Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Evaluation of the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria

Study Overview

• Status: Completed
• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Intervention / Treatment: Drug: Pegcetacoplan; Drug: Soliris

• Study Type: Interventional
• Enrollment (Actual): 80
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Melbourne, Victoria, Australia, 3000
      • Royal Melbourne Hospital
• Belgium
  • Vlaams-Brabant
    • Woluwe-Saint-Lambert, Vlaams-Brabant, Belgium, 1200
      • Cliniques Universitaires Saint-Luc
  • West-Vlaanderen
    • Roeselare, West-Vlaanderen, Belgium, 8800
      • AZ Delta Campus Wilgenstraat
• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2G3
      • University of Alberta
  • Ontario
    • Toronto, Ontario, Canada, M9A1G2
      • Toronto General Hospital
• France
  • Annecy, France, 74374
    • Centre Hospitalier Annecy Genevois
  • Chalon-sur-Saône, France, 71100
    • Centre Hospitalier William Morey
  • Lille, France, 59037
    • Centre Hospitalier Universitaire de Lille
  • Marseille, France, 13009
    • Institut Paoli-Calmettes Marseille
  • Paris, France, 75010
    • Hôpital Saint-Louis
  • Pierre-Bénite, France, 69495
    • Centre Hospitalier Lyon Sud
  • Saint-Quentin, France, 02321
    • Centre Hospitalier de Saint-Quentin
  • Toulouse, France, 31059
    • Institut Universitaire Du Cancer Toulouse - Oncopole
• Germany
  • Hamburg, Germany, 20246
    • Universitätsklinikum Hamburg Eppendorf
  • Baden-Württemberg
    • Ulm, Baden-Württemberg, Germany, 89081
      • Universitätsklinikum Ulm
  • North Rhine-Westphalia
    • Aachen, North Rhine-Westphalia, Germany, 52074
      • Uniklinik RWTH Aachen
    • Essen, North Rhine-Westphalia, Germany, 45147
      • Universitätsklinikum Essen
• Japan
  • Aichi
    • Nagoya, Aichi, Japan, 453-8511
      • Japanese Red Cross Nagoya Daiichi Hospital
    • Showa-ku, Aichi, Japan, 453-8650
      • Japanese Red Cross Nagoya Daini Hospital
  • Ibaraki
    • Tsukuba, Ibaraki, Japan, 305-8576
      • University of tsukuba hospital
  • Nagano
    • Matsumoto, Nagano, Japan, 390-8621
      • Shinshu University Hospital
  • Okayama
    • Okayama-shi, Okayama, Japan, 700-8558
      • Okayama University Hospital
  • Tokyo
    • Bunkyo-ku, Tokyo, Japan, 113-8421
      • Juntendo University Hospital
    • Shinagawa-ku, Tokyo, Japan, 141-8625
      • NTT Medical Center Tokyo
  • Wakayama
    • Tanabe, Wakayama, Japan, 646-8588
      • Kinan Hospital
• Korea, Republic of
  • Bucheon, Korea, Republic of, 14584
    • Soonchunhyang University Bucheon Hospital
  • Daejeon, Korea, Republic of, 35015
    • Chungnam National University Hospital
  • Seoul, Korea, Republic of, 06351
    • Samsung Medical Center
• Russian Federation
  • Saint Petersburg, Russian Federation, 197022
    • Pavlov First Saint Petersburg State Medical University
  • Saint Petersburg, Russian Federation, 197022
    • Pavlov First Saint Petersburg State Medical University of Russian Ministry of Health
  • Tyumen, Russian Federation, 625023
    • Institution of Health Care of Tyumen Region
• Spain
  • Las Palmas De Gran Canaria, Spain, 35010
    • Hospital Universitario de Gran Canaria Dr. Negrin
  • Valencia, Spain, 46026
    • Hospital Universitario Politecnico La Fe
• United Kingdom
  • Leeds, United Kingdom, LS9 7TF
    • St. James' Institute of Oncology, Leeds Teaching Hospitals
• United States
  • California
    • Los Angeles, California, United States, 90033
      • University of Southern California
    • Santa Monica, California, United States, 90403
      • Sarcoma Oncology Research Center
  • Colorado
    • Denver, Colorado, United States, 80204
      • Denver Health
  • District of Columbia
    • Washington, District of Columbia, United States, 20057
      • Georgetown University Hospital
  • Florida
    • Jacksonville, Florida, United States, 32256
      • Cancer Specialists of North Florida
    • Miami Lakes, Florida, United States, 33014
      • Lakes Research
    • Orange City, Florida, United States, 32763
      • Mid Florida Hematology and Oncology
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Winship Cancer Institute of Emory University
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Investigative Clinical Research of Indiana
  • Michigan
    • Grand Rapids, Michigan, United States, 49503
      • Cancer & Hematology Centers of Western Michigan
  • New York
    • Bronx, New York, United States, 10469
      • New York Cancer & Blood Specialists
    • Buffalo, New York, United States, 14263
      • Roswell Park Cancer Institute
    • East Setauket, New York, United States, 11733
      • New York Cancer & Blood Specialists
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Oregon
    • Corvallis, Oregon, United States, 97330
      • Good Samaritan Hospital
  • Tennessee
    • Knoxville, Tennessee, United States, 37920
      • University of Tennessee Medical Center
    • Memphis, Tennessee, United States, 38120
      • Baptist Cancer Center
  • Texas
    • Tyler, Texas, United States, 75701
      • HOPE Cancer Center of East Texas

