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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04170023
Registration number
NCT04170023
Ethics application status
Date submitted
6/11/2019
Date registered
20/11/2019
Date last updated
7/06/2024
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 In HgB Relative To Baseline
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Assessment method [1]
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Timepoint [1]
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Week 12
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Secondary outcome [1]
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Number Of Patients Who Have Transfusion Avoidance
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Assessment method [1]
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Timepoint [1]
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Up to Week 12
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Secondary outcome [2]
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Number Of RBC Units Transfused and Transfusion Instances
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Assessment method [2]
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Timepoint [2]
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Up to Week 12
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Secondary outcome [3]
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Change In LDH Relative To Baseline
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Assessment method [3]
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Timepoint [3]
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Week 12
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Secondary outcome [4]
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Change From Baseline In Absolute Reticulocyte Count
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Assessment method [4]
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Timepoint [4]
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Week 12
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Secondary outcome [5]
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Change From Baseline In Direct Bilirubin
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Assessment method [5]
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Timepoint [5]
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Week 12
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Secondary outcome [6]
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Change From Baseline In Total Bilirubin
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Assessment method [6]
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Timepoint [6]
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Week 12
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Secondary outcome [7]
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Change From Baseline In PNH RBC Clone Size
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Assessment method [7]
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Timepoint [7]
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Week 12
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Secondary outcome [8]
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Change From Baseline In C3 Complement Protein Fragment Deposition On PNH RBCs
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Assessment method [8]
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Timepoint [8]
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Week 12
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Secondary outcome [9]
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Incidence of TEAEs, SAEs, and Events Leading To Discontinuation Of Study Medication
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Assessment method [9]
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Timepoint [9]
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Through Study Completion
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Secondary outcome [10]
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Change in HgB Relative To Baseline
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Assessment method [10]
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Timepoint [10]
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Long-term Extension Period
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Secondary outcome [11]
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Change in LDH Relative To Baseline
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Assessment method [11]
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Timepoint [11]
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Long-term Extension Period
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Secondary outcome [12]
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Change in FACIT Fatigue Scale (Version 4) Scores Relative To Baseline
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Assessment method [12]
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Timepoint [12]
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Week 12, 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
Terminated
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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
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Canada
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Ontario
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Canada
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Quebec
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Italy
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Avellino
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Italy
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Firenze
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Italy
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Florence
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Korea, Republic of
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Seoul
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New Zealand
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Auckland
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New Zealand
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Christchurch
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New Zealand
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Grafton
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Spain
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Albacete
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Turkey
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Istanbul
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Turkey
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Izmir
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United Kingdom
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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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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/ct2/show/NCT04170023
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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 public queries
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04170023
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