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A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis

A study to test efficacy and safety of rozanolixizumab in adult patients with generalized myasthenia gravis

Yes

Plan Description : Data from this study may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.clinicalstudydatarequest.com and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. This plan may change if a determination is made that the data cannot be adequately anonymized. IPD Time Frame : Data from this study may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion. IPD Access Criteria : Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.clinicalstudydatarequest.com and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. IPD URL https://clinicalstudydatarequest.com

UCB Japan Co., Ltd.

8-17-1 Nishi-shinjuku, Shinjuku-ku, Tokyo

CTR_SCC_UCBJapan@UCB.com

UCB Japan Co., Ltd.

8-17-1 Nishi-shinjuku, Shinjuku-ku, Tokyo

CTR_SCC_UCBJapan@UCB.com

completed

Mar. 11, 2020

Interventional

Multicenter, randomized, double-blind, placebo-controlled study

treatment purpose

3

- Study participant must be >=18 years of age, at the time of signing the informed consent
- Study participant has documented diagnosis of generalized myasthenia gravis (gMG) at Visit 1, based on study participants history and supported by previous evaluations
- Study participant has a confirmed positive record of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) at Screening (Visit 1). The presence of autoantibodies may be confirmed with repeat testing at Visit 1
- Study participant has Myasthenia Gravis Foundation of America (MGFA) Class ll to IVa at Visit 1
- Study participant with a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score of at least 3 AND a quantitative myasthenia gravis (QMG) score of at least 11 at Visit 1 and at Baseline (Visit 2)
- Study participant is considered for additional treatment such as intravenous immunoglobulin g(IVIg) or plasma exchange (PEX) by the Investigator

- Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP)
- Study participant has experienced hypersensitivity reaction after exposure to other antineonatal Fc receptor (FcRn) drugs
- Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Visit 1 or Visit 2

Both

Generalized myasthenia gravis

investigational material(s)
Generic name etc : Rozanolixizumab
INN of investigational material : rozanolixizumab
Therapeutic category code : 399 Agents affecting metabolism, n.e.c.
Dosage and Administration for Investigational material : Rozanolixizumab will be administered by subcutaneous infusion in 2 different dosages
(dose 1 and dose 2) in dosage regimen 1 and 2.

control material(s)
Generic name etc : Placebo
INN of investigational material : -
Therapeutic category code : --- Other
Dosage and Administration for Investigational material : -

efficacy
Change from Baseline to Day 43 (Visit 10) in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score

safety
efficacy
1. Percentage of participants achieving Myasthenia Gravis-Activities of Daily Living (MG-ADL) response at Visit 10
2. Change from Baseline to (Day 43) Visit 10 in the Myasthenia Gravis-Composite (MG-C) score
3. Change from Baseline to (Day 43) Visit 10 in Quantitative Myasthenia Gravis (QMG) score
4. Change from Baseline to (Day 43) Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) [Muscle Weakness Fatigability] score
5. Change from Baseline to (Day 43) Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) [Physical Fatigue] score
6. Change from Baseline to (Day 43) Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) [Bulbar Symptoms] score
7. Occurrence of treatment-emergent adverse events (TEAEs)
8. Treatment-emergent adverse events (TEAEs) leading to withdrawal of investigational medicinal product (IMP)

+81-11-611-2111

Feb. 14, 2020