NCT05796440

Brief Summary

This is a multi-national trial. The trial aims to study the long-term safety of LUM-201 in subjects with Idiopathic Pediatric Growth Hormone Deficiency (iPGHD). This study will also assess pharmacodynamics and efficacy response to therapy with LUM-201.

Trial Health

78
On Track

Trial Health Score

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

Enrollment
60

participants targeted

Target at P50-P75 for phase_2

Timeline
23mo left

Started Aug 2023

Longer than P75 for phase_2

Geographic Reach
2 countries

7 active sites

Status
enrolling by invitation

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

Study Progress59%
Aug 2023Apr 2028

First Submitted

Initial submission to the registry

March 19, 2023

Completed
15 days until next milestone

First Posted

Study publicly available on registry

April 3, 2023

Completed
4 months until next milestone

Study Start

First participant enrolled

August 1, 2023

Completed
4.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2028

Expected
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2028

Last Updated

February 15, 2024

Status Verified

February 1, 2024

Enrollment Period

4.6 years

First QC Date

March 19, 2023

Last Update Submit

February 14, 2024

Conditions

Keywords

LUM-201PGHDGHDGrowth hormone secretagogueOralHeight

Outcome Measures

Primary Outcomes (1)

  • Incidence of treatment-emergent adverse events

    Number of events

    Day 1 to Month 36

Secondary Outcomes (6)

  • Growth Hormone Concentrations in subjects

    Day 1 to Month 36

  • Insulin-like growth factor 1 concentration

    Day 1 to Month 36

  • Height standard deviation score (SDS)

    Day 1 to Month 36

  • Change in Weight SDS

    Day 1 to Month 36

  • Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201

    Day 1 to Month 36

  • +1 more secondary outcomes

Study Arms (1)

LUM-201 (1.6 mg/kg/day)

EXPERIMENTAL
Drug: LUM-201

Interventions

Administered orally once daily

LUM-201 (1.6 mg/kg/day)

Eligibility Criteria

Age5 Years - 14 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 successfully participated in a pediatric LUM-201 GHD study through at least the 12-month visit, and be eligible for continuation of treatment, pending all other enrollment criteria are met.

You may not qualify if:

  • Medical or genetic condition that, in the opinion of the PI and/or MMs, adds unwarranted risk to the use of LUM-201
  • 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. Subjects receiving shorter-term (two weeks or less) treatment with these medications should be evaluated on case-by-case basis by the PI in consultation with the MMs.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

University of Iowa

Iowa City, Iowa, 52242, United States

Location

UMass Memorial Medical Center

Worcester, Massachusetts, 01655, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64111, United States

Location

University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Texas Tech University Health Sciences Center

Amarillo, Texas, 79106, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Canberra Hospital

Garran, Australian Capital Territory, 2605, Australia

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

Study Design

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

Study Record Dates

First Submitted

March 19, 2023

First Posted

April 3, 2023

Study Start

August 1, 2023

Primary Completion (Estimated)

March 1, 2028

Study Completion (Estimated)

April 1, 2028

Last Updated

February 15, 2024

Record last verified: 2024-02

Locations