Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 2NCT04170023
Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
A Phase 2 study testing ALXN2050 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Alexion Pharmaceuticals, Inc. at 11 sites in 7 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- What is being tested?
- ALXN2050
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 11 sitesin 7 countries
- Study dates
- 16 Dec 2019to 20 Mar 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of PNH.
- Male or female, ≥ 18 years of age
- Eligibility Criteria:
- Eligibility Criteria Specific for Group 1:
- PNH Patients who have no history of treatment with any complement inhibitor at any dose.
- PNH Type III erythrocyte or granulocyte clone size ≥10%
Show all 19Show fewer
- Absolute reticulocyte count ≥100×10^9/liter [L].
- Anemia (Hgb <10.5 grams/deciliter [g/dL]).
- LDH ≥1.5× upper limit of normal.
- Platelet count ≥30,000/microliter (µL)
- Absolute neutrophil count (ANC) ≥750/ µL.
- Eligibility Criteria Specific for Group 2:
- Stable background regimen of at least 24 weeks for eculizumab without change in dose or interval for at least the past 8 weeks
- Anemia (Hgb <10 g/dL)
- Absolute reticulocyte count ≥100×10^9/L
- Platelet count ≥30,000/µL
- Absolute neurophil count (ANC) ≥750/ µL
- Eligibility Criteria Specific for Group 3:
- 1. Patient received danicopan during Study ACH471-103
Who cannot join
- History of a major organ transplant or hematopoietic stem cell/marrow transplant .
- Known aplastic anemia or other bone marrow failure that requires HSCT, or if these patients are on immunosuppressive agents for less than 24 weeks.
- Known underlying bleeding disorders or any other conditions leading to anemia not primarily associated with PNH.
- Estimated glomerular filtration rate <30 milliliters/minute/1.73 meters squared and/or are on dialysis.
Trial locations and contacts
11 sites in 7 countries.
11 sites in 7 countries
Canada2 sites
Research Site
Lévis, Quebec, G6V 3Z1
Research Site
Toronto, Ontario, M5G2C4
New Zealand2 sites
Research Site
Christchurch, 8011
Research Site
Grafton, 1010
Turkey (Türkiye)2 sites
Research Site
Istanbul, 34093
Research Site
Izmir, 35100
About this study
Official 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
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.
Read the detailed description
Experimental: Open-label ALXN2050 Monotherapy orally
Group 1: Patients with PNH who are treatment naïve
Group 2: Patients 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) who will switch to ALXN2050 monotherapy
Group 3: Patients with PNH receiving danicopan monotherapy in study ACH471-103 will switch to ALXN2050 monotherapy
After signing the informed consent form, participants will enter the screening period. During the Screening Period, eligibility and screening assessments will be performed. Screening assessments may be spread over more than one visit if necessary. At the baseline visit, screened participants who continue to meet eligibility criteria will enter the Treatment Period.
The treatment phase will be followed by a long-term extension phase, where ALXN2050 will continue to be administered.
Blood will be collected to assess the efficacy endpoints, such as, change in hemoglobin (Hgb), lactate dehydrogenase (LDH), and other measures of hemolysis. Safety and transfusion requirements will also be assessed.
Participants will continue on treatment past 12 weeks in a long-term extension portion of the trial.
- Being tested
- ALXN2050
- Keywords
- paroxysmal nocturnal hemoglobinuria, PNH, extravascular hemolysis, EVH, factor D inhibitor, complement, C5 inhibitor, danicopan
- Medical subject headings
- Hemoglobinuria, Paroxysmal, Hemolysis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN2050Drug
Also called ACH-0145228
Oral FD inhibitor
| Study group | Role | What they receive |
|---|---|---|
| Open-label ALXN2050 Monotherapy | Experimental | 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 Receives: Drug: ALXN2050 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Hgb at Week 12
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.
Measured: Baseline, Week 12
Secondary outcomes (13)
Number of Participants Who Had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050
Transfusion avoidance: participants remained transfusion-free and did not require a transfusion during the period of interest.
Measured: Baseline up to Week 12
Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment
Measured: Baseline up to Week 12
Number of Transfusion Instances During 12 Weeks of Treatment
Measured: Baseline up to Week 12
Change From Baseline in Lactate Dehydrogenase (LDH) at Week 12
Change from Baseline = Serum LDH levels at Week 12 - Baseline Serum LDH levels.
Measured: Baseline, Week 12
Change From Baseline in Absolute Reticulocyte Count at Week 12
Change from Baseline = absolute reticulocyte count at Week 12 - Baseline reticulocyte count.
Measured: Baseline, Week 12
Change From Baseline in Direct and Total Bilirubin at Week 12
Measured: Baseline, Week 12
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size at Week 12
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.
Measured: Baseline, Week 12
Change From Baseline in Component 3 (C3) Fragment Deposition on PNH RBCs at Week 12
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.
Measured: Baseline, Week 12
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading to Discontinuation of Study Medication
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.
Measured: From first dose of study drug up to Week 217
Change From Baseline in Hgb at the End of Treatment (EOT) During the LTE Period
Hgb baseline was defined as the lowest Hgb value observed between and including screening and first dose date. Change from Baseline = Hgb at the EOT visit - Baseline Hgb.
Measured: Baseline, EOT visit (Maximum exposure: 213.4 weeks)
Change From Baseline in LDH at the EOT During the LTE Period
Change from Baseline = Serum LDH levels at the EOT visit - Baseline Serum LDH levels.
Measured: Baseline, EOT visit (Maximum exposure: 213.4 weeks)
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale (Version 4) Total Score at Week 12
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.
Measured: Baseline, Week 12
Change From Baseline in FACIT-Fatigue Scale (Version 4) Total Score at the EOT During the LTE Period
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.
