A Phase III, Randomized, Placebo-controlled, Parallel Group, Double-blind Study to Evaluate the Efficacy and Safety of NIO752 in Participants With Progressive Supranuclear Palsy Followed by an Open Label Extension
Novartis Pharmaceuticals
Summary
This Phase III study is intended to evaluate the efficacy and safety of NIO752 in participants with Progressive Supranuclear Palsy (PSP). Eligible participants will be randomized to receive either NIO752 or placebo followed by an open-label extension.
Description
This is a randomized, double-blind, placebo-controlled, parallel group, study to evaluate the efficacy of NIO752 in participants with Progressive Supranuclear Palsy followed by an open-label extension (OLE). The participants with or without symptomatic therapy will be randomly allocated to either NIO752 or placebo treatment in a 2:1 randomization ratio. Upon completion of the core double-blind treatment period, participants will be offered to continue with NIO752 treatment in the OLE.
Eligibility
- Age range
- 41–81 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Signed informed consent must be obtained prior to participation in the study. 2. Male or female participants, age between 41-81 yrs inclusive. 3. Diagnosis of mild-moderate, probable/possible PSP Richardson syndrome as per MDS-PSP 2017 criteria with symptoms onset \< 5 years. 4. PSPRS total score less than 40 at Baseline. 5. Reliable study partner such as spouse, sibling, close friend, or caregiver able and willing to provide accurate information (including clinical symptoms and medical history) about the participant and to participate in study visits and informant-base…
Interventions
- OtherNIO752
Solution of antisense oligonucleotide.
- DrugPlacebo
Placebo solution
Location
- CenExcel Rocky Mtn Clin ResearchEnglewood, Colorado