Early Phase 1 Myasthenia Gravis Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

General criteria (all participants):

• People who are willing to take part and sign a consent form agreeing to participate.
• People aged 18 to 70 years old, of any gender.
• People whose liver function test results are within an acceptable range (specific blood test values required — confirm with trial site).
• People whose kidney function test results are within an acceptable range (specific blood test values required — confirm with trial site).
• People whose blood cell counts (white cells, red cells, platelets, and lymphocytes) are within acceptable ranges (specific values required — confirm with trial site).
• People whose blood clotting test results are within an acceptable range (confirm with trial site).
• People whose blood oxygen level is at least 92% when breathing normal room air at rest.
• People whose heart pumping function, measured by an ultrasound of the heart, shows the heart is pumping at least 50% of its capacity.
• Women who could become pregnant must have a negative pregnancy test at the screening stage.
• Women who could become pregnant must use effective contraception from at least 28 days before a blood cell collection procedure until 12 months after receiving the study treatment. Men who could father a child must use effective barrier contraception during the same period and must not donate sperm throughout the study.

Additional criteria for people with Multiple Sclerosis (MS):

• People formally diagnosed by a qualified neurologist with Relapsing-Remitting MS (RRMS), Primary Progressive MS (PPMS), or Secondary Progressive MS (SPMS), based on the 2017 McDonald Criteria, with supporting documentation provided.
• People whose MS-related disability score (EDSS) falls between 3.0 and 7.5 (confirm with trial site).
• People who have had a brain MRI within the past 12 months showing features consistent with MS activity, including certain types of lesions (confirm with trial site for required scan sequences).
• People who have had a spinal fluid test showing either an elevated IgG index or the presence of certain immune proteins called oligoclonal bands — either from a previous test or done during the screening period.
• People who have been on a high-strength MS disease-modifying treatment for at least 6 months and whose MS has still worsened during that time — either through a confirmed relapse or measurable disability progression (confirm with trial site for specific medications that qualify).
• For RRMS: People with at least one confirmed relapse in the past year, or at least two confirmed relapses in the past two years, or active lesions on a recent MRI, all supported by medical records.
• For PPMS or SPMS: People with documented evidence of disability worsening within the past two years, supported by medical records.

Additional criteria for people with Myasthenia Gravis (MG):

• People diagnosed with generalised myasthenia gravis (gMG) according to recognised international guidelines (such as the 2020 MGFA Guidelines).
• People classified as MGFA Class II, III, or IV based on the standard MG severity classification system (confirm with trial site).
• People who have tested positive for at least one of three specific antibodies associated with MG (AChR-Ab, MuSK-Ab, or LRP4-Ab) — either at screening or based on previous documented test results.
• People who score 6 or more on the MG Activities of Daily Living scale, where symptoms affecting the eyes make up less than half of that score.
• People who score 8 or more on the Quantitative MG score, with at least 4 individual items each scoring 2 or more (confirm with trial site).
• People who have previously been treated with at least one immunosuppressant medication or a biologic agent for MG, with supporting medical documentation.
• People who have been on a stable MG treatment for at least 3 months and whose condition has still worsened — as shown by increasing scores on standardised MG scales, or a need for higher medication doses, hospitalisation, or emergency treatment.

Additional criteria for people with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP):

• People diagnosed with progressive or relapsing CIDP, or possible CIDP, according to 2021 international neurology guidelines, with supporting evidence such as nerve conduction test results, elevated spinal fluid protein levels, MRI findings, or nerve biopsy results (confirm with trial site).
• People who score 2 or more on the CIDP Disease Activity Status scale at screening.
• People who score 2 or more on the INCAT disability scale at screening, with specific scoring requirements depending on whether the score is 2 or higher (confirm with trial site for details).
• People who have been treated for at least 3 months with at least one of the following — intravenous immunoglobulin (IVIG), oral prednisone, plasma exchange, or an FcRn blocker (such as efgartigimod) — and whose condition either has not improved sufficiently or who had to stop treatment due to side effects (confirm with trial site for specific dose and course requirements).
• People who, if currently taking steroids or immunosuppressant medications, have been on a stable dose for at least 2 weeks before screening.

