Safety, tolerability and PK of NIO752 in Patients With PSP
Research type
Research Study
Full title
A Randomized, Participant, Investigator and Sponsor Blinded, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of Intrathecally Administered NIO752 in Patients With Progressive Supranuclear Palsy
IRAS ID
285103
Contact name
Pierre Zimmermann
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2019-004398-26
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 10 months, 10 days
Research summary
The purpose of this study is to find out if the treatment NIO752 is safe and tolerable in people who have Progressive Supranuclear Palsy (PSP). This study will also look at how NIO752 distributes in the body and how fast it leaves the body.
Potential effects of NIO752 on PSP will be measured using several methods, including brain magnetic resonance imaging (MRI; a scan that uses a magnetic field to look inside the brain), quality of life questionnaires and physical and neurological exams.
The study is planned to take place in 5 countries (UK, Canada, Germany, Italy, USA), with 5 hospital sites in the UK. Approximately 64 participants with Progressive Supranuclear Palsy between the ages of 40 and 75 years old (inclusive) will be asked to join this study.
This study will test 6 different doses of NIO752 and placebo (“dummy treatment”). The placebo looks like NIO752 but does not have any active drug in it. In the study, 3 out of 4 participants will receive NIO752 treatments and 1 out of 4 will receive placebo treatments. Neither the participants nor their Study Doctor will know what treatment they are getting.
The study treatment is given by injection into the spine (known as intrathecal injection). Patients will be treated for 3 months: 4 injections, 4 weeks apart. After this, patients will be followed up for 9 months with 6 study visits 4-10 weeks apart. Total study duration for each patient will be 12 months.
REC name
HSC REC B
REC reference
20/NI/0146
Date of REC Opinion
24 Dec 2020
REC opinion
Further Information Favourable Opinion