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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04170023
Registration number
NCT04170023
Ethics application status
Date submitted
6/11/2019
Date registered
20/11/2019
Titles & IDs
Public title
Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
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Scientific title
A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ALXN2050 (ACH-0145228) in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy
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Secondary ID [1]
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ACH228-110
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Condition category
Condition code
Blood
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Haematological diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - ALXN2050
Experimental: Open-label ALXN2050 Monotherapy - Experimental: Open-label ALXN2050 Monotherapy ALXN2050 orally administered
Group 1: Patients with PNH who are treatment naïve
Group 2: Patient with PNH who have received complement component 5 (C5) inhibition with eculizumab for at least 6 months, who continue to experience anemia and reticulocytes above the upper limit of normal (ULN)
Group 3: Patients with PNH who have received danicopan monotherapy during study ACH471-103
Treatment: Drugs: ALXN2050
Oral FD inhibitor
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline in Hgb at Week 12
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Assessment method [1]
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Hgb baseline was defined as the lowest Hgb value observed between and including screening and first dose date. To address the impact of transfusion, Hgb values collected within 4 weeks after transfusion were not included in the primary efficacy analysis. Change from Baseline = Hgb at Week 12 - Baseline Hgb.
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Timepoint [1]
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Baseline, Week 12
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Secondary outcome [1]
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Number of Participants Who Had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050
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Assessment method [1]
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Transfusion avoidance: participants remained transfusion-free and did not require a transfusion during the period of interest.
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Timepoint [1]
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Baseline up to Week 12
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Secondary outcome [2]
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Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment
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Assessment method [2]
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Timepoint [2]
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Baseline up to Week 12
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Secondary outcome [3]
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Number of Transfusion Instances During 12 Weeks of Treatment
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Assessment method [3]
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Timepoint [3]
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Baseline up to Week 12
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Secondary outcome [4]
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Change From Baseline in Lactate Dehydrogenase (LDH) at Week 12
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Assessment method [4]
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Change from Baseline = Serum LDH levels at Week 12 - Baseline Serum LDH levels.
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Timepoint [4]
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Baseline, Week 12
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Secondary outcome [5]
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Change From Baseline in Absolute Reticulocyte Count at Week 12
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Assessment method [5]
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Change from Baseline = absolute reticulocyte count at Week 12 - Baseline reticulocyte count.
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Timepoint [5]
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Baseline, Week 12
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Secondary outcome [6]
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Change From Baseline in Direct and Total Bilirubin at Week 12
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Assessment method [6]
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Timepoint [6]
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Baseline, Week 12
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Secondary outcome [7]
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Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size at Week 12
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Assessment method [7]
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The PNH RBC clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 12 - Baseline PNH clone size.
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Timepoint [7]
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Baseline, Week 12
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Secondary outcome [8]
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Change From Baseline in Component 3 (C3) Fragment Deposition on PNH RBCs at Week 12
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Assessment method [8]
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C3 fragment deposition on PNH RBC was used as a marker of intra and extravascular hemolysis. Data are presented for the change from baseline to Week 12 in percentage of PNH RBCs with C3 fragment deposition.
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Timepoint [8]
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Baseline, Week 12
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Secondary outcome [9]
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Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading to Discontinuation of Study Medication
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Assessment method [9]
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An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, resulted in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), or an important medical event or reaction. A TEAE was defined as an AE that emerged during treatment, had been absent prior to treatment, or worsened relative to the pretreatment state. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
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Timepoint [9]
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From first dose of study drug up to Week 217
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Secondary outcome [10]
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Change From Baseline in Hgb During the LTE Period
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Assessment method [10]
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Timepoint [10]
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Baseline, Week 160
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Secondary outcome [11]
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Change From Baseline in LDH During the LTE Period
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Assessment method [11]
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Timepoint [11]
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Baseline, Week 160
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Secondary outcome [12]
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Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale (Version 4) Score at Week 12
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Assessment method [12]
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The FACIT-Fatigue scale is a collection of quality-of-life questionnaires pertaining to the management of fatigue symptoms due to chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores ranged from 0 to 52, with higher score indicating better quality of life.