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• At least 18 years of age
• Primary diagnosis of PNH confirmed by high-sensitivity flow cytometry
• On treatment with eculizumab. Dose of eculizumab must have been stable for at least 3 months prior to the Screening Visit
• Hb <10.5 g/dL at the Screening Visit
• Absolute reticulocyte count > 1.0x ULN at the Screening Visit
• Platelet count of >50,000/mm3 at the Screening Visit
• Absolute neutrophil count >500/mm3 at the Screening Visit
• Vaccination against Neisseria meningitides types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing, or within 14 days after starting treatment with APL-2. Unless documented evidence exists that subjects are non-responders to vaccination as evidenced by titers or display titer levels within acceptable local limits
• Women of child-bearing potential (WOCBP) must have a negative pregnancy test at the Screening and Day -28 Visit (Run-in Period) and must agree to use protocol defined methods of contraception for the duration of the study and 90 days after their last dose of study drug
• Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study and 90 days after their last dose of study drug
• Willing and able to give informed consent
• Willing and able to self-administer APL-2 (administration by caregiver will be allowed)
• Have a body mass index (BMI) ≤35.0 kg/m2

Exclusion Criteria:

• Active bacterial infection that has not resolved within 14 week of Day -28 (first dose of APL-2)
• Receiving iron, folic acid, vitamin B12 and EPO, unless the dose is stable, in the 4 weeks prior to Screening
• Hereditary complement deficiency
• History of bone marrow transplantation
• History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration
• Participation in any other investigational drug trial or exposure to other investigational agent within 30 days or 5 half-lives (whichever is longer)
• Currently breast-feeding women
• Inability to cooperate or any condition that, in the opinion of the investigator, could increase the subject's risk of participating in the study or confound the outcome of the study

This study includes cardiac safety evaluations. The following cardiac eligibility criteria are necessary to avoid confounding the cardiac safety outcomes:

• History or family history of Long QT Syndrome or torsade de pointes, unexplained syncope, syncope from an uncorrected cardiac etiology, or family history of sudden death
• Myocardial infarction, CABG, coronary or cerebral artery stenting and /or angioplasty, stroke, cardiac surgery, or hospitalization for congestive heart failure within 3 months or greater than Class 2 Angina Pectoris or NYHA Heart Failure Class >2
• QTcF > 470 ms, PR > 280 ms
• Mobitz II 2nd degree AV Block, 2:1 AV Block, High Grade AV Block, or Complete Heart Block unless the patient has an implanted pacemaker or implantable cardiac defibrillator (ICD) with backup pacing capabilities
• Receiving Class 1 or Class 3 antiarrhythmic agents, or arsenic, methadone, ondansetron or pentamidine at screening
• Receiving any other QTc-prolonging drugs (see Appendix 4 in Section 19.4), at a stable dose for less than 3 weeks prior to dosing
• Receiving prophylactic ciprofloxacin, erythromycin or azithromycin for less than one week prior to the first dose of study medication (must have a repeat screening ECG after one week of prophylactic antibiotics with QTcF < 470 ms)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pegcetacoplan; 1080 mg pegcetacoplan administered subcutaneously twice-weekly or every three days.	Drug: Pegcetacoplan; Complement (C3) Inhibitor; Other Names: APL-2; Drug: Soliris; Complement (C5) Inhibitor
Active Comparator: Eculizumab; Complement (C5) Inhibitor.	Drug: Pegcetacoplan; Complement (C3) Inhibitor; Other Names: APL-2; Drug: Soliris; Complement (C5) Inhibitor

