Warm Autoimmune Hemolytic AnemiaPhase 2NCT04256148
ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia
A Phase 2 study testing ALXN1830 in people with Warm Autoimmune Hemolytic Anemia, run by Alexion Pharmaceuticals, Inc. at 1 site in United States.
Trial summary
The main objective of the study is to evaluate the safety and efficacy of ALXN1830 compared to placebo in adult participants with warm autoimmune hemolytic anemia (WAIHA). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Warm Autoimmune Hemolytic Anemia
- What is being tested?
- ALXN1830
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2021to 30 Apr 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Primary or secondary WAIHA, diagnosed at least 6 weeks prior to Screening
- Failed or not tolerated at least 1 prior WAIHA treatment regimen, for example, corticosteroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, danazol, or vincristine
- Hemoglobin < 10 g/dL and ≥ 6 g/dL
- Positive direct antiglobulin test (Coombs) (IgG positive, complement C3 [C3] positive or negative)
- Evidence of active hemolysis including any of the following: a) Lactate dehydrogenase (LDH) > upper limit of normal (ULN), b) Haptoglobin < lower limit of normal (LLN), c) Indirect bilirubin > ULN
- Total IgG > 500 mg/dL
Who cannot join
- Human immunodeficiency virus (HIV) infection (positive HIV-1 or HIV-2 antibody test)
- Positive hepatitis B surface antigen (HBsAg) or hepatitis C antibody test
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Study of ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia
The main objective of the study is to evaluate the safety and efficacy of ALXN1830 compared to placebo in adult participants with warm autoimmune hemolytic anemia (WAIHA).
Read the detailed description
This study will consist of a 4-week Screening period, 13-week Primary Treatment period, and optional Extended Treatment period (up to 2 years).
- Conditions
- Warm Autoimmune Hemolytic Anemia
- Being tested
- ALXN1830, Placebo
- Keywords
- Warm autoimmune hemolytic anemia, WAIHA, Immunoglobulin G (IgG)-mediated autoimmune disorder, Pathogenesis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN1830Biological
Also called SYNT001
Administered via intravenous (IV) infusion
PlaceboOther
Matching placebo (sterile liquid diluent) administered via IV infusion
| Study group | Role | What they receive |
|---|---|---|
| ALXN1830 Dosing Regimen 1 | Experimental | Not described Receives: Biological: ALXN1830 |
| ALXN1830 Dosing Regimen 2 | Experimental | Not described Receives: Biological: ALXN1830 |
| ALXN1830 Dosing Regimen 3 | Experimental | Not described Receives: Biological: ALXN1830 |
| Placebo | Active comparator | Not described Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage Of Participants With ≥ 2 Grams (g)/Deciliter (dL) Increase In Hemoglobin (Hgb) From Baseline
Measured: Baseline (Day 1) through Day 92
Secondary outcomes (5)
Total Number Of Units Of Packed Red Blood Cells (pRBCs) Transfused
Measured: Day 15 through Day 92
Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via EuroQoL 5 Dimension 5 Level (EQ-5D-5L) Questionnaire
Measured: Baseline, Day 92
Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via Functional Assessment Of Cancer Therapy Anemia (FACT-AN) Questionnaire
Measured: Baseline, Day 92
Number Of Weekly Hgb Measurements With Change From Baseline ≥ 2 g/dL
Measured: Day 1 through Day 92
Number Of Participants Needing New WAIHA Rescue Medication Or Increase In Dose Of WAIHA Medication
Measured: Day 15 through Day 92
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2020
- Study startJul 2021 (estimated)
- Primary completion30 Oct 2022 (estimated)
- Study completion30 Apr 2023 (estimated)
Why it stopped: Study is withdrawn due to the global impact of the COVID-19 pandemic.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Warm Autoimmune Hemolytic AnemiaStudies 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- Participants randomized in 1:1:1:1 ratio to 1 of 4 study arms.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 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
- NCT04256148
- Sponsor's protocol ID
- ALXN1830-WAI-201
- Status last verified
- Jul 2020
- First submitted
- 27 Jan 2020
- Last updated
- 13 Jul 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Study of ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia
- ClinicalTrials.gov ID
- NCT04256148
- Acronym / sponsor ID
- ALXN1830-WAI-201
- Conditions
- Warm Autoimmune Hemolytic Anemia
- Keywords
- Warm autoimmune hemolytic anemia, WAIHA, Immunoglobulin G (IgG)-mediated autoimmune disorder, Pathogenesis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study is withdrawn due to the global impact of the COVID-19 pandemic.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Jul 2021 (estimated)
- Primary completion
- 30 Oct 2022 (estimated)
- Study completion
- 30 Apr 2023 (estimated)
- First posted
- 5 Feb 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main objective of the study is to evaluate the safety and efficacy of ALXN1830 compared to placebo in adult participants with warm autoimmune hemolytic anemia (WAIHA).
