ClinicalTrials.gg
TerminatedNCT04170023Updated Nov 20, 2024Results posted

Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy

A Phase 2 interventional study of ALXN2050 in Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by Alexion Pharmaceuticals, Inc.. Terminated at 11 sites in 7 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-11-20.

Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 2, Interventional, and Treatment

Why this study was terminated
Did not appropriately control IVH; significantly increased rates of BTH and LDH excursions (LDH excursions is defined by LDH values \> X2ULN) compared to ravulizumab.
Phase
Phase 2
Study type
Interventional
Enrollment
29
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
01

Study 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.

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.

02

Conditions studied

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)

Keywords

  • paroxysmal nocturnal hemoglobinuria
  • PNH
  • extravascular hemolysis
  • EVH
  • factor D inhibitor
  • complement
  • C5 inhibitor
  • danicopan
03

In context

Hemoglobinuria, Paroxysmal

191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.

This study's enrollment of 29 is below the median of 33 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.

Browse Hemoglobinuria, Paroxysmal studies →

Lead sponsor

Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.

Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.

First submitted before Alexion Pharmaceuticals, Inc. became part of AstraZeneca.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

  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 ( \<10.5 grams/deciliter [g/dL]).
  5. LDH ≥1.5× .
  6. count ≥30,000/microliter (µL)
  7. (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 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 , or if these patients are on 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 \<30 milliliters/minute/1.73 meters squared and/or are on dialysis.
Terms in these criteria, in plain language(7)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Absolute neutrophil count (ANC)

The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.

It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.

Allogeneic and autologous transplant

Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.

Immunosuppression

A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.

Read whether the line is about a condition or about medicines, and which medicines and doses it means.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Single group
Masking
None (open label)
Enrollment
29 participants (actual)

Study arms

  • 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

    Drug: ALXN2050

Interventions

  • Oral FD inhibitor

    Also known as: ACH-0145228

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Baseline, Week 12

Secondary outcomes

  1. 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.

    Time frame: Baseline up to Week 12

  2. Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment

    Time frame: Baseline up to Week 12

  3. Number of Transfusion Instances During 12 Weeks of Treatment

    Time frame: Baseline up to Week 12

  4. Change From Baseline in Lactate Dehydrogenase (LDH) at Week 12

    Change from Baseline = Serum LDH levels at Week 12 - Baseline Serum LDH levels.

    Time frame: Baseline, Week 12

  5. Change From Baseline in Absolute Reticulocyte Count at Week 12

    Change from Baseline = absolute reticulocyte count at Week 12 - Baseline reticulocyte count.

    Time frame: Baseline, Week 12

  6. Change From Baseline in Direct and Total Bilirubin at Week 12

    Time frame: Baseline, Week 12

  7. 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.

    Time frame: Baseline, Week 12

  8. 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.

    Time frame: Baseline, Week 12

  9. 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.

    Time frame: From first dose of study drug up to Week 217

  10. 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.

    Time frame: Baseline, EOT visit (Maximum exposure: 213.4 weeks)

  11. 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.

    Time frame: Baseline, EOT visit (Maximum exposure: 213.4 weeks)

  12. 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.

    Time frame: Baseline, Week 12

  13. 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.

    Time frame: Baseline, EOT visit (Maximum exposure: 213.4 weeks)

07

Results

Posted Aug 9, 2024

Participant flow

Treatment Period
Participant flow — Treatment Period
MilestoneGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Started12116
Received at least 1 dose of study drug12116
Completed12116
Not completed000
LTE Period
Participant flow — LTE Period
MilestoneGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Started12116
Received at least 1 dose of study drug12116
Completed001
Not completed12115
Withdrew: Withdrawal by subject100
Withdrew: Study termination11115

Outcome measures

PrimaryChange 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.

Time frame:
Baseline, Week 12
Reported as:
Mean · grams/liter
Change From Baseline in Hgb at Week 12
grams/literGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Change From Baseline in Hgb at Week 1235.6 ± 14.7032.5 ± 20.03-3.7 ± 15.77
SecondaryNumber 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.

