ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Last updated: April 18, 2024
Sponsor: Alexion Pharmaceuticals, Inc.
Overall Status: Completed

Phase

3

Condition

Proteinuria

Anemia

Hemoglobinuria, Paroxysmal

Treatment

Eculizumab

Ravulizumab

Clinical Study ID

NCT02946463
ALXN1210-PNH-301
2016-002025-11
  • All Genders

Study Summary

The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who had never been treated with a complement inhibitor (treatment-naïve).

Eligibility Criteria

Inclusion

Criteria For Patient Cohort Originally Enrolled in ALXN1210-PNH-301 Study: Inclusion Criteria: 1. Male or female ≥18 years of age. 2. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry. 3. Presence of 1 or more of the following PNH-related signs or symptoms within 3 months of screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia (hemoglobin <10 gram/deciliter), history of a major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction; or history of packed red blood cells (pRBC) transfusion due to PNH. 4. Lactate dehydrogenase (LDH) level ≥1.5 times the upper limit of normal at screening. 5. Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment. 6. Female participants of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab. 7. Willing and able to give written informed consent

Exclusion

and comply with study visit schedule. Exclusion Criteria: 1. Treatment with a complement inhibitor at any time. 2. History of bone marrow transplantation. 3. Body weight <40 kg. 4. Females who are pregnant, breastfeeding, or who have a positive pregnancy test at screening or Day 1. 5. Participation in another interventional clinical study or use of any experimental therapy within 30 days before initiation of study drug on Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater. 6. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the investigator or sponsor, would preclude participation. 7. Unstable medical conditions (for example, myocardial ischemia, active gastrointestinal bleed, severe congestive heart failure, anticipated need for major surgery within 6 months of randomization, coexisting chronic anemia unrelated to PNH). Eligibility Criteria For Roll-over Cohort: 1. All participants regardless of age, who are currently receiving ALXN1210 IV in an ongoing ALXN1210 study in patients with PNH 2. Participants must be willing and able to give written informed consent and to comply with all Extension study visits and procedures, including the use of any data collection device(s) to directly record patient data 3. Females of childbearing potential and male patients with female partners of childbearing potential must use highly effective contraception continuing until at least 8 months after the last dose of ravulizumab.

Study Design

Total Participants: 272
Treatment Group(s): 2
Primary Treatment: Eculizumab
Phase: 3
Study Start date:
December 12, 2016
Estimated Completion Date:
February 28, 2023

Study Description

The study consisted of a 4-week screening period and a 26-week randomized treatment period (Primary Evaluation Period). After completion of the 26-week Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, wherein participants will receive ravulizumab for up to 5 years.

