Growth Hormone Deficiency
Conditions
Keywords
LUM-201, PGHD, Height, Oral
Brief summary
This is a multi-national trial. The goals of the trial are to study 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.
Detailed description
This trial will have one screening/baseline visit with tests to assess if subjects are eligible to start study therapy. The screening/baseline visit can coincide with the Month 12 visit in the LUM-201-01 trial. Subjects will receive the same dose of LUM-201 as a daily oral dose each morning. LUM-201 will be adjusted for body weight increments at each clinic visit, as applicable. The trial consists of 12 months of treatment. After screening, subjects will return to the clinic for 5 visits. During four of these clinic visits, safety laboratory samples will be collected.
Interventions
DRUGLUM-201
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
3 Years to 13 Years
Healthy volunteers
No
Inclusion criteria
* Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable. * Must have previously completed 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial. * Is eligible for study participation as confirmed by the principal investigator (PI)
Exclusion criteria
* Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201. * 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. * 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Annualized Height Velocity (AHV) Measured as Standing Height With Stadiometer | Day 1 to Month 12 | Mean change from day 1 to month 12, Annualized Height Velocity (AHV) Height is measured in triplicate, by a calibrated stadiometer and taking the mean value AVH equation is (h2-h1/t2-t1)\*365.25; h1 = height measured at day 1 h2 = height measured at month 12 t1 = day 1 t2 = month 12 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Occurrence of Adverse Events in Children With Growth Hormone Deficiency (GHD) | Day 1 to Month 12 | Number of events |
| Growth Hormone (GH) Values | Day 1 to Month 12 | Mean GH Values change from Day 1 to month 12 (Pre-Dose and 1 hour Post-Dose) |
| Insulin-Like Growth Factor 1 (IGF-1) Values | Day 1 to Month 12 | Mean change from Day 1 to month 12 (IGF-1 Values Pre-Dose) |
| Insulin-Like Growth Factor Binding Protein 3 (IGFBP-3) Values | Day 1 to Month 12 | Mean change from day 1 to month 12 (IGFBP-3 Values Pre-Dose) |
| Height (HT) Standard Deviation Score (SDS) | Day 1 to Month 12 | Mean change from day 1 to month 12 in HT-SDS |
| Change in Weight | Day 1 to Month 12 | Mean change in Weight from day 1 to month 12 |
| Change in Weight - Standard Deviation Score (SDS) | Day 1 to Month 12 | Mean change in Weight-SDS from day 1 to month 12 |
| Change in Body Mass Index (BMI) | Day 1 to Month 12 | Mean change from day 1 to month 12 in BMI |
| Change in Body Mass Index (BMI) - Standard Deviation Score (SDS) | Day 1 to Month 12 | Mean change from Day 1 to month 12 in BMI-SDS |
| Change in Bone Age | Month 6 | Mean change from day 1 to month 6 in bone age, measured by X-ray of left hand and wrist using Greulich \& Pyle atlas |
| Insulin-Like Growth Factor 1 (IGF-1) - Standard Deviation Score (SDS) | Day 1 to Month 12 | Mean Change from day 1 to month 12 for IGF-1 - SDS Pre-dose Values |
Countries
Poland, United States
Participant flow
Pre-assignment details
Each screened subject will enter the Subject Number previously assigned in the LUM-201-01 trial, at the Screening/Baseline visit (visit 1). Subject Numbers will not be reassigned. All subjects will receive a single oral 3.2 mg/kg dose of LUM-201 at the Screening/Baseline visit (visit 1) which will include measurement of GH concentrations at baseline and 60 ± 10 minutes after dosing. All subjects will commence their dosing at the Screening/Baseline visit (visit 1).
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 15 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 102 months STANDARD_DEVIATION 25.5 |
| Baseline Height-Standard Deviation Score (SDS) | -1.32492 Height-SDS STANDARD_DEVIATION 0.37944 |
| Baseline Insulin-like growth factor 1 (IGF-1) SDS | 0.9343 SDS STANDARD_DEVIATION 1.1716 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 14 Participants |
| Region of Enrollment Poland | 6 participants |
| Region of Enrollment United States | 9 participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 9 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 15 |
| other Total, other adverse events | 13 / 15 |
| serious Total, serious adverse events | 0 / 15 |
Outcome results
None listed