Measured: Baseline, EOT visit (Maximum exposure: 213.4 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2019
- Study start16 Dec 2019
- Primary completion27 Apr 2023
- Study completion20 Mar 2024
Why it stopped: Did not appropriately control IVH; significantly increased rates of BTH and LDH excursions (LDH excursions is defined by LDH values \> X2ULN) compared to ravulizumab.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)Studies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04170023
- Sponsor's protocol ID
- ACH228-110
- Other IDs
- 2019-003830-17, 2019
- Status last verified
- Oct 2024
- First submitted
- 6 Nov 2019
- Last updated
- 20 Nov 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Publications
Kulasekararaj A, Browett P, Risitano AM, Patriquin CJ, Yenerel MN, Marceau D, Sahin F, Algarra JL, Ogawa M, Yu J, Cross NB, Notaro R, Lee JW, Brodsky RA. Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria. Blood Adv. 2026 Feb 10;10(3):540-554. doi: 10.1182/bloodadvances.2025017731.
PubMed 41124654
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT04170023
- Acronym / sponsor ID
- ACH228-110
- Keywords
- paroxysmal nocturnal hemoglobinuria, PNH, extravascular hemolysis, EVH, factor D inhibitor, complement, C5 inhibitor, danicopan
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Did not appropriately control IVH; significantly increased rates of BTH and LDH excursions (LDH excursions is defined by LDH values \> X2ULN) compared to ravulizumab.
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 16 Dec 2019 (actual)
- Primary completion
- 27 Apr 2023 (actual)
- Study completion
- 20 Mar 2024 (actual)
- First posted
- 20 Nov 2019 (actual)
- Results
- Posted 9 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Experimental: Open-label ALXN2050 Monotherapy orally
Group 1: Patients with PNH who are treatment naïve
Group 2: Patients 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) who will switch to ALXN2050 monotherapy
Group 3: Patients with PNH receiving danicopan monotherapy in study ACH471-103 will switch to ALXN2050 monotherapy
After signing the informed consent form, participants will enter the screening period. During the Screening Period, eligibility and screening assessments will be performed. Screening assessments may be spread over more than one visit if necessary. At the baseline visit, screened participants who continue to meet eligibility criteria will enter the Treatment Period.
The treatment phase will be followed by a long-term extension phase, where ALXN2050 will continue to be administered.
Blood will be collected to assess the efficacy endpoints, such as, change in hemoglobin (Hgb), lactate dehydrogenase (LDH), and other measures of hemolysis. Safety and transfusion requirements will also be assessed.
Participants will continue on treatment past 12 weeks in a long-term extension portion of the trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open-label ALXN2050 Monotherapy | Experimental | Drug: ALXN2050Experimental: 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Diagnosis of PNH.
- Male or female, ≥ 18 years of age
- PNH Patients who have no history of treatment with any complement inhibitor at any dose.
- PNH Type III erythrocyte or granulocyte clone size ≥10%
- Absolute reticulocyte count ≥100×10^9/liter [L].
Key exclusion criteria (4 listed)
- History of a major organ transplant or hematopoietic stem cell/marrow transplant .
- Known aplastic anemia or other bone marrow failure that requires HSCT, or if these patients are on immunosuppressive agents for less than 24 weeks.
- Known underlying bleeding disorders or any other conditions leading to anemia not primarily associated with PNH.
- Estimated glomerular filtration rate <30 milliliters/minute/1.73 meters squared and/or are on dialysis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALXN2050Also: ACH-0145228Oral FD inhibitor | Drug | Open-label ALXN2050 Monotherapy |
Primary outcome
Change From Baseline in Hgb at Week 12
Time frame: Baseline, Week 12
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.
13 secondary outcomes
Number of Participants Who Had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050
Time frame: Baseline up to Week 12
Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment
Time frame: Baseline up to Week 12
Number of Transfusion Instances During 12 Weeks of Treatment
Time frame: Baseline up to Week 12
Change From Baseline in Lactate Dehydrogenase (LDH) at Week 12
Time frame: Baseline, Week 12
Change From Baseline in Absolute Reticulocyte Count at Week 12
Time frame: Baseline, Week 12
Change From Baseline in Direct and Total Bilirubin at Week 12
Time frame: Baseline, Week 12
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size at Week 12
Time frame: Baseline, Week 12
Change From Baseline in Component 3 (C3) Fragment Deposition on PNH RBCs at Week 12
Time frame: Baseline, Week 12
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading to Discontinuation of Study Medication
Time frame: From first dose of study drug up to Week 217
Change From Baseline in Hgb at the End of Treatment (EOT) During the LTE Period
Time frame: Baseline, EOT visit (Maximum exposure: 213.4 weeks)
Change From Baseline in LDH at the EOT During the LTE Period
Time frame: Baseline, EOT visit (Maximum exposure: 213.4 weeks)
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale (Version 4) Total Score at Week 12
Time frame: Baseline, Week 12
Change From Baseline in FACIT-Fatigue Scale (Version 4) Total Score at the EOT During the LTE Period
Time frame: Baseline, EOT visit (Maximum exposure: 213.4 weeks)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41124654Kulasekararaj A, Browett P, Risitano AM, Patriquin CJ, Yenerel MN, Marceau D, Sahin F, Algarra JL, Ogawa M, Yu J, Cross NB, Notaro R, Lee JW, Brodsky RA. Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria. Blood Adv. 2026 Feb 10;10(3):540-554. doi: 10.1182/bloodadvances.2025017731.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ALXN2050 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Alexion Pharmaceuticals, Inc. at 11 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 20 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Canada, Italy, New Zealand, Turkey (Türkiye), South Korea and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Hgb at Week 12 (time frame: Baseline, Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04170023, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Nov 2024.