Growth Hormone Deficiency (GHD)
Conditions
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.
Interventions
DRUGLUM-201
1.6 mg/kg/day, administered orally once daily
Sponsors
Lumos Pharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
4 Years to 12 Years
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Contacts
CONTACTLumos Pharma
Outcome results
None listed