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siRNA

Pozelimab + Cemdisiran for Myasthenia Gravis (NIMBLE Trial)

Phase 3

Recruiting

Research Sponsored by Regeneron Pharmaceuticals

Eligibility Criteria Checklist

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

Must have

Currently receiving an immunosuppressive therapy (IST) for MG, or documented reason why the patient is not taking an IST per investigator

Patient with documented diagnosis of myasthenia gravis (MG) based on medical history and supported by previous evaluations as described in the protocol

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through study duration, approximate 172 weeks

Awards & highlights

NIMBLE Trial Summary

This trial is testing a combination of two drugs, pozelimab and cemdisiran, to see if it improves daily functioning in people with myasthenia gravis. The secondary objectives are to see if the combination improves symptoms of myasthenia gravis, muscle strength, quality of life, and more. The trial will also assess the safety and side effects of the combination.

Who is the study for?

Adults over 18 with a confirmed diagnosis of myasthenia gravis (MG), specifically those who have MGFA Class II to IVa and an MG-ADL score ≥6. Participants should be on or have a reason for not using acetylcholinesterase inhibitors or immunosuppressive therapy, without plans to change dosages during the trial. Exclusions include MuSK-only positive patients, recent thymectomy, certain cancer histories, HIV infection, no recent meningococcal vaccination unless planned before treatment starts.Check my eligibility

What is being tested?

The study is testing how well Pozelimab combined with Cemdisiran improves daily functioning affected by MG symptoms compared to each drug alone and placebo. It also looks at muscle strength, quality of life improvements, safety/tolerability of the drugs, their levels in blood/plasma and effects on immune response.See study design

What are the potential side effects?

While specific side effects are not listed here, participants will be monitored for general safety and tolerability which typically includes tracking any adverse reactions like allergic responses or infusion-related issues that may arise from the treatments.

NIMBLE Trial Eligibility Criteria

Inclusion Criteria

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

Select...

I am on immunosuppressive therapy for myasthenia gravis or have a reason for not taking it.

Select...

I have been diagnosed with myasthenia gravis based on my medical history.

Select...

My myasthenia gravis is moderate to severe but not requiring a ventilator.

NIMBLE Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through study duration, approximate 172 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through study duration, approximate 172 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and through study duration, approximate 172 weeks 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 Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score

Secondary outcome measures

Change from baseline in Myasthenia Gravis Quality of Life (MG QOL15r) total score

Change from baseline in Quantitative Myasthenia Gravis (QMG) score

Change from baseline in the Myasthenia Gravis Composite (MGC) total score

+15 more

NIMBLE Trial Design

4Treatment groups

Experimental Treatment

Group I: Group 4Experimental Treatment1 Intervention

Pozelimab monotherapy in DBTP followed by combination in ETP and OLTP

Group II: Group 3Experimental Treatment1 Intervention

Cemdisiran throughout the study

Group III: Group 2Experimental Treatment1 Intervention

Combination regimen throughout the study

Group IV: Group 1Experimental Treatment3 Interventions

Placebo in DBTP; Re-randomized to Combination or Cemdisiran in ETP and OLTP

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pozelimab

2020

Completed Phase 2

~120

Cemdisiran

2020

Completed Phase 2

~80

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Regeneron PharmaceuticalsLead Sponsor

619 Previous Clinical Trials

380,020 Total Patients Enrolled

Clinical Trial ManagementStudy DirectorRegeneron Pharmaceuticals

261 Previous Clinical Trials

250,829 Total Patients Enrolled

Media Library

Cemdisiran (siRNA) Clinical Trial Eligibility Overview. Trial Name: NCT05070858 — Phase 3

Myasthenia Gravis Research Study Groups: Group 1, Group 2, Group 3, Group 4

Myasthenia Gravis Clinical Trial 2023: Cemdisiran Highlights & Side Effects. Trial Name: NCT05070858 — Phase 3

Cemdisiran (siRNA) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05070858 — Phase 3

Frequently Asked Questions

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

Are new participants currently being recruited for this experiment?

"That is correct, the online clinicaltrial.gov registry reflects that this study is ongoing and actively recruiting patients. The 235 participants are being recruited from 19 different locations. The study was originally posted on December 14th, 2021 and was last updated on October 31st, 2022."

Answered by AI

How many hospitals or other medical facilities are participating in this clinical trial?

"There are a total of 19 different clinical trial sites. Some exemplar locations are Philadelphia, London, and Phoenix. If you are selected for this trial, it would be convenient for you to choose a location near to reduce travel requirements."

Answered by AI

How many individuals are currently involved in this research?

"Yes, according to the information available on clinicaltrials.gov, this trial is still looking for patients. The study was created on December 14th, 2021 and was last updated on October 31st, 2022. There are a total of 19 sites enrolling 235 patients."

Answered by AI

What is the efficacy of Pozelimab in regards to other medical treatments?

"Pozelimab was first trialled in 2019, and there have been a total of 18,245 completed clinical trials since then. There are currently 7 ongoing studies that are actively recruiting participants, most of which are based in Philadelphia, Pennsylvania."

Answered by AI

Does this research build on pre-existing investigations?

"Pozelimab is being trialed in 43 cities, across 21 nations, with 7 active studies as of now. The first trial began in 2019 and completed Phase 2 drug approval that same year. Alnylam Pharmaceuticals sponsored the study, which had 31 participants. Since 2019, a total of 18,245 trials have been completed."

Answered by AI

When will Pozelimab likely receive FDA approval?

"Pozelimab's safety is often estimated to be a 3 because it has completed Phase 3 trials. This means that not only is there some data supporting its efficacy, but multiple rounds of data have been collected to support its safety."

Answered by AI

Who else is applying?

What site did they apply to?

Penn Medicine University City

What portion of applicants met pre-screening criteria?

Did not meet criteria

Why did patients apply to this trial?

I am considered Triple Seronegative been on IVIG infusions for almost 3 yrs. I recieve 45g daily for 3 days every 3 weeks with no real improvement on the MG-ADL scale (it stays in the 11-14point range). My last Neurologist wanted to put me on Cellcept but would not until I got my Shingles Vaccine.. Due to being under the after of 50+ my insurance would not cover the price of the vaccine. That is the only reason I have not been able to give any kind of immunosuppressants a try. My current Neurologist told me to reach out to other avenues to try and get more help and answers with treating my Myasthenia Gravis. Hoping one day the perfect clinical trial will be starting in my area that include seronegative paitents like me!

PatientReceived no prior treatments

Recent research and studies

clinicaltrials.govStudy

A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis

2021. "A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05070858.

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