Phase 3 Long Term Safety Extension Study of LUM-201 in Children With Growth Hormone Deficiency

NCT07129759 | Not Yet Recruiting Phase 3 FDA Drug

• 1 other identifier: LUM-201-11
• Study Type: interventional
• Target: 150
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a Multi-national Trial. The Goal of the Trial is to Offer Subjects Who Complete 12 Months in the LUM-201-10 Phase 3 Trial up to an Additional 36 Months of Treatment of LUM-201 While Evaluating Safety and Tolerability of LUM-201.

Conditions

Growth Hormone Deficiency (GHD)

Outcome Measures

Primary Outcomes (1)

• To evaluate the long-term safety and tolerability of LUM-201

  * The number of subjects with at least 1 (serious) treatment-related adverse event (TEAE). * The number of subjects with at least 1 suspected unexpected serious adverse reaction. * The number of subjects with clinically significant abnormalities related to trial laboratory tests or electrocardiograms (ECGs). * The number of subjects with at least 1 adverse event of special interest (AESI). * Annual change from baseline in body weight standard deviation score (SDS). * Annual change from baseline in body mass index (BMI) SDS.

  Day 1 - Month 36

Secondary Outcomes (2)

• To evaluate the long-term effect of LUM-201 on growth.

  Day 1 - Month 36

• To evaluate the long-term effect of LUM-201 on pharmacodynamic (PD) markers.

  Day 1 - Month 36

Study Arms (1)

LUM-201

EXPERIMENTAL

Drug: LUM-201

Interventions

LUM-201 DRUG

1.6 mg/kg/day, administered orally once daily

LUM-201

Eligibility Criteria

• Age: 4 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Parent/caregiver must be willing to provide written informed consent, and the subject must sign the assent, as applicable.
• Subject must have successfully completed 12 months of participation in the LUM-201 Phase 3 GHD trial, and be eligible for continuation of treatment, pending all other enrollment criteria are met.
• Subject who is sexually active must use an acceptable form of contraception.
• Subject must be eligible for the Day 1 visit as confirmed by the Investigator.

You may not qualify if:

• Subject has a medical or genetic condition that, in the opinion of the Investigator and/or MMs, adds unwarranted risk to use of LUM-201.
• Pregnancy.
• Subject has planned or is receiving current long-term treatment with medications known to prolong the QT interval or act as substrates, inducers, or inhibitors of the cytochrome system cytochrome P450 type 3A4 that metabolizes LUM-201 (see Appendix 6 for list of example medications). Subjects receiving shorter-term (two weeks or less) treatment with these medications should be evaluated on case-by-case basis by the Investigator in consultation with the MMs.

Sponsors & Collaborators

• Lumos Pharma: lead

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

Central Study Contacts

Lumos Pharma

CONTACT

515-598-2921; clinical.trials@lumos-pharma.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 5, 2025
• First Posted: August 19, 2025
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): February 1, 2030
• Study Completion (Estimated): February 1, 2030
• Last Updated: August 19, 2025

Record last verified: 2025-08