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Least Squares (LS) Mean Change From Baseline to Week 16 in Hemoglobin (Hb) Level During the RCP	Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.	Baseline and Week 16

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Subjects Who Did Not Require a Transfusion (Transfusion Avoidance) During the RCP	Subjects who experienced more than 1 transfusion during the RCP are only counted once. Subjects who did not have a transfusion but withdrew before Week 16 were considered as having a transfusion in the analysis of transfusion avoidance.	Day 1 to Week 16
LS Mean Change From Baseline to Week 16 in Absolute Reticulocyte Count (ARC) During the RCP	Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.	Baseline and Week 16
LS Mean Change From Baseline to Week 16 in Lactate Dehydrogenase (LDH) Level During the RCP	Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.	Baseline and Week 16
LS Mean Change From Baseline to Week 16 in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Score During the RCP	The FACIT-fatigue scale version 4 is a 13-item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. A higher score corresponds to a higher quality of life (QoL). Baseline was the last available, nonmissing observation before taking the first dose of pegcetacoplan. Data collected after transfusion is excluded from analysis.	Baseline and Week 16
Percentage of Subjects Who Achieved a Hb Response in the Absence of Transfusions at Week 16	Hb response was defined as an increase of at least 1 g/dL in Hb from Baseline at Week 16. Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.	Baseline and Week 16
Percentage of Subjects Who Achieved Reticulocyte Normalization in the Absence of Transfusions at Week 16	Reticulocyte normalization was defined as the ARC being below the upper limit of the gender-specific normal range at Week 16, censored for transfusions. Subjects who received a transfusion between Day 1 and Week 16 or withdrew without providing efficacy data at Week 16 were classified as nonresponders.	Week 16
Percentage of Subjects Who Achieved Hb Normalization in the Absence of Transfusions at Week 16	Hb normalization was defined as the Hb level being above the lower limit of the normal range at Week 16, censored for transfusions. Subjects who received a transfusion between Day 1 and Week 16 or withdrew without providing efficacy data at Week 16 are classified as nonnormalization.	Week 16
LS Mean Change From Baseline to Week 16 in Indirect Bilirubin Level During the RCP	Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.	Baseline and Week 16
LS Mean Change From Baseline to Week 16 in Haptoglobin Level During the RCP	Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.	Baseline and Week 16
LS Mean Change From Baseline to Week 16 in Linear Analog Scale Assessment (LASA) Scores During the RCP	The LASA consists of 3 items, where the respondents were asked to rate their perceived level of functioning. Specific domains included activity level, ability to carry out daily activities, and an item for overall QoL. Their level of functioning was reported on a 0 to 100 scale with 0 indicates "As low as could be" and 100 indicates "As high as could be". The combined score ranged from 0 to 300, with higher scores corresponding to a higher QoL.	Baseline and Week 16
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP	The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are "Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score.	Baseline and Week 16
Total Number of PRBC Units Transfused During the RCP	Subjects who withdrew during the RCP before Week 16 will have their number of units of PRBC estimated from the duration they were in the study.	Day 1 to Week 16
Mean Change From Baseline to Week 48 in Hb Level During the Treatment Period	Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.	Baseline and Week 48
Mean Change From Week 17 to Week 48 in Hb Level During the Open-label Period	Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.	Week 17 and Week 48
Mean Change From Baseline to Week 48 in ARC During the Treatment Period	Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.	Baseline and Week 48
Mean Change From Week 17 to Week 48 in ARC During the Open-label Period	Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.	Week 17 and Week 48
Mean Change From Baseline to Week 48 in LDH Level During the Treatment Period	Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.	Baseline and Week 48
Mean Change From Week 17 to Week 48 in LDH Level During the Open-label Period	Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.	Week 17 and Week 48
Mean Change From Baseline to Week 48 in FACIT-Fatigue Scale Score During the Treatment Period	The FACIT-fatigue scale version 4 is a 13-item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. A higher score corresponds to a higher QoL. Baseline was the last available, nonmissing observation before taking the first dose of pegcetacoplan. Data collected after transfusion is excluded from analysis.	Baseline and Week 48
Mean Change From Week 17 to Week 48 in FACIT-Fatigue Scale Score During the Open-label Period	The FACIT-fatigue scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. Higher score corresponds to a higher QoL.	Week 17 and Week 48
Mean Change From Baseline to Week 48 in LASA Scores During the Treatment Period	The LASA consists of 3 items, where the respondents were asked to rate their perceived level of functioning. Specific domains included activity level, ability to carry out daily activities, and an item for overall QoL. Their level of functioning was reported on a 0 to 100 scale with 0 indicates "As low as could be" and 100 indicates "As high as could be". The combined score ranged from 0 to 300, with higher scores corresponding to a higher QoL.	Baseline and Week 48
Mean Change From Week 17 to Week 48 in LASA Scores During the Open-label Period	The FACIT-fatigue scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. Higher score corresponds to a higher QoL.	Week 17 and Week 48
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period	The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are 'Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score.	Baseline and Week 48
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period	The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are 'Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score.	Week 17 and Week 48
Total Number of PRBC Units Transfused During the Open-Label Period	Number of units of PRBC transfused to subjects in the open-label period are reported.	Week 17 to Week 48