Connect with a study center

  • Clinical Trial Site

    Buenos Aires, C1425AUM
    Argentina

    Site Not Available

  • Research Site

    Buenos Aires, C1425AUM
    Argentina

    Site Not Available

  • Clinical Trial Site

    Caba,
    Argentina

    Site Not Available

  • Clinical Trial Site

    Córdoba,
    Argentina

    Site Not Available

  • Research Site

    Córdoba, X5004BAL
    Argentina

    Site Not Available

  • Clinical Trial Site

    Perth,
    Australia

    Site Not Available

  • Research Site

    Perth, 6000
    Australia

    Site Not Available

  • Clinical Trial Site

    Linz,
    Austria

    Site Not Available

  • Research Site

    Linz, 4020
    Austria

    Site Not Available

  • Clinical Trial Site

    Vienna,
    Austria

    Site Not Available

  • Research Site

    Vienna, A-1090
    Austria

    Site Not Available

  • Research Site

    Bruxelles, 1200
    Belgium

    Site Not Available

  • Clinical Trial Site

    Hasselt,
    Belgium

    Site Not Available

  • Research Site

    Hasselt, 3500
    Belgium

    Site Not Available

  • Clinical Trial Site

    Leuven,
    Belgium

    Site Not Available

  • Research Site

    Leuven, 3000
    Belgium

    Site Not Available

  • Clinical Trial Site

    Belém,
    Brazil

    Site Not Available

  • Research Site

    Rio De Janeiro,
    Brazil

    Site Not Available

  • Clinical Trial Site

    Rio de Janeiro,
    Brazil

    Site Not Available

  • Clinical Trial Site

    Salvador,
    Brazil

    Site Not Available

  • Research Site

    Salvador, 41253-190
    Brazil

    Site Not Available

  • Research Site

    Sao Paulo,
    Brazil

    Site Not Available

  • Clinical Trial Site

    São Paulo, 05403-000
    Brazil

    Site Not Available

  • Research Site

    Edmonton, Alberta T6G 2G3
    Canada

    Site Not Available

  • Research Site

    Toronto, Ontario M4N 3M5
    Canada

    Site Not Available

  • Clinical Trial Site

    Edmonton,
    Canada

    Site Not Available

  • Clinical Trial Site

    Toronto,
    Canada

    Site Not Available

  • Clinical Trial Site

    Bogotá,
    Colombia

    Site Not Available

  • Clinical Trial Site

    Cali,
    Colombia

    Site Not Available

  • Clinical Trial Site

    Medellín,
    Colombia

    Site Not Available

  • Clinical Trial Site

    Plzen,
    Czechia

    Site Not Available

  • Research Site

    Plzeň, 323 00
    Czechia

    Site Not Available

  • Research Site

    Prague,
    Czechia

    Site Not Available

  • Clinical Trial Site

    Praha,
    Czechia

    Site Not Available

  • Clinical Trial Site

    Tallinn,
    Estonia

    Site Not Available

  • Research Site

    Tallinn, 13419
    Estonia

    Site Not Available

  • Clinical Trial Site

    Poitiers, Vienne
    France

    Site Not Available

  • Clinical Trial Site

    Limoges,
    France

    Site Not Available

  • Research Site

    Limoges, 87042
    France

    Site Not Available

  • Research Site

    MONTPELLIER Cedex 5, 34295
    France

    Site Not Available

  • Clinical Trial Site

    Montpellier,
    France

    Site Not Available

  • Clinical Trial Site

    Paris,
    France

    Site Not Available

  • Research Site

    Paris Cedex 10, 75475
    France

    Site Not Available

  • Research Site

    Pierre Benite, 69310
    France

    Site Not Available

  • Clinical Trial Site

    Pierre-Bénite,
    France

    Site Not Available

  • Research Site

    Poitiers, 86021
    France

    Site Not Available

  • Clinical Trial Site

    Rennes,
    France

    Site Not Available

  • Research Site

    Rennes Cedex 9, 35033
    France

    Site Not Available

  • Research Site

    Aachen, 52074
    Germany

    Site Not Available

  • Clinical Trial Site

    Essen,
    Germany

    Site Not Available

  • Research Site

    Essen, 45122
    Germany

    Site Not Available

  • Clinical Trial Site

    Ulm,
    Germany

    Site Not Available

  • Research Site

    Ulm, 89081
    Germany

    Site Not Available

  • Clinical Trial Site

    Ascoli Piceno,
    Italy

    Site Not Available

  • Research Site

    Ascoli Piceno, 63100
    Italy

    Site Not Available

  • Clinical Trial Site

    Firenze,
    Italy

    Site Not Available

  • Research Site

    Firenze, 50134
    Italy

    Site Not Available

  • Clinical Trial Site

    Milano,
    Italy

    Site Not Available

  • Research Site

    Milano, 20122
    Italy

    Site Not Available

  • Clinical Trial Site

    Napoli,
    Italy

    Site Not Available

  • Research Site

    Napoli, 80131
    Italy

    Site Not Available

  • Clinical Trial Site

    Vicenza,
    Italy

    Site Not Available

  • Research Site

    Vicenza, 36100
    Italy

    Site Not Available

  • Clinical Trial Site

    Ogaki, Gifu-ken
    Japan

    Site Not Available

  • Clinical Trial Site

    Kanazawa, Ishikawa-Ken
    Japan

    Site Not Available

  • Clinical Trial Site

    Koshigaya, Saitama
    Japan

    Site Not Available

  • Clinical Trial Site

    Shinjuku, Tokyo-To
    Japan

    Site Not Available

  • Clinical Trial Site

    Bunkyo-ku,
    Japan

    Site Not Available

  • Research Site

    Bunkyo-ku, 113-8519
    Japan

    Site Not Available

  • Clinical Trial Site

    Bunkyō,
    Japan

    Site Not Available

  • Clinical Trial Site

    Bunkyō-Ku, 113-8431
    Japan

    Site Not Available

  • Clinical Trial Site

    Fukuoka,
    Japan

    Site Not Available

  • Research Site

    Fukuoka-Shi, 812-8582
    Japan

    Site Not Available

  • Clinical Trial Site

    Fukushima,
    Japan

    Site Not Available

  • Clinical Trial Site

    Fukushima-shi,
    Japan

    Site Not Available

  • Research Site

    Fukushima-shi, 960-1295
    Japan

    Site Not Available

  • Clinical Trial Site

    Hamamatsu,
    Japan

    Site Not Available

  • Clinical Trial Site

    Hamamatsu-shi,
    Japan

    Site Not Available

  • Research Site

    Hamamatsu-shi, 432-8580
    Japan

    Site Not Available

  • Clinical Trial Site

    Kanazawa-shi,
    Japan

    Site Not Available

  • Research Site

    Kanazawa-shi, 920-8641
    Japan

    Site Not Available

  • Research Site

    Kitakyusyu-shi, 806-8501
    Japan

    Site Not Available

  • Clinical Trial Site

    Koshigaya-shi,
    Japan

    Site Not Available

  • Research Site

    Koshigaya-shi, 343-8555
    Japan

    Site Not Available

  • Clinical Trial Site

    Kumamoto,
    Japan

    Site Not Available

  • Research Site

    Kumamoto-shi, 860-8556
    Japan

    Site Not Available

  • Research Site

    Nagoya-shi, 453-8511
    Japan

    Site Not Available

  • Clinical Trial Site

    Nishinomiya,
    Japan

    Site Not Available

  • Clinical Trial Site

    Nishinomiya-shi,
    Japan

    Site Not Available

  • Research Site

    Nishinomiya-shi, 663-8501
    Japan

    Site Not Available

  • Clinical Trial Site

    Ogaki-shi,
    Japan

    Site Not Available

  • Research Site

    Ogaki-shi, 503-8502
    Japan

    Site Not Available

  • Clinical Trial Site

    Okayama,
    Japan

    Site Not Available

  • Clinical Trial Site

    Okayama-city,
    Japan

    Site Not Available

  • Clinical Trial Site

    Okayama-shi,
    Japan

    Site Not Available

  • Research Site

    Okayama-shi, 700-8558
    Japan

    Site Not Available

  • Clinical Trial Site

    Osakasayama,
    Japan

    Site Not Available

  • Clinical Trial Site

    Osakasayama-shi,
    Japan

    Site Not Available

  • Research Site

    Osakasayama-shi, 589-8511
    Japan

    Site Not Available

  • Clinical Trial Site

    Sapporo,
    Japan

    Site Not Available

  • Research Site

    Sapporo-shi, 060-8543
    Japan

    Site Not Available

  • Clinical Trial Site

    Shimotsuke,
    Japan

    Site Not Available

  • Clinical Trial Site

    Shimotsuke-shi,
    Japan

    Site Not Available

  • Research Site

    Shimotsuke-shi, 329-0498
    Japan

    Site Not Available

  • Clinical Trial Site

    Shinagawa,
    Japan

    Site Not Available

  • Clinical Trial Site

    Shinagawa-Ku,
    Japan

    Site Not Available

  • Clinical Trial Site

    Shinjuku-Ku, 160-0023
    Japan

    Site Not Available

  • Research Site

    Shinjuku-ku, 160-0023
    Japan

    Site Not Available

  • Clinical Trial Site

    Suita,
    Japan

    Site Not Available

  • Research Site

    Suita, 565-0871
    Japan

    Site Not Available

  • Clinical Trial Site

    Suita-shi,
    Japan

    Site Not Available

  • Clinical Trial Site

    Tokorozawa,
    Japan

    Site Not Available

  • Clinical Trial Site

    Tokorozawa-shi,
    Japan

    Site Not Available

  • Research Site

    Tokorozawa-shi,
    Japan

    Site Not Available

  • Research Site

    Tokyo,
    Japan

    Site Not Available

  • Clinical Trial Site

    Toyoake-shi,
    Japan

    Site Not Available

  • Research Site

    Toyoake-shi, 470-1192
    Japan

    Site Not Available

  • Clinical Trial Site

    Toyohashi,
    Japan

    Site Not Available

  • Clinical Trial Site

    Tsukuba,
    Japan

    Site Not Available

  • Research Site

    Tsukuba-shi, 305-8576
    Japan

    Site Not Available

  • Clinical Trial Site

    Wakayama,
    Japan

    Site Not Available

  • Clinical Trial Site

    Wakayama-shi,
    Japan

    Site Not Available

  • Research Site

    Wakayama-shi, 641-8510
    Japan

    Site Not Available

  • Clinical Trial Site

    Yokohama,
    Japan

    Site Not Available

  • Clinical Trial Site

    Yokohama-City, 236-0004
    Japan

    Site Not Available

  • Clinical Trial Site

    Anyang-si,
    Korea, Republic of

    Site Not Available

  • Clinical Trial Site

    Busan,
    Korea, Republic of

    Site Not Available

  • Clinical Trial Site

    Daegu,
    Korea, Republic of

    Site Not Available

  • Clinical Trial Site

    Daejeon,
    Korea, Republic of

    Site Not Available

  • Research Site

    Daejeon, 35015
    Korea, Republic of

    Site Not Available

  • Research Site

    Goyang-si, 10408
    Korea, Republic of

    Site Not Available

  • Clinical Trial Site

    Incheon,
    Korea, Republic of

    Site Not Available

  • Research Site

    Incheon, 21565
    Korea, Republic of

    Site Not Available

  • Clinical Trial Site

    Jeonju,
    Korea, Republic of

    Site Not Available

  • Research Site

    Jeonju-si, 561-712
    Korea, Republic of

    Site Not Available

  • Research Site

    JinJoo, 52727
    Korea, Republic of

    Site Not Available

  • Clinical Trial Site

    Jinju-si,
    Korea, Republic of

    Site Not Available

  • Research Site

    Jung-gu, 41944
    Korea, Republic of

    Site Not Available

  • Clinical Trial Site

    Seoul, 03722
    Korea, Republic of

    Site Not Available

  • Research Site

    Seoul, 135-710
    Korea, Republic of

    Site Not Available

  • Research Site

    Songpa-gu, 05505
    Korea, Republic of

    Site Not Available

  • Clinical Trial Site

    Suwon-si, 16499
    Korea, Republic of

    Site Not Available

  • Research Site

    Suwon-si, 16247
    Korea, Republic of

    Site Not Available

  • Clinical Trial Site

    Ulsan,
    Korea, Republic of

    Site Not Available

  • Research Site

    Ulsan, 44033
    Korea, Republic of

    Site Not Available

  • Clinical Trial Site

    Miri, Sarawak
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Sibu, Sarawak
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Ampang,
    Malaysia

    Site Not Available

  • Research Site

    George, 10990
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Johor Bahru,
    Malaysia

    Site Not Available

  • Research Site

    Johor Bahru, 80100
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Kelantan,
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Kota Bahru, 16150
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Kota Bharu, 15586
    Malaysia

    Site Not Available

  • Research Site

    Kota Bharu, 16150
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Kota Kinabalu,
    Malaysia

    Site Not Available

  • Research Site

    Kota Kinabalu, 88586
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Kubang Kerian,
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Kuching,
    Malaysia

    Site Not Available

  • Research Site

    Kuching, 93586
    Malaysia

    Site Not Available

  • Research Site

    Miri, 98000
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Pulau Pinang,
    Malaysia

    Site Not Available

  • Research Site

    Sibu, 96000
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Monterrey,
    Mexico

    Site Not Available

  • Research Site

    Monterrey, 64460
    Mexico

    Site Not Available

  • Clinical Trial Site

    Gdańsk,
    Poland

    Site Not Available

  • Research Site

    Gdańsk, 80-214
    Poland

    Site Not Available

  • Clinical Trial Site

    Warsaw,
    Poland

    Site Not Available

  • Research Site

    Warszawa, 02-172
    Poland

    Site Not Available

  • Clinical Trial Site

    Arkhangel'sk,
    Russian Federation

    Site Not Available

  • Research Site

    Arkhangelsk, 163045
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Barnaul,
    Russian Federation

    Site Not Available

  • Research Site

    Barnaul, 656024
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Bryansk,
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Dzerzhinsk,
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Irkutsk,
    Russian Federation

    Site Not Available

  • Research Site

    Irkutsk, 664079
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Kaluga,
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Kirov,
    Russian Federation

    Site Not Available

  • Research Site

    Kirov, 610027
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Krasnodar, 350007
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Krasnoyarsk, 660022
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Moscow, 125167
    Russian Federation

