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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
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
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 In HgB Relative To Baseline
Timepoint [1] 0 0
Week 12
Secondary outcome [1] 0 0
Number Of Patients Who Have Transfusion Avoidance
Timepoint [1] 0 0
Up to Week 12
Secondary outcome [2] 0 0
Number Of RBC Units Transfused and Transfusion Instances
Timepoint [2] 0 0
Up to Week 12
Secondary outcome [3] 0 0
Change In LDH Relative To Baseline
Timepoint [3] 0 0
Week 12
Secondary outcome [4] 0 0
Change From Baseline In Absolute Reticulocyte Count
Timepoint [4] 0 0
Week 12
Secondary outcome [5] 0 0
Change From Baseline In Direct Bilirubin
Timepoint [5] 0 0
Week 12
Secondary outcome [6] 0 0
Change From Baseline In Total Bilirubin
Timepoint [6] 0 0
Week 12
Secondary outcome [7] 0 0
Change From Baseline In PNH RBC Clone Size
Timepoint [7] 0 0
Week 12
Secondary outcome [8] 0 0
Change From Baseline In C3 Complement Protein Fragment Deposition On PNH RBCs
Timepoint [8] 0 0
Week 12
Secondary outcome [9] 0 0
Incidence of TEAEs, SAEs, and Events Leading To Discontinuation Of Study Medication
Timepoint [9] 0 0
Through Study Completion
Secondary outcome [10] 0 0
Change in HgB Relative To Baseline
Timepoint [10] 0 0
Long-term Extension Period
Secondary outcome [11] 0 0
Change in LDH Relative To Baseline
Timepoint [11] 0 0
Long-term Extension Period
Secondary outcome [12] 0 0
Change in FACIT Fatigue Scale (Version 4) Scores Relative To Baseline
Timepoint [12] 0 0
Week 12, Week 160

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
Terminated
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
Italy
State/province [5] 0 0
Florence
Country [6] 0 0
Korea, Republic of
State/province [6] 0 0
Seoul
Country [7] 0 0
New Zealand
State/province [7] 0 0
Auckland
Country [8] 0 0
New Zealand
State/province [8] 0 0
Christchurch
Country [9] 0 0
New Zealand
State/province [9] 0 0
Grafton
Country [10] 0 0
Spain
State/province [10] 0 0
Albacete
Country [11] 0 0
Turkey
State/province [11] 0 0
Istanbul
Country [12] 0 0
Turkey
State/province [12] 0 0
Izmir
Country [13] 0 0
United Kingdom
State/province [13] 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
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04170023
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04170023