Collaborators and Investigators

• Sponsor: Apellis Pharmaceuticals, Inc.

Publications and helpful links

General Publications

• de Latour RP, Szer J, Weitz IC, Röth A, Höchsmann B, Panse J, Usuki K, Griffin M, Kiladjian JJ, de Castro CM, Nishimori H, Ajayi T, Al-Adhami M, Deschatelets P, Francois C, Grossi F, Risitano AM, Hillmen P. Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase 3, active-comparator, controlled trial. Lancet Haematol. 2022 Sep;9(9):e648-e659. doi: 10.1016/S2352-3026(22)00210-1.
• Cella D, Sarda SP, Hsieh R, Fishman J, Hakimi Z, Hoffman K, Al-Adhami M, Nazir J, Cutts K, Lenderking WR. Changes in hemoglobin and clinical outcomes drive improvements in fatigue, quality of life, and physical function in patients with paroxysmal nocturnal hemoglobinuria: post hoc analyses from the phase III PEGASUS study. Ann Hematol. 2022 Sep;101(9):1905-1914. doi: 10.1007/s00277-022-04887-8. Epub 2022 Jul 23.
• Hakimi Z, Wilson K, McAughey E, Pochopien M, Wojciechowski P, Toumi M, Knight C, Sarda SP, Patel N, Wiseman C, de Castro NP, Nazir J, Kelly RJ. The cost-effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting. J Comp Eff Res. 2022 Sep;11(13):969-985. doi: 10.2217/cer-2022-0076. Epub 2022 Jul 7.
• Bhak RH, Mody-Patel N, Baver SB, Kunzweiler C, Yee CW, Sundaresan S, Swartz N, Duh MS, Krishnan S, Sarda SP. Comparative effectiveness of pegcetacoplan versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a matching-adjusted indirect comparison. Curr Med Res Opin. 2021 Nov;37(11):1913-1923. doi: 10.1080/03007995.2021.1971182. Epub 2021 Sep 3.
• Hillmen P, Szer J, Weitz I, Roth A, Hochsmann B, Panse J, Usuki K, Griffin M, Kiladjian JJ, de Castro C, Nishimori H, Tan L, Hamdani M, Deschatelets P, Francois C, Grossi F, Ajayi T, Risitano A, Peffault de la Tour R. Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2021 Mar 18;384(11):1028-1037. doi: 10.1056/NEJMoa2029073.

Study record dates

Study Major Dates

• Study Start (Actual): June 14, 2018
• Primary Completion (Actual): November 14, 2019
• Study Completion (Actual): August 13, 2020

Study Registration Dates

• First Submitted: April 10, 2018
• First Submitted That Met QC Criteria: April 10, 2018
• First Posted (Actual): April 18, 2018

Study Record Updates

• Last Update Posted (Actual): March 25, 2022
• Last Update Submitted That Met QC Criteria: March 1, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Urological Manifestations; Bone Marrow Diseases; Hematologic Diseases; Urination Disorders; Anemia; Proteinuria; Anemia, Hemolytic; Myelodysplastic Syndromes; Hemoglobinuria; Hemoglobinuria, Paroxysmal; Physiological Effects of Drugs; Immunosuppressive Agents; Immunologic Factors; Complement Inactivating Agents; Eculizumab
• Other Study ID Numbers: APL2-302

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No