Phase 3 Long Term Safety Extension Study of LUM-201 in Children With Growth Hormone Deficiency
- Registration Number
- NCT07129759
- Lead Sponsor
- Lumos Pharma
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 150
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description LUM-201 LUM-201 -
- Primary Outcome Measures
Name Time Method To evaluate the long-term safety and tolerability of LUM-201 Day 1 - Month 36 * 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.
- Secondary Outcome Measures
Name Time Method To evaluate the long-term effect of LUM-201 on growth. Day 1 - Month 36 * Annual height velocity.
* Annual change in height standard deviation score (SDS).
* Annual change in body mass index (BMI).
* Annual change in BMI SDS.
* Annual change in bone age (BA)/chronological age (CA) ratio.To evaluate the long-term effect of LUM-201 on pharmacodynamic (PD) markers. Day 1 - Month 36 * Annual change in insulin-like growth factor (IGF)-1 SDS.
* Annual change in insulin-like growth binding protein 3 (IGFBP-3) SDS.