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A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis

PHASE3RECRUITING

This study is researching an experimental combination treatment with pozelimab and cemdisiran, and cemdisiran monotherapy. The study is focused on patients with generalized myasthenia gravis (gMG). Myasthenia gravis is a disease that causes weakness and fatigue in muscles in the body because the nerves and muscles are not communicating properly.

The aim of the study is to see how effective pozelimab and cemdisiran are when used in combination and when pozelimab and cemdisiran are used alone for patients with gMG. The study is looking at several other research questions, including: * What side effects may happen from taking the study drugs * How the study drugs work inside the body * How much study drugs are in the blood at different times * Whether the body makes antibodies against pozelimab and cemdisiran (which could make the drugs less effective or could lead to side effects).

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Study details:

DBTP- Double blind treatment period (24 weeks) ETP - Extension treatment period (28 weeks) OLTP- Open label treatment period (68 weeks) Off-treatment follow up period (52 weeks).

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

Male or female patients ≥18 years of age at screening (or ≥ legal age of adulthood based on local regulations, whichever is older)

Patient with documented diagnosis of myasthenia gravis (MG) based on medical history and supported by previous evaluations as described in the protocol

Documented prior history of positive serologic test or a positive result during screening of anti-acetylcholine receptor (AChR) antibodies or anti-LRP4 antibodies.

Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II to IVa at screening

Myasthenia Gravis-Activities of Daily Living (MG-ADL) score ≥6 at screening. Ocular items should not contribute more than 50% of MG-ADL total score

Currently receiving an acetylcholinesterase inhibitor or documented reason for not using acetylcholinesterase inhibitor therapy per investigator

Currently receiving an immunosuppressive therapy (IST) for MG, or documented reason why the patient is not taking an IST per investigator

If currently receiving an IST, not anticipated to have IST dosage changed before randomization or during double-blind treatment period (DBTP).

Willing and able to comply with clinic visits and study-related procedures, including completion of the primary series of the meningococcal vaccinations required per protocol

Exclusion criteria

Patients with antibody profile that is only positive for muscle specific tyrosine kinase (MuSK) (MuSK positivity is based on a documented prior history of positive serologic test for antibodies to MuSK or a positive result during screening

History of thymectomy within 12 months prior to screening or planned during the study

History of malignant thymoma (patients with stage 1 may be enrolled), or history of cancer within the past 5 years, except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer

Myasthenic crisis or Myasthenia Gravis Foundation of America (MGFA) Class V within 1 month of screening

Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to the screening visit and serotype B vaccine within 3 years prior to the screening visit as described in the protocol

Known contraindication to meningococcal vaccines (group ACWY conjugate and group B vaccines) as described in the protocol

Patients who require antibiotics for meningococcal prophylaxis and have a contraindication, warning, or precaution precluding the use of penicillin class and penicillin-alternative antibiotics planned to be used for prophylaxis, or a history of intolerance leading to the discontinuation of these antibiotics

Positive hepatitis B surface antigen or hepatitis C virus ribonucleic acid (RNA) during screening. NOTE: Cases with unclear interpretation should be discussed with the medical monitor

History of HIV infection or a positive test at screening per local requirements

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Eligibility

Age eligible for study : 18 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2021-12-14

Primary completion: 2025-05-21

Study completion finish: 2028-03-23

Study type

TREATMENT

Phase

PHASE3

Trial ID

NCT05070858

Intervention or treatment

DRUG: Pozelimab + Cemdisiran

DRUG: Cemdisiran

OTHER: Placebo

DRUG: Pozelimab

Conditions

• Generalized Myasthenia Gravis

Image related to Generalized Myasthenia Gravis

Condition: Generalized Myasthenia Gravis
DRUG: Pozelimab + Cemdisiran and other drugs
Sydney, New South Wales, Australia and more
Sponsor: Regeneron Pharmaceuticals

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Find a site

Closest Location:

Southern Neurology

Research sites nearby

Select from list below to view details:

Southern Neurology
Sydney, New South Wales, Australia
Lyell McEwin Hospital
Elizabeth Vale, South Australia, Australia
St. Vincent's Hospital Melbourne
Fitzroy, Victoria, Australia
Perron Institute for Neurological and Translational Science
Nedlands, Western Australia, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Group 1 Placebo in DBTP; Re-randomized to Combination or Cemdisiran in ETP and OLTP	DRUG: Pozelimab + Cemdisiran Subcutaneous administration as described in the protocol
EXPERIMENTAL: Group 2 Combination regimen throughout the study	DRUG: Pozelimab + Cemdisiran Subcutaneous administration as described in the protocol
EXPERIMENTAL: Group 3 Cemdisiran throughout the study	DRUG: Cemdisiran SC administration as described in the protocol
EXPERIMENTAL: Group 4 Pozelimab monotherapy in DBTP followed by combination in ETP and OLTP	DRUG: Pozelimab SC administration as described in the protocol

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score	The total MG-ADL score ranges from 0 to 24 points, with higher scores indicating greater functional impairment and disability	From baseline to week 24

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
Change from baseline in Quantitative Myasthenia Gravis (QMG) score	QMG total scores range from 0 to 39, with higher scores representing greater impairment	Week 24
Proportion of patients responding on the MG-ADL	≥3-point improvement	From baseline to week 24
Proportion of patients responding on the QMG	≥5-point improvement	From baseline to week 24
Proportion of patients with consistent response on the MG-ADL	At least a 2-point MG-ADL improvement on 2 or more consecutive assessments spanning 4 or more weeks during the DBTP	From baseline to week 24
Proportion of patients with minimal symptom expression (MSE)	Score of 0 to 1 on the MG-ADL	Week 24
Change from baseline in the Myasthenia Gravis Composite (MGC) total score	MGC score ranges from 0 to 50, with higher score indicating higher impairment	Week 24
Change from baseline in Myasthenia Gravis Quality of Life (MG QOL15r) total score	Total score ranges from 0 to 30 points; a higher score represents greater impairment	Week 24
Proportion of patients with improvement point thresholds on MG-ADL	≥2, 4, 5, 6, 7, 8, 9, or 10	From baseline to week 24
Proportion of patients with improvement point thresholds on QMG	≥3, 4, 6, 7, 8, 9, or 10	From baseline to week 24
Incidence and severity of treatment-related adverse events (TEAEs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo	Not Specified	Through week 24
Incidence and severity of serious adverse events (SAEs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo	Not Specified	Through week 24
Incidence and severity of adverse events of special interest (AESIs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo	Not Specified	Through week 24
Concentrations of total pozelimab in serum	Not Specified	Through study duration, approximate 172 weeks
Concentrations of total complement component 5 (C5) in plasma	Not Specified	Through study duration, approximate 172 weeks
Concentrations of cemdisiran and its metabolites in plasma	Not Specified	Through study duration, approximate 172 weeks
Incidence of treatment-emergent anti-drug antibodies (ADAs) to pozelimab over time	Not Specified	Through study duration, approximately 172 weeks
Incidence of treatment-emergent ADAs to cemdisiran over time	Not Specified	Through study duration, approximate 172 weeks
Change in total complement hemolysis activity assay (CH50) over time	Not Specified	Through study duration, approximately 172 weeks
Percent change in CH50 over time	Not Specified	Through study duration, approximately 172 weeks

Frequently Asked Questions

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References

Clinical Trials Gov: A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis