • Minnesota
    • Minneapolis, Minnesota, United States, 55454
      • M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic
  • Missouri
    • Kansas City, Missouri, United States, 64111
      • The Children's Mercy Hospital
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
  • Virginia
    • Charlottesville, Virginia, United States, 22908
      • University Of Virginia Health System

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 13 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable.
• Must have previously completed 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial.
• Is eligible for study participation as confirmed by the principal investigator (PI)

Exclusion Criteria:

• Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201.
• Uses any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors.
• Has planned or is receiving current long-term treatment with medications known to act as substrates, inducers, or inhibitors of the cytochrome system CYP3A4 that metabolizes LUM-201

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: LUM-201 (3.2 mg/kg/day)	Drug: LUM-201; Administered orally once daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized Height Velocity (AHV) Measured as Standing Height With Stadiometer	Mean change from day 1 to month 12, Annualized Height Velocity (AHV) Height is measured in triplicate, by a calibrated stadiometer and taking the mean value AVH equation is (h2-h1/t2-t1)*365.25; h1 = height measured at day 1 h2 = height measured at month 12 t1 = day 1 t2 = month 12	Day 1 to Month 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Growth Hormone (GH) Values	Mean GH Values change from Day 1 to month 12 (Pre-Dose and 1 hour Post-Dose)	Day 1 to Month 12
Insulin-Like Growth Factor 1 (IGF-1) Values	Mean change from Day 1 to month 12 (IGF-1 Values Pre-Dose)	Day 1 to Month 12
Insulin-Like Growth Factor Binding Protein 3 (IGFBP-3) Values	Mean change from day 1 to month 12 (IGFBP-3 Values Pre-Dose)	Day 1 to Month 12
Change in Weight	Mean change in Weight from day 1 to month 12	Day 1 to Month 12
Change in Weight - Standard Deviation Score (SDS)	Mean change in Weight-SDS from day 1 to month 12	Day 1 to Month 12
Change in Body Mass Index (BMI)	Mean change from day 1 to month 12 in BMI	Day 1 to Month 12
Change in Body Mass Index (BMI) - Standard Deviation Score (SDS)	Mean change from Day 1 to month 12 in BMI-SDS	Day 1 to Month 12
Change in Bone Age	Mean change from day 1 to month 6 in bone age, measured by X-ray of left hand and wrist using Greulich & Pyle atlas	Month 6
Insulin-Like Growth Factor 1 (IGF-1) - Standard Deviation Score (SDS)	Mean Change from day 1 to month 12 for IGF-1 - SDS Pre-dose Values	Day 1 to Month 12
Occurrence of Adverse Events in Children With Growth Hormone Deficiency (GHD)	Number of events	Day 1 to Month 12
Height (HT) Standard Deviation Score (SDS)	Mean change from day 1 to month 12 in HT-SDS	Day 1 to Month 12

Collaborators and Investigators

• Sponsor: Lumos Pharma

Study record dates

Study Major Dates

• Study Start (Actual): February 18, 2022
• Primary Completion (Actual): January 22, 2025
• Study Completion (Actual): January 22, 2025

Study Registration Dates

• First Submitted: February 10, 2022
• First Submitted That Met QC Criteria: February 10, 2022
• First Posted (Actual): February 22, 2022

Study Record Updates

• Last Update Posted (Actual): May 15, 2026
• Last Update Submitted That Met QC Criteria: April 24, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Oral; Height; LUM-201; PGHD
• Additional Relevant MeSH Terms: Endocrine System Diseases; Bone Diseases; Musculoskeletal Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hypothalamic Diseases; Pituitary Diseases; Bone Diseases, Endocrine; Bone Diseases, Developmental; Dwarfism; Hypopituitarism; Dwarfism, Pituitary
• Other Study ID Numbers: LUM-201-04

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No