Angelman syndrome
Conditions
Interventions
The volunteer will be given one single dose of 10 mg RO7248824 mixed with a
maximum of 100 µg of the labeled form of RO7248824 through an intrathecal
injection. The dose of tracer may be adapted dur
Sponsors
F. Hoffmann-La Roche Ltd
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: 1. Able and willing to provide written informed consent and to comply with the study protocol according to ICH and local regulations 2. Aged 25 to 55 years at the time of dosing 3. Overtly healthy (defined by absence of evidence of any active or chronic disease) as determined by medical evaluation including: - A detailed medical and surgical history - A complete physical and neurological examination - Vital signs - 12-lead ECG - Hematology - Coagulation - Blood chemistry - Serology and urinalysis 4. Fluent in the language of the Investigator and study staff, and able to communicate with the study staff 5. Body mass index of 18 to 30 kg/m2 at screening 6. Male participants only who, for 3 months after the dosing of RO7248824, agree to: - Remain abstinent or use contraceptive barrier measures such as a condom, with a female partner of childbearing potential, or pregnant female partner, to avoid exposing the embryo - Refrain from donating sperm The reliability of sexual abstinence for male enrollment eligibility needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or post-ovulation methods) and withdrawal are not acceptable methods of preventing drug exposure.
Exclusion criteria
Exclusion criteria: 1. Any condition or disease detected during the medical interview/physical examination that would render the participant unsuitable for the study, place the participant at undue risk or interfere with the ability of the participant to complete the study, as determined by the Investigator 2. History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, or neurological disorders capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study treatment; or interfering with the interpretation of data 3. History or presence of clinically significant cardiovascular disease in the opinion of the Investigator, including, but not limited to: left ventricular ejection fraction 220 ms, QTcF > 450 ms) 5. Uncontrolled arrhythmias or history of clinically significant arrhythmias including: ventricular arrhythmias or risk factors for ventricular arrhythmias (such as clinically significant electrolyte abnormalities (e.g., hypokalemia, hypomagnesemia, hypocalcemia); second- or third-degree atrioventricular block; family history of sudden unexplained death or long QT syndrome; congenital hypertrophic or dilated cardiomyopathy For the complete overview see the protocol
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To quantify the [89Zr]DFO-RO7248824 distribution within the central nervous system (CNS) following a single intrathecal (IT) administration of [89Zr]DFO-RO7248824 mixed with 10 mg RO7248824 | — |
Secondary
| Measure | Time frame |
|---|---|
| To assess the safety and tolerability of single IT administrations of [89Zr]DFO-RO7248824 mixed with 10 mg RO7248824 | — |
Countries
Netherlands
Outcome results
None listed