Time frame:
Baseline up to Week 12
Reported as:
Count of participants · Participants
Number of Participants Who Had Transfusion Avoidance During 12 Weeks of Treatment With ALXN2050
ParticipantsGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Number of Participants Who Had Transfusion Avoidance During 12 Weeks of Treatment With ALXN20509105
SecondaryNumber of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment
Time frame:
Baseline up to Week 12
Reported as:
Mean · RBC units
Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment
RBC unitsGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Number of Red Blood Cell (RBC) Units Transfused During 12 Weeks of Treatment0.6 ± 1.240.1 ± 0.300.8 ± 2.04
SecondaryNumber of Transfusion Instances During 12 Weeks of Treatment
Time frame:
Baseline up to Week 12
Reported as:
Mean · transfusion instances
Number of Transfusion Instances During 12 Weeks of Treatment
transfusion instancesGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Number of Transfusion Instances During 12 Weeks of Treatment0.4 ± 0.900.1 ± 0.300.5 ± 1.22
SecondaryChange From Baseline in Lactate Dehydrogenase (LDH) at Week 12

Change from Baseline = Serum LDH levels at Week 12 - Baseline Serum LDH levels.

Time frame:
Baseline, Week 12
Reported as:
Mean · units/liter
Change From Baseline in Lactate Dehydrogenase (LDH) at Week 12
units/literGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Change From Baseline in Lactate Dehydrogenase (LDH) at Week 12-1310.8 ± 424.89160.2 ± 279.5788.7 ± 265.00
SecondaryChange From Baseline in Absolute Reticulocyte Count at Week 12

Change from Baseline = absolute reticulocyte count at Week 12 - Baseline reticulocyte count.

Time frame:
Baseline, Week 12
Reported as:
Mean · 10^3 cells/microliter (μL)
Change From Baseline in Absolute Reticulocyte Count at Week 12
10^3 cells/microliter (μL)Group 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Change From Baseline in Absolute Reticulocyte Count at Week 12-100.6 ± 72.18-179.0 ± 117.30-24.0 ± 22.10
SecondaryChange From Baseline in Direct and Total Bilirubin at Week 12
Time frame:
Baseline, Week 12
Reported as:
Mean · micromoles (μmol)/liter
Change From Baseline in Direct and Total Bilirubin at Week 12
micromoles (μmol)/literGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Direct Bilirubin-2.1 ± 2.10-6.3 ± 5.160.4 ± 0.89
Total Bilirubin-12.9 ± 10.07-23.3 ± 24.891.5 ± 2.81
SecondaryChange 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.

Time frame:
Baseline, Week 12
Reported as:
Mean · percentage of the total cell population
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size at Week 12
percentage of the total cell populationGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size at Week 1241.9 ± 13.6827.5 ± 21.60-12.0 ± 26.94
SecondaryChange 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.

Time frame:
Baseline, Week 12
Reported as:
Mean · percentage of PNH RBC
Change From Baseline in Component 3 (C3) Fragment Deposition on PNH RBCs at Week 12
percentage of PNH RBCGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Change From Baseline in Component 3 (C3) Fragment Deposition on PNH RBCs at Week 120.0 ± 0.00-388.6 ± 590.080.3 ± 0.35
SecondaryNumber 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.

Time frame:
From first dose of study drug up to Week 217
Reported as:
Count of participants · Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading to Discontinuation of Study Medication
ParticipantsGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Any AEs12106
SAEs752
AEs Leading to Discontinuation of Study Medication110
SecondaryChange 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.

Time frame:
Baseline, EOT visit (Maximum exposure: 213.4 weeks)
Reported as:
Mean · grams/liter
Change From Baseline in Hgb at the End of Treatment (EOT) During the LTE Period
grams/literGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Change From Baseline in Hgb at the End of Treatment (EOT) During the LTE Period44.9 ± 21.0732.5 ± 7.172.4 ± 19.44
SecondaryChange 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.

Time frame:
Baseline, EOT visit (Maximum exposure: 213.4 weeks)
Reported as:
Mean · units/liter
Change From Baseline in LDH at the EOT During the LTE Period
units/literGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Change From Baseline in LDH at the EOT During the LTE Period-1084.1 ± 738.2695.7 ± 223.88-61.4 ± 62.85
SecondaryChange 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.

Time frame:
Baseline, Week 12
Reported as:
Mean · units on a scale
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale (Version 4) Total Score at Week 12
units on a scaleGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale (Version 4) Total Score at Week 1211.2 ± 13.205.1 ± 8.470.2 ± 3.13
SecondaryChange 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.

