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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
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
Secondary ID [1] 0 0
2019-003830-17
Secondary ID [2] 0 0
ACH228-110
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria (PNH) 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Hgb at Week 12
Timepoint [1] 0 0
Baseline, Week 12
Secondary outcome [1] 0 0
Number of Participants Who Had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050
Timepoint [1] 0 0
Baseline up to Week 12
Secondary outcome [2] 0 0
Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment
Timepoint [2] 0 0
Baseline up to Week 12
Secondary outcome [3] 0 0
Number of Transfusion Instances During 12 Weeks of Treatment
Timepoint [3] 0 0
Baseline up to Week 12
Secondary outcome [4] 0 0
Change From Baseline in Lactate Dehydrogenase (LDH) at Week 12
Timepoint [4] 0 0
Baseline, Week 12
Secondary outcome [5] 0 0
Change From Baseline in Absolute Reticulocyte Count at Week 12
Timepoint [5] 0 0
Baseline, Week 12
Secondary outcome [6] 0 0
Change From Baseline in Direct and Total Bilirubin at Week 12
Timepoint [6] 0 0
Baseline, Week 12
Secondary outcome [7] 0 0
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size at Week 12
Timepoint [7] 0 0
Baseline, Week 12
Secondary outcome [8] 0 0
Change From Baseline in Component 3 (C3) Fragment Deposition on PNH RBCs at Week 12
Timepoint [8] 0 0
Baseline, Week 12
Secondary outcome [9] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading to Discontinuation of Study Medication
Timepoint [9] 0 0
From first dose of study drug up to Week 217
Secondary outcome [10] 0 0
Change From Baseline in Hgb at the End of Treatment (EOT) During the LTE Period
Timepoint [10] 0 0
Baseline, EOT visit (Maximum exposure: 213.4 weeks)
Secondary outcome [11] 0 0
Change From Baseline in LDH at the EOT During the LTE Period
Timepoint [11] 0 0
Baseline, EOT visit (Maximum exposure: 213.4 weeks)
Secondary outcome [12] 0 0
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale (Version 4) Total Score at Week 12
Timepoint [12] 0 0
Baseline, Week 12
Secondary outcome [13] 0 0
Change From Baseline in FACIT-Fatigue Scale (Version 4) Total Score at the EOT During the LTE Period
Timepoint [13] 0 0
Baseline, EOT visit (Maximum exposure: 213.4 weeks)

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

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/12/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
20/03/2024
Sample size
Target
Accrual to date
Final
29
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Ontario
Country [2] 0 0
Canada
State/province [2] 0 0
Quebec
Country [3] 0 0
Italy
State/province [3] 0 0
Avellino
Country [4] 0 0
Italy
State/province [4] 0 0
Firenze
Country [5] 0 0
Korea, Republic of
State/province [5] 0 0
Seoul
Country [6] 0 0
New Zealand
State/province [6] 0 0
Christchurch
Country [7] 0 0
New Zealand
State/province [7] 0 0
Grafton
Country [8] 0 0
Spain
State/province [8] 0 0
Albacete
Country [9] 0 0
Turkey
State/province [9] 0 0
Istanbul
Country [10] 0 0
Turkey
State/province [10] 0 0
Izmir
Country [11] 0 0
United Kingdom
State/province [11] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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)
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04170023