Duchenne Muscular Dystrophy (DMD)
Conditions
Brief summary
The purpose of this trial is to assess the safety, tolerability, and PK of 3 doses of GRT6019 in healthy male participants. This Phase I trial will be a multiple dose trial in healthy male participants with administration of GRT6019 in 3 cohorts. For each participant, the trial consists of a Screening Period of up to 28 days, a 4 week Treatment Period (including a 2-week clinic stay and 2 weeks in an outpatient setting), and a 5 week Follow-up Period.
Detailed description
There are 8 participants planned per cohort to ensure sufficient sample size for a reliable estimate of PK parameters.
Interventions
DRUGGRT6019
Multiple dose (MD)
Sponsors
Grünenthal GmbH
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Cohorts 1 and 2 will be dosed in parallel, followed by Cohort 3 sequentially
Eligibility
Sex/Gender
MALE
Age
18 Years to 55 Years
Healthy volunteers
Yes
Inclusion criteria
Main Inclusion Criteria: 1. Participant must be male, 18 to 55 years of age (inclusive) at the time of signing the informed consent form and affiliated to the social security system. 2. Participant must be capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. 3. Participant must sign the informed consent form before any trial-related assessments. 4. The participant is in good health as determined by the medical history, physical examination, 12-lead ECG, vital signs (heart rate, respiratory rate, systolic and diastolic blood pressure \[BP\]), body temperature, and clinical laboratory parameters (clinical chemistry, hematology, coagulation, and urinalysis) without clinically relevant (per investigator judgement) deviations from reference ranges unless further specified in the
Exclusion criteria
. The population of this clinical trial is healthy volunteer participants. Main
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with Adverse Events (AEs) | Through study completion, an average of 4 Weeks | Assessment of safety and tolerability of GRT6019 following 4 weeks of treatment |
| Number of participants with Serious Adverse Events (SAEs) | Through study completion, an average of 4 Weeks | Assessment of safety and tolerability of GRT6019 following 4 weeks of treatment |
| Number of participants with AEs leading to discontinuation | Through study completion, an average of 4 Weeks | Assessment of safety and tolerability of GRT6019 following 4 weeks of treatment |
| Number of participants with AEs related to investigational medicinal product [IMP] | Through study completion, an average of 4 Weeks | Assessment of safety and tolerability of GRT6019 following 4 weeks of treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Area under the concentration-time curve over the dosing interval (AUCtau) | Following the first dose (Day 1), then at Day 14, and after the last dose (Day 28) | Assessment of the pharmacokinetics (PK) of GRT6019 following 4 weeks of treatment, including dose proportionality assessment and investigation of steady state attainment |
| Maximum observed concentration (Cmax) | Following the first dose (Day 1), then at Day 14, and after the last dose (Day 28) | Assessment of the pharmacokinetics (PK) of GRT6019 following 4 weeks of treatment, including dose proportionality assessment and investigation of steady state attainment |
Countries
France
Outcome results
None listed