Clinical Research Alert: Clinical Study for Adults with Generalized Myasthenia Gravis (gMG)

Researchers at Vor Bio are conducting a global phase 3 clinical study (UPSTREAM MG) for adults with generalized myasthenia gravis (gMG) who are AChR or MuSK+. The study will evaluate whether treatment with the investigational drug telitacicept can reduce the level of disease-causing B cells and autoantibodies, and improve muscle weakness and fatigue, in people with gMG.

The study

This study will compare the effects of telitacicept with a placebo. During the 24-week main double-blinded period, participants will be randomly assigned to receive either the investigational study drug or a placebo control via weekly subcutaneous (under the skin) injections using prefilled syringes.

Participation in the study will include approximately one weekly in-office visit for the first four weeks, followed by one clinic visit every four weeks for the remainder of the study. On weeks without a clinic visit, the drug can be administered at home.

The effects of telitacicept will be evaluated using a number of tests and procedures including but not limited to: physical exams, safety assessments, laboratory testing, and validated questionnaires measuring MG outcomes (e.g., MG-ADL, QMG, MG-QoL15r).

After the double-blinded period, participants may enter an open-label extension period during which all participants will receive the investigational study drug. This period may last until commercialization of telitacicept.

Study criteria

To be eligible, individuals must meet the following inclusion criteria:

• Male or female patient aged ≥18 years at screening
• Prior confirmed diagnosis of gMG with generalized muscle weakness
• Positive antibodies against AChR or MuSK at screening
• MG-ADL score ≥6 points at screening and baseline with ocular-related score <50% of the total score
• QMG score ≥8 points, and ≥ 4 items score at least 2 points at screening and baseline

Individuals may not be eligible to participate if they are affected by another condition or receiving another treatment that might interfere with the ability to undergo safe testing.

Please visit this link for the full listing of inclusion and exclusion criteria.

Interested in participating?

Travel support is available for eligible participants and their families.

To learn more about the study or inquire about participation, please visit the sponsor’s trial website or contact the study coordinator by email: study@vorbio.com.

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