N-MOmentum: A Clinical Research Study of Inebilizumab in Neuromyelitis Optica Spectrum Disorders

NCT ID: NCT02200770

Last Updated: 2021-12-03

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

231 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-04-01

Study Completion Date

2020-11-06

Brief Summary

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To compare the efficacy of inebilizumab (MEDI-551) versus placebo in reducing the risk of an neuromyelitis optica/neuromyelitis optica- spectrum disorders (NMO/NMOSD) attack in participants with NMO/NMOSD.

Detailed Description

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Inebilizumab is a genetically engineered humanized monoclonal antibody that binds to the B cell specific surface antigen cluster of differentiation (CD19) resulting in the depletion of B cells. Inebilizumab depletes antibody-secreting plasmablasts and some plasma cells, which are generally CD19 positive and CD20 negative.

The main objective of this study is to determine whether inebilizumab compare to placebo decreases the risk of an attack in participants with NMO/NMOSD.

This is a multicenter, multinational, randomized, double-masked, placebo controlled study with an open-label extension period to evaluate the efficacy and safety of intravenous (IV) inebilizumab in adult participants with NMO/NMOSD. After a screening period, eligible participants will enter a randomized-controlled period (RCP) of maximum 197 days where they will be randomized in a 3:1 ratio to receive either IV inebilizumab or placebo. NMO/NMOSD attacks will be evaluated by the investigator and confirmed against the attack criteria by an independent Adjudication Committee (AC). Participants for whom the attack was confirmed by the AC will be given the option to enroll into an open label period (OLP) with inebilizumab treatment. Participants who complete the RCP without experiencing an attack will be given the option to enroll into an OLP with inebilizumab treatment. The OLP will continue for a minimum of 1 year and a maximum of 3 years after the last participant enter the OLP. All participants who discontinue from the RCP or the OLP will continue in a Safety Follow-up for a total of 12 months from last dose to evaluate the long-term safety of the investigational product.

Conditions

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Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders

Keywords

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NMO, NMOSD, Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorders, autoimmune, demyelination, MEDI-551, monoclonal antibody, Devic's syndrome, B-cell

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Placebo/Inebilizumab

Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants will receive IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who enter OLP will receive IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants will have choice to enter in the SFP at any point during RCP or OLP and will be free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants will continue in the SFP for 12 months from last dose of study drug.

Group Type PLACEBO_COMPARATOR

Inebilizumab

Intervention Type DRUG

Participants will receive IV inebilizumab 300 mg.

Placebo

Intervention Type OTHER

Participants will receive IV placebo matched to inebilizumab.

Inebilizumab/Inebilizumab

AQP4-IgG sero positive and sero negative participants will IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who enter OLP will receive IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants will have choice to enter in the SFP at any point during RCP or OLP and will be free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants will continue in the SFP for 12 months from last dose of study drug.

Group Type EXPERIMENTAL

Inebilizumab

Intervention Type DRUG

Participants will receive IV inebilizumab 300 mg.

Placebo

Intervention Type OTHER

Participants will receive IV placebo matched to inebilizumab.

Interventions

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Inebilizumab

Participants will receive IV inebilizumab 300 mg.

Intervention Type DRUG

Placebo

Participants will receive IV placebo matched to inebilizumab.

Intervention Type OTHER

Other Intervention Names

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MEDI-551

Eligibility Criteria

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Inclusion Criteria

1. Men and women 18 years or older with diagnosis of NMO/NMOSD
2. Confirmation of NMO/NMOSD status:

1. AQP4-IgG sero-positive NMO/NMOSD with at least one attack requiring rescue therapy in the last year or two attacks requiring rescue therapy in the last 2 years
2. AQP4-IgG sero-negative NMO with at least one attack requiring rescue therapy in the last year or two attacks requiring rescue therapy in the last 2 years
3. Able and willing to give written informed consent and comply with the requirements of the study protocol.
4. EDSS \<= 7.5 (8 in special circumstances)
5. Men and women of reproductive potential must agree to use a highly effective method of birth control from screening to 6 months after final dose of the investigational product.

Exclusion Criteria

1. Lactating and pregnant females
2. Treatment with any investigational agent within 4 weeks of screening
3. Known history of a severe allergy or reaction to any component of the investigational product formulation or history of anaphylaxis following any biologic therapy.
4. Known active severe bacterial, viral, or other infection or any major episode of infection requiring hospitalization.
5. History of alcohol, drug, or chemical abuse, or a recent history of such abuse \< 1 year prior to randomization
6. Receipt of the following at any time prior to randomization:

1. Alemtuzumab
2. Total lymphoid irradiation
3. Bone marrow transplant
4. T-cell vaccination therapy
7. Receipt of rituximab or any experimental B-cell depleting agent within 6 months prior screening and B-cells below the lower limit of normal.
8. Receipt of intravenous immunoglobulin (IVIG) within 1 month prior to randomization.
9. Receipt of any of the following within 3 months prior to randomization:

1. Natalizumab (Tysabri®).
2. Cyclosporin
3. Methotrexate
4. Mitoxantrone
5. Cyclophosphamide
6. Tocilizumab
7. Eculizumab
10. History of Hepatitis B and/or Hepatitis C (Hep B/C at screening)
11. Known history of a primary immunodeficiency (congenital or acquired) or an underlying condition such as human immunodeficiency virus (HIV) infection
12. History of malignancies, apart from squamous cell or basal cell carcinoma of the skin treated with documented success of curative therapy \> 3 months prior to randomization
13. Any concomitant disease other than NMO/NMOSD that required treatment with oral or intravenous steroids at doses over 20 mg a day for over 21 days
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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MedImmune LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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MedImmune, LLC MedImmune, LLC

Role: STUDY_DIRECTOR

MedImmune LLC

Locations

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Research Site

Birmingham, Alabama, United States

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Sacramento, California, United States

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San Francisco, California, United States

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Aurora, Colorado, United States

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New Haven, Connecticut, United States

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Maitland, Florida, United States

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Chicago, Illinois, United States

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Kansas City, Kansas, United States

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Baltimore, Maryland, United States

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Detroit, Michigan, United States

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Rochester, Minnesota, United States

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St Louis, Missouri, United States

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Raleigh, North Carolina, United States

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Cincinnati, Ohio, United States

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Cleveland, Ohio, United States

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Mansfield, Ohio, United States

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Dallas, Texas, United States

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Houston, Texas, United States

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Richmond, Virginia, United States

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Melbourne, , Australia

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Sofia, , Bulgaria

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Sofia, , Bulgaria

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Sofia, , Bulgaria

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Varna, , Bulgaria

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Vancouver, British Columbia, Canada

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Barranquilla, , Colombia

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Bogotá, , Colombia

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Bogotá, , Colombia

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Cali, , Colombia

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Olomouc, , Czechia

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Prague, , Czechia

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Teplice, , Czechia

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Tallinn, , Estonia

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Tartu, , Estonia

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Berlin, , Germany

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Dresden, , Germany

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Düsseldorf, , Germany

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Leipzig, , Germany

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Münster, , Germany

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Rostock, , Germany

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Hong Kong, , Hong Kong

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Esztergom, , Hungary

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Nyíregyháza, , Hungary

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Szeged, , Hungary

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Jerusalem, , Israel

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Ramat Gan, , Israel

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Tel Aviv, , Israel

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Aomori, , Japan

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Bunkyō City, , Japan

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Kyoto, , Japan

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Ōta-ku, , Japan

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Sendai, , Japan

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Tsukuba, , Japan

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Mexico City, , Mexico

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Mexico City, , Mexico

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Monterrey, , Mexico

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San Luis Potosí City, , Mexico

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Chisinau, , Moldova

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Auckland, , New Zealand

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Bellavista, , Peru

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Lima, , Peru

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Katowice, , Poland

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Krakow, , Poland

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Lódz, , Poland

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Lublin, , Poland

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Olsztyn, , Poland

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Warsaw, , Poland

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Warsaw, , Poland

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Belgorod, , Russia

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Kazan', , Russia

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Khabarovsk, , Russia

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Krasnoyarsk, , Russia

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Moscow, , Russia

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Moscow, , Russia

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Nizhny Novgorod, , Russia

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Novosibirsk, , Russia

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Omsk, , Russia

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Saint Petersburg, , Russia

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Ufa, , Russia

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Belgrade, , Serbia

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Cape Town, , South Africa

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Cape Town, , South Africa

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Goyang, , South Korea

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Jongno-gu, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Madrid, , Spain

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Changhua, , Taiwan

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Hualien City, , Taiwan

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Tainan City, , Taiwan

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Bangkok, , Thailand

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Muang, , Thailand

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Muang, , Thailand

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Istanbul, , Turkey (Türkiye)

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Istanbul, , Turkey (Türkiye)

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Istanbul, , Turkey (Türkiye)

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Izmir, , Turkey (Türkiye)

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Samsun, , Turkey (Türkiye)

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Countries

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China Greece India Netherlands Portugal Ukraine United States Australia Bulgaria Canada Colombia Czechia Estonia Germany Hong Kong Hungary Israel Japan Mexico Moldova New Zealand Peru Poland Russia Serbia South Africa South Korea Spain Taiwan Thailand Turkey (Türkiye)

References

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Cree BAC, Kim HJ, Weinshenker BG, Pittock SJ, Wingerchuk DM, Fujihara K, Paul F, Cutter GR, Marignier R, Green AJ, Aktas O, Hartung HP, She D, Rees W, Smith M, Cimbora D, Katz E, Bennett JL; N-MOmentum study investigators. Safety and efficacy of inebilizumab for the treatment of neuromyelitis optica spectrum disorder: end-of-study results from the open-label period of the N-MOmentum trial. Lancet Neurol. 2024 Jun;23(6):588-602. doi: 10.1016/S1474-4422(24)00077-2.

