Myelodysplastic Syndrome Clinical Trial
ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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).
Full 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.
Eligibility Criteria
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 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.
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There are 112 Locations for this study
Los Angeles California, 90033, United States
Whittier California, 90603, United States
Fort Worth Texas, 76104, United States
Buenos Aires , C1015, Argentina
Buenos Aires , C1425, Argentina
Córdoba , X5004, Argentina
Perth , 6000, Australia
Linz , 4020, Austria
Vienna , A-109, Austria
Bruxelles , 1200, Belgium
Hasselt , 3500, Belgium
Leuven , 3000, Belgium
Rio De Janeiro , , Brazil
Salvador , 41253, Brazil
Sao Paulo , 05403, Brazil
Sao Paulo , , Brazil
Edmonton Alberta, T6G 2, Canada
Toronto Ontario, M4N 3, Canada
Plzeň , 323 0, Czechia
Prague , , Czechia
Tallinn , 13419, Estonia
Limoges , 87042, France
MONTPELLIER Cedex 5 , 34295, France
Paris Cedex 10 , 75475, France
Pierre Benite , 69310, France
Poitiers , 86021, France
Rennes Cedex 9 , 35033, France
Aachen , 52074, Germany
Essen , 45122, Germany
Ulm , 89081, Germany
Ascoli Piceno , 63100, Italy
Firenze , 50134, Italy
Milano , 20122, Italy
Napoli , 80131, Italy
Vicenza , 36100, Italy
Bunkyo-ku , 113-8, Japan
Bunkyo-ku , 113-8, Japan
Fukuoka-Shi , 812-8, Japan
Fukushima-shi , 960-1, Japan
Hamamatsu-shi , 432-8, Japan
Kanazawa-shi , 920-8, Japan
Kitakyusyu-shi , 806-8, Japan
Koshigaya-shi , 343-8, Japan
Kumamoto-shi , 860-8, Japan
Nagoya-shi , 453-8, Japan
Nishinomiya-shi , 663-8, Japan
Ogaki-shi , 503-8, Japan
Okayama-shi , 700-8, Japan
Okayama-shi , 701-1, Japan
Osakasayama-shi , 589-8, Japan
Sapporo-shi , 060-8, Japan
Shimotsuke-shi , 329-0, Japan
Shinjuku-ku , 160-0, Japan
Shinjuku-ku , 160-8, Japan
Suita , 565-0, Japan
Tokorozawa-shi , , Japan
Tokyo , , Japan
Toyoake-shi , 470-1, Japan
Tsukuba-shi , 305-8, Japan
Wakayama-shi , 641-8, Japan
Daejeon , 35015, Korea, Republic of
Goyang-si , 10408, Korea, Republic of
Incheon , 21565, Korea, Republic of
Jeonju-si , 561-7, Korea, Republic of
JinJoo , 52727, Korea, Republic of
Jung-gu , 41944, Korea, Republic of
Seoul , 02841, Korea, Republic of
Seoul , 03080, Korea, Republic of
Seoul , 03722, Korea, Republic of
Seoul , 04401, Korea, Republic of
Seoul , 06591, Korea, Republic of
Seoul , 07985, Korea, Republic of
Seoul , 135-7, Korea, Republic of
Seoul , 15270, Korea, Republic of
Songpa-gu , 05505, Korea, Republic of
Suwon-si , 16247, Korea, Republic of
Ulsan , 44033, Korea, Republic of
George , 10990, Malaysia
Johor Bahru , 80100, Malaysia
Kota Bharu , 15586, Malaysia
Kota Bharu , 16150, Malaysia
Kota Kinabalu , 88586, Malaysia
Kuching , 93586, Malaysia
Miri , 98000, Malaysia
Sibu , 96000, Malaysia
Monterrey , 64460, Mexico
Gdańsk , 80-21, Poland
Warszawa , 02-17, Poland
Arkhangelsk , 16304, Russian Federation
Barnaul , 65602, Russian Federation
Irkutsk , 66407, Russian Federation
Kirov , 61002, Russian Federation
Moscow , 11799, Russian Federation
Moscow , 12528, Russian Federation
Murmansk , 18304, Russian Federation
Novosibirsk , 63009, Russian Federation
Omsk , 64401, Russian Federation
Petrozavodsk , 18501, Russian Federation
Rostov-on-Don , 34402, Russian Federation
Saint-Petersburg , 19702, Russian Federation
Saratov , 41002, Russian Federation
St. Petersburg , , Russian Federation
Ufa , 45000, Russian Federation
Belgrade , 11000, Serbia
Singapore , 11922, Singapore
Madrid , 28040, Spain
Majadahonda , 28220, Spain
Uppsala , 75185, Sweden
Changhua , 50006, Taiwan
Hualien City , 97002, Taiwan
Taichung , 404, Taiwan
Tainan , 70403, Taiwan
Taipei , 100, Taiwan
Bangkok , 10330, Thailand
Bangkok , 10700, Thailand
Songkhla , 90110, Thailand
Eskisehir , 26040, Turkey
Airdrie , ML6 0, United Kingdom
Leeds , , United Kingdom
London , SE5 9, United Kingdom
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