Detailed description
This study will consist of a 4-week Screening period, 13-week Primary Treatment period, and optional Extended Treatment period (up to 2 years).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
- Model description
- Participants randomized in 1:1:1:1 ratio to 1 of 4 study arms.
| Arm | Type | Receives |
|---|---|---|
| ALXN1830 Dosing Regimen 1 | Experimental | Biological: ALXN1830 |
| ALXN1830 Dosing Regimen 2 | Experimental | Biological: ALXN1830 |
| ALXN1830 Dosing Regimen 3 | Experimental | Biological: ALXN1830 |
| Placebo | Active comparator | Other: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Primary or secondary WAIHA, diagnosed at least 6 weeks prior to Screening
- Failed or not tolerated at least 1 prior WAIHA treatment regimen, for example, corticosteroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, danazol, or vincristine
- Hemoglobin < 10 g/dL and ≥ 6 g/dL
- Positive direct antiglobulin test (Coombs) (IgG positive, complement C3 [C3] positive or negative)
- Evidence of active hemolysis including any of the following: a) Lactate dehydrogenase (LDH) > upper limit of normal (ULN), b) Haptoglobin < lower limit of normal (LLN), c) Indirect bilirubin > ULN
Key exclusion criteria (2 listed)
- Human immunodeficiency virus (HIV) infection (positive HIV-1 or HIV-2 antibody test)
- Positive hepatitis B surface antigen (HBsAg) or hepatitis C antibody test
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 |
|---|---|---|
| ALXN1830Also: SYNT001Administered via intravenous (IV) infusion | Biological | ALXN1830 Dosing Regimen 1, ALXN1830 Dosing Regimen 2, ALXN1830 Dosing Regimen 3 |
| PlaceboMatching placebo (sterile liquid diluent) administered via IV infusion | Other | Placebo |
Primary outcome
Percentage Of Participants With ≥ 2 Grams (g)/Deciliter (dL) Increase In Hemoglobin (Hgb) From Baseline
Time frame: Baseline (Day 1) through Day 92
5 secondary outcomes
Total Number Of Units Of Packed Red Blood Cells (pRBCs) Transfused
Time frame: Day 15 through Day 92
Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via EuroQoL 5 Dimension 5 Level (EQ-5D-5L) Questionnaire
Time frame: Baseline, Day 92
Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via Functional Assessment Of Cancer Therapy Anemia (FACT-AN) Questionnaire
Time frame: Baseline, Day 92
Number Of Weekly Hgb Measurements With Change From Baseline ≥ 2 g/dL
Time frame: Day 1 through Day 92
Number Of Participants Needing New WAIHA Rescue Medication Or Increase In Dose Of WAIHA Medication
Time frame: Day 15 through Day 92
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jul 2020.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ALXN1830 in people with Warm Autoimmune Hemolytic Anemia, run by Alexion Pharmaceuticals, Inc. at 1 site in United States.
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 6 inclusion and 2 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: withdrawn, as of the sponsor's last update on 13 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Percentage Of Participants With ≥ 2 Grams (g)/Deciliter (dL) Increase In Hemoglobin (Hgb) From Baseline (time frame: Baseline (Day 1) through Day 92). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 13 Jul 2020; AzTrials last refreshed its copy on 7 Nov 2026.