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Timepoint [12]
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Baseline, Week 12
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Secondary outcome [13]
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Change From Baseline in FACIT-Fatigue Scale (Version 4) Scores at Week 160
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Assessment method [13]
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The FACIT-Fatigue scale is a collection of quality-of-life questionnaires pertaining to the management of fatigue symptoms due to chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores ranged from 0 to 52, with higher score indicating better quality of life.
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Timepoint [13]
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Baseline, Week 160
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Eligibility
Key inclusion criteria
Key
1. Diagnosis of PNH.
2. Male or female, = 18 years of age
Eligibility Criteria:
Eligibility Criteria Specific for Group 1:
1. PNH Patients who have no history of treatment with any complement inhibitor at any dose.
2. PNH Type III erythrocyte or granulocyte clone size =10%
3. Absolute reticulocyte count =100×10^9/liter [L].
4. Anemia (Hgb <10.5 grams/deciliter [g/dL]).
5. LDH =1.5× upper limit of normal.
6. Platelet count =30,000/microliter (µL)
7. Absolute neutrophil count (ANC) =750/ µL.
Eligibility Criteria Specific for Group 2:
1. Stable background regimen of at least 24 weeks for eculizumab without change in dose or interval for at least the past 8 weeks
2. Anemia (Hgb <10 g/dL)
3. Absolute reticulocyte count =100×10^9/L
4. Platelet count =30,000/µL
5. Absolute neurophil count (ANC) =750/ µL
Eligibility Criteria Specific for Group 3:
1. Patient received danicopan during Study ACH471-103
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. History of a major organ transplant or hematopoietic stem cell/marrow transplant .
2. Known aplastic anemia or other bone marrow failure that requires HSCT, or if these patients are on immunosuppressive agents for less than 24 weeks.
3. Known underlying bleeding disorders or any other conditions leading to anemia not primarily associated with PNH.
4. Estimated glomerular filtration rate <30 milliliters/minute/1.73 meters squared and/or are on dialysis.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
16/12/2019
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
20/03/2024
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Sample size
Target
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Accrual to date
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Final
29
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
Country [1]
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Canada
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State/province [1]
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Ontario
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Country [2]
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Canada
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State/province [2]
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Quebec
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Country [3]
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Italy
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State/province [3]
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Avellino
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Country [4]
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Italy
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State/province [4]
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Firenze
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Country [5]
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Korea, Republic of
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State/province [5]
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Seoul
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Country [6]
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New Zealand
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State/province [6]
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Christchurch
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Country [7]
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New Zealand
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State/province [7]
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Grafton
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Country [8]
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Spain
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State/province [8]
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Albacete
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Country [9]
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Turkey
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State/province [9]
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Istanbul
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Country [10]
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Turkey
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State/province [10]
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Izmir
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Country [11]
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United Kingdom
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State/province [11]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Alexion Pharmaceuticals, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The study will evaluate the efficacy and safety of the oral Factor D (FD) inhibitor ALXN2050 (ACH-0145228) monotherapy in patients with PNH that are treatment naïve, or patients currently treated with eculizumab who still experience anemia and reticulocytosis, or patients currently treated with ALXN2040 (danicopan) as monotherapy. After signing consent, participants will have periodic visits through Week 12, at which time the primary endpoint and key secondary assessments will be analyzed. Participants will continue on treatment past 12 weeks into a long-term extension portion of the trial.
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Trial website
https://clinicaltrials.gov/study/NCT04170023
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Phone
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Fax
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Email
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
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What supporting documents are/will be available?
No Supporting Document Provided
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Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/23/NCT04170023/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/23/NCT04170023/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT04170023