Additional criteria for people with Autoimmune Encephalitis (AE):

• People clinically diagnosed with autoimmune encephalitis according to 2016 international diagnostic criteria, who have tested positive for at least one relevant autoantibody, whose symptoms have not been adequately controlled or who could not tolerate treatment with steroids and at least one immunosuppressant or immune-modifying therapy (including anti-CD20 antibody), who have had an autoimmune encephalitis episode within 3 months before signing the consent form, and who meet a specific disability threshold on either the modified Rankin Scale (score of 2 or more) or the CASE scale (score of 4 or more) at screening.

Who may not be able to join:

• People whose primary diagnosis is a different autoimmune disease that the treating doctor believes could interfere with evaluating this study's results.
• People who have another significant brain or nervous system condition, such as a history of stroke, epilepsy, seizures, dementia, Parkinson's disease, severe brain injury, or psychosis.
• People who have previously had a bone marrow, stem cell, or solid organ transplant (such as kidney, lung, heart, or liver), or who are planning to have one.
• MG patients who have had an uncontrolled myasthenic crisis within 2 weeks before screening.
• CIDP patients whose condition affects only sensation (pure sensory CIDP).
• People who have had a significant heart problem within the past 12 months, such as severe heart failure, a heart attack, unstable chest pain, or uncontrolled irregular heart rhythms.
• People who have significant heart or lung complications at the time of screening that the treating doctor considers would make participation unsafe.
• People with severe asthma or severe chronic obstructive pulmonary disease (COPD). (People with mild or moderate asthma or COPD who are on stable treatment may still be considered — confirm with trial site.)
• People who have been diagnosed with cancer in the past 5 years, with an exception for certain fully treated skin cancers or very early-stage cancers with no remaining disease (confirm with trial site).
• Women who are pregnant or breastfeeding.
• People with a history of repeated serious infections requiring hospitalisation and intravenous antibiotics — for example, three or more infections of the same type within the past year.
• People who have an active infection requiring systemic treatment (such as pneumonia or tuberculosis) within 2 weeks before the lymphodepletion procedure used in this study.
• People who test positive for hepatitis B (in certain combinations of test results), hepatitis C, HIV, or syphilis.
• People who have received a live vaccine within 4 weeks before the lymphodepletion procedure, or who plan to receive a live vaccine during the study.
• People who have been taking high-dose steroids (prednisone 60 mg per day or equivalent) within 4 weeks before the lymphodepletion procedure, or who are unable to reduce their steroid dose to 20 mg per day or less within 3 days before that procedure.
• People who are unable to gradually reduce or stop certain background medications before the lymphodepletion procedure, as specified in the study protocol (confirm with trial site).
• People who have received plasma exchange, immunoadsorption, or intravenous immunoglobulin (IVIG) within 4 weeks before screening.
• People with a known history of allergy or intolerance to a class of medications called calcineurin inhibitors.
• People who have needed kidney dialysis or similar kidney replacement therapy within the past 3 months, or who are expected to need it during the study.
• People with a history of drug or alcohol misuse within the past year.
• People with a history of suicidal thoughts within the past 6 months, or any suicidal behaviour within the past 12 months, where the treating doctor considers there to be a significant risk.
• People who have taken part in another clinical trial and received a different experimental medication within the past 4 weeks or within 5 half-lives of that drug (whichever is longer).
• People with a known severe allergic reaction or life-threatening response to any component of the study treatment, including the medications used in the lymphodepletion procedure.
• People with any other condition that the treating doctor believes could affect their ability to participate safely or could interfere with the study results.

Important: Always verify eligibility with the trial site directly before applying.