Growth hormone deficiency MedDRA version: 20.0 Level: PT Classification code 10056438 Term: Growth hormone deficiency System Organ Class: 10014698 - Endocrine disorders
Conditions
Sponsors
Lumos Pharma, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable. 2. Must have previously completed 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial. 3. Is eligible for study participation as confirmed by the principal investigator (PI). Are the trial subjects under 18? yes Number of subjects for this age range: 20 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201. 2. 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. 3. 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 (see Appendix 1 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 PI in consultation with the MM. 4. Has been randomized to LUM-201 in the LUM-201-01 trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Assess 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.;Secondary Objective: Assess safety in response to LUM-201 treatment.;Primary end point(s): Growth Endpoints: Growth parameters of efficacy (at Month 3 [visit 3], Month 6 [visit 4] and Month 12 [visit 6]) will be assessed based on: Annualized height velocity, Change in HT-SDS, Change in Weight and Weight SDS, Change in BMI and BMI SDS, Change in Bone Age (Only Month 6 [visit 4]) PD Endpoints: PD will be assessed by GH, IGF-1 and IGFBP-3. Safety Endpoints: Safety of LUM-201 will be assessed by AEs and SAEs, routine laboratory tests (hematology, chemistry panel including liver enzymes and urinalysis), HbA1c, blood glucose, free thyroxine (T4), triiodothyronine;Timepoint(s) of evaluation of this end point: M3, M6, M12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): N/A;Timepoint(s) of evaluation of this end point: N/A | — |
Countries
Australia, Israel, New Zealand, Poland, United States
Contacts
Public ContactRegulatory Affairs, Lumos Pharma
Lumos Pharma, Inc.
Outcome results
None listed