Efgartigimod for Thrombotic Thrombocytopenic Purpura

Overseen By Diondra Howard

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Minnesota

Disqualifiers: Pregnancy, Lactation, cTTP, 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: 13

Duration: 60 days

15 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called efgartigimod for individuals with immune-mediated thrombotic thrombocytopenic purpura (iTTP), a rare disorder causing sudden episodes of low blood platelets and organ damage. The study aims to determine if efgartigimod can safely and temporarily increase a protein called ADAMTS13, which aids in blood clotting, to prevent relapses. Ideal candidates have previously experienced iTTP, are currently in remission, and have specific levels of ADAMTS13 activity. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but if you are on oral immunosuppressants, you must not change the dose for at least 60 days before joining the study.

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

Research has shown that efgartigimod is generally safe for people. In studies involving individuals with primary immune thrombocytopenia (a condition where the immune system attacks blood cells called platelets), efgartigimod was well tolerated. Participants did not face a higher risk of infections, a common concern with treatments affecting the immune system.

Additional studies on various autoimmune diseases involving IgG antibodies found that patients tolerated efgartigimod well, regardless of the dose. Side effects were manageable and not severe. This consistent safety record suggests that efgartigimod could be a reliable treatment option for immune-mediated thrombotic thrombocytopenic purpura (iTTP), the condition under study in this trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard care for thrombotic thrombocytopenic purpura (TTP), which often involves plasma exchange and immunosuppressive therapies, efgartigimod offers a novel approach by targeting the immune system more precisely. Efgartigimod works by modulating the activity of the neonatal Fc receptor (FcRn), which reduces the levels of pathogenic autoantibodies in the bloodstream. This mechanism differs from traditional treatments, aiming to provide a more targeted and potentially quicker response in patients. Researchers are excited about efgartigimod because it could offer a more effective and focused treatment with fewer side effects compared to existing therapies.

What evidence suggests that efgartigimod might be an effective treatment for iTTP?

Research has shown that efgartigimod can significantly lower certain harmful antibodies in the blood. In studies with patients who have chronic immune thrombocytopenia—a condition where the immune system mistakenly attacks platelets, causing low platelet counts—about 22% of those treated with efgartigimod achieved the desired results, compared to only 5% in the untreated group. Efgartigimod reduces the levels of specific antibodies that attack healthy cells. In this trial, participants with immune-mediated thrombotic thrombocytopenic purpura (iTTP) will receive efgartigimod, which is expected to increase the activity of ADAMTS13, an important enzyme for blood clotting. This could potentially prevent relapses and complications like strokes, suggesting that efgartigimod might effectively manage iTTP by temporarily reducing harmful antibodies. ^{2 5 6 7 8}

Who Is on the Research Team?

Marshall Mazepa, MD

Principal Investigator

University of Minnesota

Are You a Good Fit for This Trial?

This trial is for individuals who have had immune-mediated Thrombotic thrombocytopenic purpura (iTTP) and are currently in remission with specific levels of ADAMTS13 activity. They must be at least 6 months past their last rituximab or similar treatment, not have changed oral immunosuppressant doses recently, and agree to use effective birth control.

Inclusion Criteria

I have signed the consent form for the trial.

I am not pregnant and agree to use effective birth control during the study.

My ADAMTS13 activity levels were between 30% and 70% on two tests at least a week apart.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive efgartigimod therapy to increase ADAMTS13 activity

60 days

Regular visits for treatment administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

Follow-up visits to assess ADAMTS13 activity and antibody titers

What Are the Treatments Tested in This Trial?

Interventions

• Efgartigimod

Trial Overview The study tests efgartigimod's ability to increase ADAMTS13 activity temporarily in iTTP patients. It aims to show that this drug can safely prevent relapses by reducing harmful antibodies. Participants will receive efgartigimod therapy and then be monitored for changes in ADAMTS13 levels after stopping the treatment.

How Is the Trial Designed?

Group I: iTTP patients Experimental Treatment 1 Intervention

participants with a history of iTTP in clinical remission but with ADAMTS13 deficiency (\>30% but \< 70% activity)

Who Is Running the Clinical Trial?

University of Minnesota

Lead Sponsor

Trials

1,459

Recruited

1,623,000+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37778358/

Efficacy and safety of the neonatal Fc receptor inhibitor ... 22% (17/78) of patients with chronic immune thrombocytopenia receiving efgartigimod reached the primary endpoint compared with 5% (2/40) of ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06831058?term=Efgartigimod&viewType=Table&rank=7

NCT06831058 | A Pilot Study of Efgartigimod for Immune ... This would demonstrate the potential efficacy for efgartigimod as a maintenance therapy to safely prevent relapse of iTTP to be further explored in a larger ...

3. argenx.com argenx.com/news/2022/argenx-announces-positive-phase-3-data-advance-trial-vyvgartr-efgartigimod-alfa-fcab-adults

argenx Announces Positive Phase 3 Data from ADVANCE ... ADVANCE met its primary endpoint demonstrating that a higher proportion of chronic ITP patients receiving VYVGART achieved a sustained platelet count response ...

4. ashpublications.org ashpublications.org/blood/article/140/Supplement%201/6/490458/Efficacy-and-Safety-of-Intravenous-Efgartigimod-in

Efficacy and Safety of Intravenous Efgartigimod in Adults with ... This is the first report of topline results from a phase 3 trial evaluating the efficacy and safety of efgartigimod in primary ITP.

5. thelancet.com thelancet.com/journals/lancet/article/PIIS0140-6736(24)01263-7/fulltext

Efgartigimod for primary immune thrombocytopenia In clinical trials, efgartigimod has resulted in a 60–80% reduction of IgG concentrations from baseline, ... 1. Broome, CM ∙ McDonald, V ∙ ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06544499

NCT06544499 | A Study to Assess the Efficacy and Safety ... The main purpose of this study is to look at the effect (efficacy) and safety of efgartigimod IV in participants with primary immune thrombocytopenia (ITP).

7. ashpublications.org ashpublications.org/ashclinicalnews/news/7231/Efgartigimod-Shows-Long-Term-Safety-Efficacy-in

Efgartigimod Shows Long-Term Safety, Efficacy in Long ... Long-term efgartigimod has an acceptable side effect profile and is not associated with increased exposure-adjusted risk for infection among patients with long ...

8. tandfonline.com tandfonline.com/doi/full/10.1080/1744666X.2025.2497840

Safety profile of efgartigimod from global clinical trials ... Across clinical trials in IgG-mediated autoimmune diseases, efgartigimod was well tolerated with similar safety profiles regardless of dosing regimen.