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CAR T-cell Therapy
MuSK-CAART for Myasthenia Gravis
Phase 1
Recruiting
Research Sponsored by Cabaletta Bio
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Positive anti-MuSK antibody test at screening
Confirmed diagnosis of MuSK-type MG with at least 1 prior positive anti-MuSK antibody test
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 36 months
Awards & highlights
Study Summary
This trial is studying an investigational cell therapy to treat a rare, severe disease called MuSK myasthenia gravis, which attacks the neuromuscular junction. If successful, it may lead to complete and durable remission of the disease.
Who is the study for?
This trial is for people with MuSK myasthenia gravis, a muscle weakness disease. Participants must have tested positive for anti-MuSK antibodies and negative for anti-AChR antibodies, with an MG severity Class I to IVa. They can't join if they're on high-dose prednisone, had other autoimmune treatments recently, received investigational MG treatments in the last 12 weeks or rituximab in the past year.Check my eligibility
What is being tested?
The study tests different doses of MuSK-CAART alone or combined with cyclophosphamide (CY) and fludarabine (FLU). It's an open-label phase 1 trial aiming to see if this cell therapy can cause remission in patients with active MuSK myasthenia gravis.See study design
What are the potential side effects?
Potential side effects may include immune system reactions due to cell therapy, as well as typical chemotherapy-related issues like nausea, fatigue, hair loss from CY and FLU. The exact side effects of MuSK-CAART are being studied.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I tested positive for anti-MuSK antibodies.
Select...
I have been diagnosed with MuSK myasthenia gravis and tested positive for anti-MuSK antibodies.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 36 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 36 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Adverse events
Secondary outcome measures
Cells
Body Weight Changes
Percent of CAAR-transduced cells
+1 moreOther outcome measures
Measurement of Clinical Symptoms using MG-ADL
Measurement of Clinical Symptoms using MGC
Measurement of Clinical Symptoms using QMG
+2 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: MuSK-CAARTExperimental Treatment1 Intervention
Cohort A: Infusion of MuSK-CAART at various dose levels with or without pre-treatment (6 groups planned).
Cohort B: Infusion of MuSK-CAART at the dose regimen selected from Part A.
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Who is running the clinical trial?
Cabaletta BioLead Sponsor
5 Previous Clinical Trials
93 Total Patients Enrolled
1 Trials studying Myasthenia Gravis
12 Patients Enrolled for Myasthenia Gravis
Medical DirectorStudy ChairCabaletta Bio
2,777 Previous Clinical Trials
8,063,716 Total Patients Enrolled
3 Trials studying Myasthenia Gravis
286 Patients Enrolled for Myasthenia Gravis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have previously tested negative for anti-AChR antibodies.Your myasthenia gravis is classified as Class I to IVa according to the MGFA Clinical Classification.I have not received Rituximab in the last 12 months.I tested positive for anti-MuSK antibodies.You have received an experimental treatment for myasthenia gravis in the last 12 weeks.Your immune system's white blood cell count is very low.I am taking more than a small dose of prednisone daily.I am on immunosuppressive therapy for an autoimmune disorder.I have been diagnosed with MuSK myasthenia gravis and tested positive for anti-MuSK antibodies.
Research Study Groups:
This trial has the following groups:- Group 1: MuSK-CAART
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What potential dangers are associated with MuSK-CAART treatment?
"Based on the data that is available, our team at Power has assigned MuSK-CAART a score of 1 due to its limited clinical research backing safety and efficacy."
Answered by AI
Is the recruitment phase of this research open to volunteers?
"Affirmative. The clinicaltrials.gov database informs that this research is currently recruiting volunteers, which began on December 1st 2022 and was last modified on November 23rd 202. 24 participants are required between two trial sites."
Answered by AI
How many participants are currently allowed to enroll in this investigation?
"Affirmative. According to the information available via clinicaltrials.gov, this research is currently gathering participants; it was first made visible on December 1st 2022 and has been refreshed on November 23rd 2022. 24 individuals are being recruited from two separate trial sites."
Answered by AI
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