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Warm Autoimmune Hemolytic AnemiaPhase 2NCT04956276

Subcutaneous ALXN1830 in Adult Participants 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.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

This is a Phase 2, multiple ascending, dose-finding, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, health-related quality of life, tolerability, pharmacokinetic, pharmacodynamic, and immunogenicity, of up to 3 dose regimens of ALXN1830 administered subcutaneous(ly) (SC) in the… 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
1 Jan 2022to 31 Jul 2024
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

  • Diagnosed with primary or secondary WAIHA at least 6 weeks prior to Screening.
  • Failed or have not tolerated at least one prior WAIHA treatment regimen, for example, corticosteroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, danazol, or vincristine.
  • Hemoglobin < 10 g/dL and ≥ 6 g/dL at Screening.
  • Positive direct antiglobulin test (Coombs) (IgG positive who are positive or negative for the presence of complement C3) at Screening.
  • Evidence of active hemolysis including any one of the below:
  • LDH > upper limit of normal (ULN) or
Show all 11Show fewer
  • Haptoglobin < lower limit of normal or
  • Indirect bilirubin > ULN
  • Total IgG > 500 mg/dL at Screening
  • Platelet count ≥ 75 x 10^9/liter (L)
  • Absolute neutrophil count greater than 1.0 x 10^9/L

Who cannot join

  • Participants with Evan's syndrome.
  • Human immunodeficiency virus (HIV) infection (positive HIV 1 or HIV 2 antibody test).
  • Positive hepatitis B surface antigen or hepatitis C antibody test.
  • Inability to travel to the clinic for specified visits during the Primary Treatment Period or fulfill the logistical requirements of study intervention administration.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Clinical Study Site

    Riverside, California, 90602-3171

Other trials in United States

About this study

Official title: A Phase 2, Multiple Ascending Dose, Randomized, Double-Blind, Placebo-Controlled Study of ALXN1830 Administered Subcutaneously in Patients With Warm Autoimmune Hemolytic Anemia (WAIHA)

This is a Phase 2, multiple ascending, dose-finding, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, health-related quality of life, tolerability, pharmacokinetic, pharmacodynamic, and immunogenicity, of up to 3 dose regimens of ALXN1830 administered subcutaneous(ly) (SC) in the treatment of WAIHA.

This study will include 2 randomized, double-blind, placebo-controlled cohorts (Cohorts 1 and 2) to evaluate an 8-week treatment regimen, and an optional third open-label cohort (Cohort 3) to evaluate an alternative 12-week dosing regimen. Participants may continue participation in this study at the participant's and investigator's discretion in an open-label extension (OLE) period, consisting of monthly visits to observe participants for relapse, which will require going back on active treatment.

Being tested
ALXN1830, Placebo
Keywords
Warm autoimmune hemolytic anemia, WAIHA, Immunoglobulin G-mediated autoimmune disorder, Pathogenesis, Anti-neonatal Fc receptor, ALXN1830

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ALXN1830Drug

    Also called SYNT001 (formerly)

    Administered as an SC infusion.

  • PlaceboDrug

    Administered as an SC infusion.

Study groups in NCT04956276
Study groupRoleWhat they receive
Cohort 1: ALXN1830/PlaceboExperimentalParticipants will be randomized 3:1 to receive ALXN1830 or placebo. Treatment will be received for 8 weeks followed by a follow-up period (no treatment) for 8 weeks. Once complete, participants may continue participation in the study at the participant's and investigator's discretion during the OLE period for up to 2 years inclusive of primary treatment period.
Receives: Drug: ALXN1830; Drug: Placebo
Cohort 2: ALXN1830/PlaceboExperimentalParticipants will be randomized 3:1 to receive ALXN1830 or placebo. Treatment will be received for 8 weeks followed by a follow-up period (no treatment) for 8 weeks. Once complete, participants may continue participation in the study at the participant's and investigator's discretion during the OLE period for up to 2 years inclusive of primary treatment period.
Receives: Drug: ALXN1830; Drug: Placebo
Cohort 3: ALXN1830ExperimentalIf initiated, participants will receive ALXN1830. Treatment will be received for 12 weeks followed by a follow-up period (no treatment) for 8 weeks. Once complete, participants may continue participation in the study at the participant's and investigator's discretion during the OLE period for up to 2 years inclusive of primary treatment period.
Receives: Drug: ALXN1830

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Proportion Of Participants Achieving A ≥ 2 Grams/Deciliter (g/dL) Increase In Hemoglobin (Hgb) From Baseline To The End Of Primary Treatment

    Participants will have to achieve this increase without requiring any increase in the dose of an existing WAIHA medication after Day 1 (baseline) and without packed red blood cells (pRBC) transfusions after Day 14.