Reference Type DERIVED
PMID: 38760098 (View on PubMed)

Aktas O, Hartung HP, Smith MA, Rees WA, Fujihara K, Paul F, Marignier R, Bennett JL, Kim HJ, Weinshenker BG, Pittock SJ, Wingerchuk DM, Cutter G, She D, Gunsior M, Cimbora D, Katz E, Cree BA; N-MOmentum study investigators. Serum neurofilament light chain levels at attack predict post-attack disability worsening and are mitigated by inebilizumab: analysis of four potential biomarkers in neuromyelitis optica spectrum disorder. J Neurol Neurosurg Psychiatry. 2023 Sep;94(9):757-768. doi: 10.1136/jnnp-2022-330412. Epub 2023 May 23.

Reference Type DERIVED
PMID: 37221052 (View on PubMed)

Bennett JL, Aktas O, Rees WA, Smith MA, Gunsior M, Yan L, She D, Cimbora D, Pittock SJ, Weinshenker BG, Paul F, Marignier R, Wingerchuk D, Cutter G, Green A, Hartung HP, Kim HJ, Fujihara K, Levy M, Katz E, Cree BAC; N-MOmentum study investigators. Association between B-cell depletion and attack risk in neuromyelitis optica spectrum disorder: An exploratory analysis from N-MOmentum, a double-blind, randomised, placebo-controlled, multicentre phase 2/3 trial. EBioMedicine. 2022 Dec;86:104321. doi: 10.1016/j.ebiom.2022.104321. Epub 2022 Nov 10.

Reference Type DERIVED
PMID: 36370634 (View on PubMed)

Flanagan EP, Levy M, Katz E, Cimbora D, Drappa J, Mealy MA, She D, Cree BAC. Inebilizumab for treatment of neuromyelitis optica spectrum disorder in patients with prior rituximab use from the N-MOmentum Study. Mult Scler Relat Disord. 2022 Jan;57:103352. doi: 10.1016/j.msard.2021.103352. Epub 2021 Oct 26.

Reference Type DERIVED
PMID: 35158461 (View on PubMed)

Marignier R, Bennett JL, Kim HJ, Weinshenker BG, Pittock SJ, Wingerchuk D, Fujihara K, Paul F, Cutter GR, Green AJ, Aktas O, Hartung HP, Lublin FD, Williams IM, Drappa J, She D, Cimbora D, Rees W, Smith M, Ratchford JN, Katz E, Cree BAC; N-MOmentum Study Investigators. Disability Outcomes in the N-MOmentum Trial of Inebilizumab in Neuromyelitis Optica Spectrum Disorder. Neurol Neuroimmunol Neuroinflamm. 2021 Mar 26;8(3):e978. doi: 10.1212/NXI.0000000000000978. Print 2021 May.

Reference Type DERIVED
PMID: 33771837 (View on PubMed)

Aktas O, Smith MA, Rees WA, Bennett JL, She D, Katz E, Cree BAC; N-MOmentum scientific group and the N-MOmentum study investigators. Serum Glial Fibrillary Acidic Protein: A Neuromyelitis Optica Spectrum Disorder Biomarker. Ann Neurol. 2021 May;89(5):895-910. doi: 10.1002/ana.26067. Epub 2021 Mar 30.

Reference Type DERIVED
PMID: 33724534 (View on PubMed)

Cree BA, Bennett JL, Kim HJ, Weinshenker BG, Pittock SJ, Wingerchuk D, Fujihara K, Paul F, Cutter GR, Marignier R, Green AJ, Aktas O, Hartung HP, Williams IM, Drappa J, She D, Cimbora D, Rees W, Ratchford JN, Katz E. Sensitivity analysis of the primary endpoint from the N-MOmentum study of inebilizumab in NMOSD. Mult Scler. 2021 Nov;27(13):2052-2061. doi: 10.1177/1352458521988926. Epub 2021 Feb 4.

Reference Type DERIVED
PMID: 33538237 (View on PubMed)

Cree BAC, Bennett JL, Kim HJ, Weinshenker BG, Pittock SJ, Wingerchuk DM, Fujihara K, Paul F, Cutter GR, Marignier R, Green AJ, Aktas O, Hartung HP, Lublin FD, Drappa J, Barron G, Madani S, Ratchford JN, She D, Cimbora D, Katz E; N-MOmentum study investigators. Inebilizumab for the treatment of neuromyelitis optica spectrum disorder (N-MOmentum): a double-blind, randomised placebo-controlled phase 2/3 trial. Lancet. 2019 Oct 12;394(10206):1352-1363. doi: 10.1016/S0140-6736(19)31817-3. Epub 2019 Sep 5.

Reference Type DERIVED
PMID: 31495497 (View on PubMed)

Cree BA, Bennett JL, Sheehan M, Cohen J, Hartung HP, Aktas O, Kim HJ, Paul F, Pittock S, Weinshenker B, Wingerchuk D, Fujihara K, Cutter G, Patra K, Flor A, Barron G, Madani S, Ratchford JN, Katz E. Placebo-controlled study in neuromyelitis optica-Ethical and design considerations. Mult Scler. 2016 Jun;22(7):862-72. doi: 10.1177/1352458515620934. Epub 2015 Dec 14.

Reference Type DERIVED
PMID: 26666258 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2014-000253-36

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CD-IA-MEDI-551-1155

Identifier Type: -

Identifier Source: org_study_id