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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.
Between 40 to 75 years old (inclusive)
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)
Awards & highlights

Study Summary

This trial is investigating a new drug for PSP, a degenerative brain disease. The drug will be given to a small group of people to see if it is safe and effective.

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.Check my eligibility
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.See study design
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 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 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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My medication doses have been stable for the last 30 days and will remain so.
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I have someone over 18 who can come with me to my study visits and knows about my health condition.
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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.

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)
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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
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

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,851 Previous Clinical Trials
4,197,530 Total Patients Enrolled

Media Library

NIO752 (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT04539041 — Phase 1
Progressive Supranuclear Palsy Research Study Groups: Cohort A NIO752, Cohort B NIO752, Placebo, Cohort C NIO752, Cohort D NIO752, Cohort E NIO752
Progressive Supranuclear Palsy Clinical Trial 2023: NIO752 Highlights & Side Effects. Trial Name: NCT04539041 — Phase 1
NIO752 (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04539041 — Phase 1
Progressive Supranuclear Palsy Patient Testimony for trial: Trial Name: NCT04539041 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has the Federal Drug Administration approved antisense oligonucleotide for public use?

"As this is a Phase 1 trial, the safety of antisense oligonucleotides has been appraised as low or 1. This suggests that while there is limited data to support efficacy and safety, it still requires clinical trials for further evaluation."

Answered by AI

Is it possible to participate in this medical experiment?

"This clinical trial is recruiting 66 individuals afflicted with paralysis, ranging in age from 40 to 75 years old. It is critical that they meet the following prerequisites: no alterations should be made to their Parkinson's or Alzheimer's medications for at least a month prior and throughout the study; 12 months of postural instability must have been documented within 3 years of disease onset; PSP Richardson Syndrome diagnosis within 5 years and its concomitant scores on MOCA Test >17 and PSPRS <40; vertical supranuclear gaze palsy or reduced velocity of saccades verified ; access to an 18+ year-old reliable participant partner available"

Answered by AI

Is the age cutoff for this trial 18 years or older?

"This clinical trial will accept patients aged 40 to 75 years of age."

Answered by AI

Is the research team actively seeking participants for this experiment?

"Affirmative. Clinicaltrials.gov's data indicates that this clinical trial is currently recruiting patients - the study was initially posted on February 16th 2021 and updated most recently on February 23rd 2022. The entirety of 66 participants will be recruited from 5 different sites."

Answered by AI

What is the current sample size for this experiment?

"Affirmative, the information on clinicaltrials.gov demonstrates that this research is actively seeking enrolment. This medical trial was initially posted in February of 2021 and was most recently revised in late February 2022; it seeks to recruit 66 patients from 5 sites."

Answered by AI

What is the ultimate aim of this research endeavor?

"Across the course of one year, researchers will be measuring a range of primary and secondary outcomes. The former include adverse events and serious adverse events while the latter involve AUCinf in blood plasma (mass x time x volume-1), concentrations of NIO752 in blood plasma, as well as Tmax post first injection."

Answered by AI

Are there copious facilities conducting this exploration in Canada?

"This experiment is currently being conducted in 5 locations, including but not limited to Montreal, Boca Raton and La Jolla. It's beneficial for any potential participants to select the closest site so as to reduce travel commitments if they decide to join the study."

Answered by AI

Who else is applying?

What state do they live in?
California
How old are they?
18 - 65
65+
What site did they apply to?
Novartis Investigative Site
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
1

Why did patients apply to this trial?

Want to take part in helping improve the care for myself and others with this condition.
PatientReceived 2+ prior treatments
As there is currently no cure via conventional medicine, I am hoping to find something that will help my condition.
PatientReceived 2+ prior treatments

How responsive is this trial?

Typically responds via
Email
Most responsive sites:
  1. Novartis Investigative Site: < 48 hours
Average response time
  • < 2 Days
~9 spots leftby Nov 2024