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A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis (UPSTREAM MG)

ConditionGeneralized Myasthenia Gravis
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorVor Biopharma

About this trial

The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male or female patient aged ≥18 years at screening.

Patients have prior confirmed diagnosis of gMG with generalized muscle weakness (typical pattern of weakness meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) clinical classification II-IV.

Patients have 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.

Disqualifiers

Patients have been diagnosed with any other autoimmune disease which can potentially pose a safety or efficacy confounding risk.

Patients having acute or chronic infection.

Patients having thymoma within 5 years or received thymectomy ≤6 months prior to screening. Patients with thymoma diagnosed 3-5 years prior to screening may be eligible if thymoma was at a localized stage and definitively treated with complete surgical resection.

Patients having current or history of primary immunodeficiency.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Telitacicept

    Biological/Vaccine

    Subcutaneous injection

  • Placebo

    Drug

    Subcutaneous injection

Treatment groups

180 Participants
are divided into 2 treatment groups
Group A: TelitaciceptExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Week 24

The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale is scored on a 0 to 3 (0=normal, 3=severe disease) point scale. The total score is the sum of all individual item scores ranging from 0 to 24. Higher scores indicate more severe disability due to MG. A decrease from Baseline score indicates improvement.

Time frame
Week 24

Secondary outcomes

1

Change from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24

The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consists of 13 items. Each item in the scale is scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicate more severe impairment. A decrease from Baseline score indicates improvement.

Time frame
Week 24
2

Change from baseline in MG Quality of Life scale (MG-QOL15r) at Week 24

The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale is scored on a 0 to 2-point scale (0=Not at all, 1=Somewhat, 2=Very much). The total score is the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicate more severe impact of the disease on aspects of the patient's life. A decrease from Baseline score indicates improvement.

Time frame
Week 24
3

Proportion of patients with a decrease of ≥2 points from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Week 24

Proportion of patients with a decrease of ≥2 points from baseline in MG-ADL score at Week 24. The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale is scored on a 0 to 3 (0=normal, 3=severe disease) point scale. The total score is the sum of all individual item scores ranging from 0 to 24. Higher scores indicate more severe disability due to MG.

Time frame
Week 24
4

Proportion of patients with a decrease of ≥3 points from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24

Proportion of patients with a decrease of ≥3 points from baseline in QMG score at Week 24. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consists of 13 items. Each item in the scale is scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicate more severe impairment. A decrease from Baseline score indicates improvement.

Time frame
Week 24

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Vor Biopharma

Lead sponsor

Source ClinicalTrials.gov
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