Time frame:
Baseline, EOT visit (Maximum exposure: 213.4 weeks)
Reported as:
Mean · units on a scale
Change From Baseline in FACIT-Fatigue Scale (Version 4) Total Score at the EOT During the LTE Period
units on a scaleGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Change From Baseline in FACIT-Fatigue Scale (Version 4) Total Score at the EOT During the LTE Period12.6 ± 12.514.4 ± 7.26-3.4 ± 10.26

Adverse events

Collected over From first dose of study drug up to Week 217. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Group 1: Treatment Naive0/12 (0%)7/12 (58.3%)12/12 (100%)
Group 2: Eculizumab Switch0/11 (0%)5/11 (45.5%)10/11 (90.9%)
Group 3: Danicopan Rollover0/6 (0%)2/6 (33.3%)6/6 (100%)
Most frequent serious events
Showing 10 of 15
Most frequent serious events
EventGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
Breakthrough haemolysisBlood and lymphatic system disorders4/122/111/6
Acute kidney injuryRenal and urinary disorders3/121/110/6
Muscle spasmsMusculoskeletal and connective tissue disorders3/120/110/6
ContusionInjury, poisoning and procedural complications0/120/111/6
GastroenteritisInfections and infestations0/121/110/6
Anaphylactic reactionImmune system disorders0/121/110/6
COVID-19Infections and infestations0/121/110/6
Intravascular haemolysisBlood and lymphatic system disorders0/121/110/6
Campylobacter gastroenteritisInfections and infestations1/120/110/6
HaemoglobinuriaRenal and urinary disorders1/120/110/6
Most frequent other events
Showing 10 of 182
Most frequent other events
EventGroup 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan Rollover
FatigueGeneral disorders6/121/113/6
HeadacheNervous system disorders6/123/111/6
COVID-19Infections and infestations4/122/113/6
Upper respiratory tract infectionInfections and infestations6/121/112/6
ArthralgiaMusculoskeletal and connective tissue disorders5/124/111/6
HaemolysisBlood and lymphatic system disorders0/124/110/6
Pain in extremityMusculoskeletal and connective tissue disorders1/124/112/6
HaematuriaRenal and urinary disorders3/124/112/6
NauseaGastrointestinal disorders4/122/111/6
VomitingGastrointestinal disorders4/122/110/6

Baseline characteristics

Full analysis set included all participants who received at least 1 dose of ALXN2050 in this study.

Age, Continuous
Age, Continuous(years)Group 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan RolloverTotal
Mean43.75 ± 17.09942.45 ± 13.41940.50 ± 16.71842.59 ± 15.186
Sex: Female, Male
Sex: Female, Male(Participants)Group 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan RolloverTotal
Female47314
Male84315
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Group 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan RolloverTotal
Hispanic or Latino0000
Not Hispanic or Latino1210628
Unknown or Not Reported0101
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Group 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan RolloverTotal
American Indian or Alaska Native0000
Asian8109
Native Hawaiian or Other Pacific Islander0000
Black or African American0000
White310619
More than one race0000
Unknown or Not Reported1001
HgB
HgB(grams/liter)Group 1: Treatment NaiveGroup 2: Eculizumab SwitchGroup 3: Danicopan RolloverTotal
Mean81.17 ± 11.73191.00 ± 9.602130.67 ± 19.18095.14 ± 22.662
08

Study locations

11 sites
  • Research Site
    Toronto, Ontario M5G2C4, Canada
  • Research Site
    Levis, Quebec G6V 3Z1, Canada
  • Research Site
    Avellino, 83100, Italy
  • Research Site
    Firenze, 50134, Italy
  • Research Site
    Seoul, 06591, Korea, Republic of
  • Research Site
    Christchurch, 8011, New Zealand
  • Research Site
    Grafton, 1010, New Zealand
  • Research Site
    Albacete, 02006, Spain
  • Research Site
    Istanbul, 34093, Turkey
  • Research Site
    Izmir, 35100, Turkey
  • Research Site
    London, SE5 9RS, United Kingdom
09

References and documents

Related links

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: 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.

Supporting information: Study protocol, Statistical analysis plan, Clinical study report

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 20, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT04170023
Responsible party
Sponsor
First posted
Nov 20, 2019
Start date
Dec 16, 2019
Primary completion
Apr 27, 2023
Completion
Mar 20, 2024
Results posted
Aug 9, 2024
Last update
Nov 20, 2024

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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