What side effects are associated with this type of intervention?

Common treatments for Progressive Supranuclear Palsy (PSP) often include medications aimed at alleviating symptoms, such as levodopa, amantadine, and antidepressants. These treatments can have side effects like nausea, dizziness, orthostatic hypotension, and hallucinations. Investigational drugs like NIO752, which are aimed at modifying disease progression, may have side effects that are not yet fully understood but could include similar adverse effects as other neurodegenerative treatments, such as gastrointestinal issues, sleep disturbances, and potential neuropsychiatric symptoms. It is important to monitor these side effects closely in clinical trials.

What prior FDA approvals exist?

There are no FDA-approved treatments specifically for Progressive Supranuclear Palsy (PSP) that modify the disease progression. Current management of PSP is symptomatic, focusing on alleviating motor symptoms and other complications. Investigational drugs like NIO752 are being studied for their potential to modify disease progression or symptoms in PSP, but as of now, no such treatments have received FDA approval.

What other types of research exist?

Promising alternatives to NIO752 for PSP patients include compounds targeting tau protein aggregation and neuroinflammation. Specifically, drugs in Phase 2 trials such as tau aggregation inhibitors and anti-inflammatory agents are being actively investigated. Additionally, compounds like davunetide and tideglusib, which have shown potential in modifying disease progression in tauopathies, are also considered promising. Patients should discuss these options with their healthcare provider to determine the most appropriate treatment based on the latest clinical data.

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Antisense Oligonucleotide

Antisense Oligonucleotide for Progressive Supranuclear Palsy

Phase 1

Waitlist Available

Research Sponsored by Novartis Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

If the participant is receiving levodopa/carbidopa, levodopa/benserazide, a dopamine agonist, catechol-o-methyltransferase (COMT) inhibitor, rasagiline, CoQ10 or other Parkinson's medications, acetylcholinesterase inhibitors, antipsychotics, memantine, or other non-tau modifying Alzheimer's medication the dose must have been stable for at least 30 days prior to the screening visit and must remain stable for the duration of the study. No such medication can be initiated during the study.

Vertical supranuclear gaze palsy, or reduced velocity of vertical saccade

Must not have

Evidence of motor neuron disease, or any other neurological disease that could explain symptoms

History of post-lumbar-puncture headache of moderate or severe intensity and/or blood patch

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline up to approximately 1 year (3-month treatment plus 9-month follow-up or 9-month treatment plus 3-month follow-up)

Summary

This trial tests a new drug, NIO752, for people with progressive supranuclear palsy (PSP). The drug is injected into the spinal fluid to help it reach the brain. The goal is to find out if NIO752 can better manage PSP symptoms.

Who is the study for?

This trial is for adults aged 40-75 with Progressive Supranuclear Palsy (PSP) diagnosed within the last 5 years, able to walk independently or with minimal assistance. Participants must have a history of postural instability or falls and score below certain thresholds on PSP and cognitive scales. They need a reliable study partner and can't be in nursing care, recently hospitalized, or show significant benefit from levodopa.

What is being tested?

The trial tests multiple doses of NIO752, an antisense oligonucleotide against placebo in people with PSP. It's double-blind meaning neither participants nor researchers know who gets the real treatment versus placebo. The goal is to assess safety, tolerability, and how the body processes the drug.

What are the potential side effects?

Potential side effects are not detailed here but may include reactions related to lumbar puncture procedures such as headache or back pain; other risks could involve typical drug-related adverse events like nausea, fatigue, allergic reactions but specifics will depend on further study results.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My Parkinson's or Alzheimer's medication dose has been stable for at least 30 days.

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I have difficulty moving my eyes up or down quickly.

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I can safely have lumbar punctures and blood tests.

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I was diagnosed with PSP less than 5 years ago, my condition is likely PSP Richardson syndrome, and my health scores are within a specific range.

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I have had balance problems or falls for at least a year within the first three years of my disease.

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I was diagnosed with PSP less than 5 years ago, have probable PSP Richardson syndrome, a PSPRS score under 40, and a MOCA score over 17.

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I can have spinal taps and blood tests.

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I can walk on my own or with little help.

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I am between 40 and 75 years old.

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I have difficulty moving my eyes up or down quickly.

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I can walk on my own or with little help.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a diagnosed neurological condition that could explain my symptoms.

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I've had severe headaches or needed a blood patch after a lumbar puncture.

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I have not been hospitalized for a major procedure under general anesthesia in the last 12 weeks and have no such plans during the study.

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I have seen significant improvement with levodopa treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up to approximately 1 year (3-month treatment plus 9-month follow-up or 9-month treatment plus 3-month follow-up)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline up to approximately 1 year (3-month treatment plus 9-month follow-up or 9-month treatment plus 3-month follow-up)

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to approximately 1 year (3-month treatment plus 9-month follow-up or 9-month treatment plus 3-month follow-up) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in severity scores for Columbia-Suicide Severity Rating Scale (C-SSRS)

Levels of infection indicators in Cerebrospinal fluid (CSF)

Number of adverse events and serious adverse events

Secondary study objectives

AUCinf in blood plasma

Plasma

Cmax, Ctrough in blood plasma

+3 more

Trial Design

6Treatment groups

Experimental Treatment

Placebo Group

Group I: Cohort E NIO752Experimental Treatment1 Intervention

4 injections of NIO752 at dose E

Group II: Cohort D NIO752Experimental Treatment1 Intervention

4 injections of NIO752 at dose D

Group III: Cohort C NIO752Experimental Treatment1 Intervention

4 injections of NIO752 at dose C

Group IV: Cohort B NIO752Experimental Treatment1 Intervention

4 injections of NIO752 at dose B

Group V: Cohort A NIO752Experimental Treatment1 Intervention

4 injections of NIO752 at dose A

Group VI: PlaceboPlacebo Group1 Intervention

4 injections of placebo

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

antisense oligonucleotide

2021

Completed Phase 1

~60

0 / 15Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Progressive Supranuclear Palsy (PSP) include medications like levodopa, amantadine, and investigational drugs such as NIO752. Levodopa works by replenishing dopamine levels in the brain, which can help improve motor symptoms. Amantadine acts as an NMDA receptor antagonist, which may help reduce motor symptoms and dyskinesias. NIO752, an investigational drug, is being studied for its potential to modify disease progression or alleviate symptoms by targeting specific pathways involved in PSP. These treatments are crucial for PSP patients as they aim to manage symptoms, improve quality of life, and potentially slow disease progression.

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