ALXN2050 for Paroxysmal Nocturnal Hemoglobinuria

No longer recruiting at 28 trial locations

Overseen By Alexion Pharmaceuticals Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Alexion

Must be taking: Eculizumab, Danicopan

Disqualifiers: Organ transplant, Aplastic anemia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 12

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition where red blood cells break down too easily. The trial tests an oral drug called ALXN2050, which may benefit individuals with PNH who are new to treatment, not fully benefiting from eculizumab, or previously treated with danicopan. Potential participants include those diagnosed with PNH who are either new to treatment or have specific ongoing treatment experiences but still experience anemia (low red blood cell count) and related symptoms. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are currently on eculizumab, you must have been on a stable dose for at least 8 weeks. If you are on danicopan, you must have received it during a specific previous study.

Is there any evidence suggesting that ALXN2050 is likely to be safe for humans?

Research has shown that ALXN2050, also known as vemircopan, is generally safe for people with paroxysmal nocturnal hemoglobinuria (PNH). Early results from a study indicated that this treatment did not cause any serious safety issues. Participants experienced mild side effects, but these were not severe enough to discontinue the treatment. ALXN2050 remains under study, so researchers will continue to closely monitor its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Paroxysmal Nocturnal Hemoglobinuria (PNH), which often involve complement component 5 (C5) inhibitors like eculizumab, ALXN2050 offers a fresh approach. Researchers are excited about ALXN2050 because it is taken orally, providing a more convenient option compared to intravenous therapies. Additionally, ALXN2050 works by targeting a different part of the complement system, which could potentially address anemia and high reticulocyte counts that persist in some patients despite existing treatments. This unique mechanism and delivery method are what make ALXN2050 a promising new option for those living with PNH.

What evidence suggests that ALXN2050 might be an effective treatment for PNH?

Research has shown that ALXN2050 may help treat Paroxysmal Nocturnal Hemoglobinuria (PNH). In earlier studies, patients experienced an 80% reduction in lactate dehydrogenase (LDH) levels, indicating less red blood cell breakdown. This suggests that ALXN2050 might reduce red blood cell destruction in PNH patients. In this trial, researchers are testing ALXN2050 as an open-label monotherapy, aiming to improve symptoms such as anemia, which occurs when there aren't enough healthy red blood cells. This is particularly important for patients who do not receive sufficient benefit from other treatments like eculizumab. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who either haven't been treated, still have anemia after eculizumab treatment, or are on danicopan. They must have certain levels of blood cells and enzymes indicating PNH. People can't join if they've had organ transplants, other bone marrow issues not related to PNH, or severe kidney problems.

Inclusion Criteria

You have a specific type of blood cell clone that is larger than 10%.

Your LDH levels in the blood are higher than 1.5 times the normal limit.

Your blood has low levels of hemoglobin (Hgb <10.5 g/dL).

Exclusion Criteria

I have a bleeding disorder or condition causing anemia not related to PNH.

I have had a major organ or bone marrow transplant.

I have a bone marrow condition needing a stem cell transplant or have been on immunosuppressants for less than 6 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ALXN2050 monotherapy for 12 weeks to assess efficacy and safety

12 weeks

Periodic visits through Week 12

Long-term Extension

Participants continue on ALXN2050 treatment beyond 12 weeks

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ALXN2050

Trial Overview The study tests ALXN2050 as a solo treatment for PNH. It checks how well it works and its safety in new patients, those previously on eculizumab with ongoing anemia, and those taking danicopan alone. Participants will be checked regularly up to Week 12 then continue into a long-term part of the study.

How Is the Trial Designed?

Group I: Open-label ALXN2050 Monotherapy Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Alexion

Lead Sponsor

Trials

247

Recruited

38,600+

Marc Dunoyer

Alexion

Chief Executive Officer since 2021

PhD in Molecular Biology, University of Brussels

Christophe Hotermans

Alexion

Chief Medical Officer since 2021

MD, University of Leuven

Alexion Pharmaceuticals

Lead Sponsor

Trials

231

Recruited

36,700+

Marc Dunoyer

Alexion Pharmaceuticals

Chief Executive Officer since 2021

B.A. in Psychology from the University of New Hampshire

Christophe Hotermans

Alexion Pharmaceuticals

Chief Medical Officer since 2023

MD

Alexion Pharmaceuticals, Inc.

Lead Sponsor

Trials

267

Recruited

141,000+

Dr. Alberto R. Martinez

Alexion Pharmaceuticals, Inc.

Chief Medical Officer since 2010

MD from University of Sao Paulo

Marc Dunoyer

Alexion Pharmaceuticals, Inc.

Chief Executive Officer since 2021

PhD in Molecular Biology

Citations

1. sciencedirect.com sciencedirect.com/science/article/pii/S0268960X23001285

Review of the patient experience and treatment landscape A 2009 study that investigated the variable hematologic benefit of eculizumab found that Hb normalization was observed in only approximately one-third (15/41) ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04170023

Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH ... Blood will be collected to assess the efficacy endpoints, such as, change in hemoglobin (Hgb), lactate dehydrogenase (LDH), and other measures of hemolysis.

3. practicingclinicians.com practicingclinicians.com/activity/ecase/downloadsectionfile/1D7785B2-5376-43CB-8132-73A152E1085A

Phase II Trial of Vemircopan (ALXN2050) Monotherapy for ... Phase II Trial of Vemircopan (ALXN2050) Monotherapy for Paroxysmal Nocturnal Hemoglobinuria. Supported by educational grants from AbbVie, Amgen, AstraZeneca ...

4. hra.nhs.uk hra.nhs.uk/planning-and-improving-research/application-summaries/research-summaries/study-of-ach-0145228-in-pnh-as-monotherapy-or-with-a-c5-inhibitor/

Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH ... This study will determine if ACH-0145228, is safe and effective in increasing red blood cells (RBCs). The only curative treatment for PNH is hematopoietic stem ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10710797/

Paroxysmal Nocturnal Hemoglobinuria: Current Management ... A phase-2 trial of nine patients, five of whom carrying the Arg885His C5 variant, demonstrated approximately 80% reduction in LD levels, ...

6. rarediseaseadvisor.com rarediseaseadvisor.com/reports/vemircopan-monotherapy-appears-safe-effective-patients-pnh/

Vemircopan Monotherapy Appears Safe and Effective in ... Vemircopan (ALXN2050) monotherapy in paroxysmal nocturnal hemoglobinuria: interim data from a phase 2 open-label proof-of-concept study.

7. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/full/10.1002/ajh.27502

American Journal of Hematology | Blood Research Journal Vemircopan (ALXN2050) monotherapy in paroxysmal nocturnal hemoglobinuria: interim data from a phase 2 open-label proof-of-concept study.