    Measured: Baseline through Week 12

Secondary outcomes (24)

  1. Total Number Of Units Of pRBCs Transfused

    Measured: Baseline through Week 12

  2. Number Of Hgb Measurements ≥ 2 g/dL From Baseline To The End Of Primary Treatment

    Measured: Baseline, Week 12

  3. Time To Hgb Increase By ≥ 2 g/dL From Baseline

    Measured: Baseline through Week 12

  4. Proportion Of Participants Who Require New WAIHA Rescue Medication Or Any Increase In The Dose Of An Existing WAIHA Medication Or pRBC Transfusions For The Treatment Of Anemia

    Measured: Day 15 through Week 12

  5. Proportion Of Participants Achieving A ≥ 2 g/dL Increase In Hgb From Baseline Through Week 4

    Participants need to achieve this increase without requiring any increase in the dose of an existing WAIHA medication after Day 1 and without pRBC transfusions after Day 14.

    Measured: Baseline through Week 4

  6. Change From Baseline To The End Of Primary Treatment In Serum Lactate Dehydrogenase (LDH) Levels

    Measured: Baseline, Week 12

  7. Change From Baseline To The End Of Primary Treatment In Absolute Reticulocyte Count

    Measured: Baseline, Week 12

  8. Change From Baseline To The End Of Primary Treatment In Serum Indirect Bilirubin

    Measured: Baseline, Week 12

  9. Change From Baseline To The End Of Primary Treatment In Serum Haptoglobin

    Measured: Baseline, Week 12

  10. Total Corticosteroid Usage From Baseline To The End Of Primary Treatment

    Measured: Baseline, Week 12

  11. Proportion Of Participants Who Require Any Increase In Corticosteroid Dose From Baseline To The End Of Follow Up After Primary Treatment

    Measured: Baseline through Week 20

  12. Change In Corticosteroid Dose From The End Of Primary Treatment To The End Of Follow Up

    Measured: Week 12, Week 20

  13. Number Of Days To Beginning Of Corticosteroid Taper During Follow Up After Primary Treatment

    Taper is defined as the first day that a lower dose of corticosteroids is prescribed/taken.

    Measured: Baseline through Week 20

  14. Number Of Days To Corticosteroid Maintenance Dose During Follow Up After Primary Treatment

    Maintenance dose will be defined as < 10 milligrams (mg)/day of prednisone or equivalent.

    Measured: Baseline through Week 20

  15. Number Of Days To Reach Corticosteroid Discontinuation From The End Of Primary Treatment To The End Of Follow Up After Primary Treatment

    Measured: Week 12 through Week 20

  16. Incidence And Titers Of Anti-drug Antibodies Against ALXN1830 Over Time

    Measured: Up to 2 years

  17. Incidence And Titers Of Neutralizing Antibodies Against ALXN1830 Over Time

    Measured: Up to 2 years

  18. Serum Trough Concentrations Of ALXN1830 Over Time

    Measured: Up to 2 years

  19. Change In Serum Total Immunoglobulin G (IgG) Levels By Dose Group And Time Point

    Measured: Up to 2 years

  20. Change From Baseline Of IgG Subtypes (IgG1 4) By Dose Group And Time Point

    Measured: Up to 2 years

  21. Change From Baseline Of IgA By Dose Group And Time Point

    Measured: Up to 2 years

  22. Change From Baseline Of IgM By Dose Group And Time Point

    Measured: Up to 2 years

  23. Change From Baseline Of Albumin By Dose Group And Time Point

    Measured: Up to 2 years

  24. Change From Baseline Of Circulating Immune Complexes By Dose Group And Time Point

    Measured: Up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Jul 2021
  2. Study start1 Jan 2022 (estimated)
  3. Primary completion31 Jul 2022 (estimated)
  4. Study completion31 Jul 2024 (estimated)

Why it stopped: Sponsor decision to terminate program

Resources

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
Sequential
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04956276
Sponsor's protocol ID
ALXN1830-WAI-202
Status last verified
Feb 2022
First submitted
30 Jun 2021
Last updated
11 Feb 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes

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 11 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: withdrawn, as of the sponsor's last update on 11 Feb 2022. 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?

Proportion Of Participants Achieving A ≥ 2 Grams/Deciliter (g/dL) Increase In Hemoglobin (Hgb) From Baseline To The End Of Primary Treatment (time frame: Baseline through 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 NCT04956276.

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 11 Feb 2022.

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