NCT05250063

Brief Summary

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Feb 2022

Typical duration for phase_2

Geographic Reach
2 countries

10 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 10, 2022

Completed
8 days until next milestone

Study Start

First participant enrolled

February 18, 2022

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 22, 2022

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 22, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 22, 2025

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

April 13, 2026

Completed
Last Updated

April 13, 2026

Status Verified

March 1, 2026

Enrollment Period

2.9 years

First QC Date

February 10, 2022

Results QC Date

March 24, 2026

Last Update Submit

March 24, 2026

Conditions

Growth Hormone Deficiency

Keywords

LUM-201PGHDHeightOral

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (11)

  • Incidence of Adverse Events in Children With GHD

    Day 1 to Month 12

  • Growth Hormone (GH) Values

    Day 1 to Month 12

  • Insulin-Like Growth Factor 1 (IGF-1) Values

    Day 1 to Month 12

  • Insulin-Like Growth Factor Binding Protein 3 (IGFBP-3) Values

    Day 1 to Month 12

  • Height Standard Deviation Score (SDS)

    Day 1 to Month 12

  • +6 more secondary outcomes

Study Arms (1)

LUM-201 (3.2 mg/kg/day)

EXPERIMENTAL
Drug: LUM-201

Interventions

LUM-201DRUG

Administered orally once daily

LUM-201 (3.2 mg/kg/day)

Eligibility Criteria

Age3 Years - 13 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Rady Children's Hospital

San Diego, California, 92123, United States

Location

Children's National Hospital

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

Location

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic

Minneapolis, Minnesota, 55454, United States

Location

The Children's Mercy Hospital

Kansas City, Missouri, 64111, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

University of Virginia Health System

Charlottesville, Virginia, 22908, United States

Location

Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki

Lodz, Poland

Location

Sonomed - Centrum Medyczne

Szczecin, Poland

Location

Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka

Warsaw, Poland

Location

MeSH Terms

Conditions

Dwarfism, Pituitary

Condition Hierarchy (Ancestors)

DwarfismBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesBone Diseases, EndocrineHypopituitarismPituitary DiseasesHypothalamic DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesEndocrine System Diseases

Results Point of Contact

Title
Lumos Pharma LLC
Organization
Lumos Pharma LLC
clinical.trials@lumos-pharma.com
515-598-2921

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
GT60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 10, 2022

First Posted

February 22, 2022

Study Start

February 18, 2022

Primary Completion

January 22, 2025

Study Completion

January 22, 2025

Last Updated

April 13, 2026

Results First Posted

April 13, 2026

Record last verified: 2026-03

Locations

US(7)PL(3)