    Site Not Available

  • Research Site

    Moscow, 117997
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Murmansk,
    Russian Federation

    Site Not Available

  • Research Site

    Murmansk, 183047
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Nizhny Novgorod,
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Novosibirsk,
    Russian Federation

    Site Not Available

  • Research Site

    Novosibirsk, 630091
    Russian Federation

    Site Not Available

  • Research Site

    Omsk, 644013
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Petrozavodsk,
    Russian Federation

    Site Not Available

  • Research Site

    Petrozavodsk, 185019
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Rostov-na-Donu,
    Russian Federation

    Site Not Available

  • Research Site

    Rostov-on-Don, 344022
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Saint Petersburg,
    Russian Federation

    Site Not Available

  • Research Site

    Saint-Petersburg, 197022
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Saratov,
    Russian Federation

    Site Not Available

  • Research Site

    Saratov, 410028
    Russian Federation

    Site Not Available

  • Research Site

    St. Petersburg,
    Russian Federation

    Site Not Available

  • Clinical Trial Site

    Ufa,
    Russian Federation

    Site Not Available

  • Research Site

    Ufa, 450005
    Russian Federation

    Site Not Available

  • Research Site

    Belgrade, 11000
    Serbia

    Site Not Available

  • Clinical Trial Site

    Singapore,
    Singapore

    Site Not Available

  • Research Site

    Singapore, 119228
    Singapore

    Site Not Available

  • Clinical Trial Site

    Barcelona,
    Spain

    Site Not Available

  • Clinical Trial Site

    Madrid,
    Spain

    Site Not Available

  • Research Site

    Madrid, 28040
    Spain

    Site Not Available

  • Clinical Trial Site

    Majadahonda,
    Spain

    Site Not Available

  • Research Site

    Majadahonda, 28220
    Spain

    Site Not Available

  • Clinical Trial Site

    Uppsala,
    Sweden

    Site Not Available

  • Research Site

    Uppsala, 75185
    Sweden

    Site Not Available

  • Clinical Trial Site

    Chang-hua,
    Taiwan

    Site Not Available

  • Research Site

    Changhua, 50006
    Taiwan

    Site Not Available

  • Clinical Trial Site

    Hualien City,
    Taiwan

    Site Not Available

  • Research Site

    Hualien City, 97002
    Taiwan

    Site Not Available

  • Clinical Trial Site

    Kaohsiung,
    Taiwan

    Site Not Available

  • Clinical Trial Site

    Taichung,
    Taiwan

    Site Not Available

  • Research Site

    Taichung, 404
    Taiwan

    Site Not Available

  • Clinical Trial Site

    Tainan,
    Taiwan

    Site Not Available

  • Research Site

    Tainan, 70403
    Taiwan

    Site Not Available

  • Clinical Trial Site

    Taipei,
    Taiwan

    Site Not Available

  • Research Site

    Taipei, 100
    Taiwan

    Site Not Available

  • Clinical Trial Site

    Bangkok,
    Thailand

    Site Not Available

  • Research Site

    Bangkok, 10330
    Thailand

    Site Not Available

  • Clinical Trial Site

    Hat Yai,
    Thailand

    Site Not Available

  • Clinical Trial Site

    Pathum Wan,
    Thailand

    Site Not Available

  • Clinical Trial Site

    Songkhla,
    Thailand

    Site Not Available

  • Research Site

    Songkhla, 90110
    Thailand

    Site Not Available

  • Research Site

    Eskisehir, 26040
    Turkey

    Site Not Available

  • Clinical Trial Site

    Eskişehir,
    Turkey

    Site Not Available

  • Research Site

    Airdrie, ML6 0JS
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Leeds,
    United Kingdom

    Site Not Available

  • Research Site

    Leeds,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    London,
    United Kingdom

    Site Not Available

  • Research Site

    London, SE5 9NU
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Los Angeles, California 90048
    United States

    Site Not Available

  • Research Site

    Los Angeles, California 90033
    United States

    Site Not Available

  • Clinical Trial Site

    Whittier, California
    United States

    Site Not Available

  • Research Site

    Whittier, California 90603
    United States

    Site Not Available

  • Clinical Trial Site

    Fort Worth, Texas 76104
    United States

    Site Not Available

  • Research Site

    Fort Worth, Texas 76104
    United States

    Site Not Available

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