What side effects are associated with this type of intervention?

Common treatments for Thrombotic Microangiopathy (TMA), such as Pegcetacoplan (a C3 complement inhibitor), often have side effects including infections due to immunosuppression, gastrointestinal symptoms (nausea, diarrhea), and infusion-related reactions (fever, chills, rash). Other potential side effects may include fatigue, headache, and increased risk of thrombotic events. It is important for patients to discuss these potential side effects with their healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Thrombotic Microangiopathy (TMA), particularly those targeting the complement system. Eculizumab, a C5 complement inhibitor, is one of the primary treatments for atypical Hemolytic Uremic Syndrome (aHUS), a type of TMA. Ravulizumab, another C5 inhibitor, has also been approved for aHUS. These drugs work by inhibiting the complement pathway, similar to Pegcetacoplan, which is a C3 complement inhibitor currently being studied for its efficacy in TMA treatment.

What other types of research exist?

Promising alternatives to Pegcetacoplan for Thrombotic Microangiopathy patients include Ravulizumab, a C5 complement inhibitor, and Narsoplimab, a MASP-2 inhibitor. Both have shown efficacy in treating TMA and offer novel mechanisms of action targeting the complement system.

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Complement Inhibitor

Pegcetacoplan for TA-TMA After Stem Cell Transplant

Phase 2

Waitlist Available

Research Sponsored by Swedish Orphan Biovitrum

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of TA-TMA established, as per laboratory markers indicating TMA

Have a diagnosis of TA-TMA that persists despite initial management of any triggering condition

Must not have

Known or suspected hereditary fructose intolerance

Active GI bleeding (hematemesis or hematochezia) at baseline

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from treatment start to end of study, an average of 6 months

Awards & highlights

Summary

This trial is testing pegcetacoplan, a medication that targets the immune system, in patients with TA-TMA after a stem cell transplant. The goal is to see if it can help these patients by blocking harmful immune responses.

Who is the study for?

Adults over 18 with TA-TMA after a stem cell transplant can join. They must have specific lab markers for TMA, agree to use contraception, and not be pregnant or fathering children. Excluded are those with certain blood disorders, active infections, known genetic conditions affecting the blood, uncontrolled bleeding or weight outside 30-100 kg range.

What is being tested?

The trial is testing Pegcetacoplan's effects on patients with TA-TMA post-stem cell transplant. It looks at how the body processes it (PK), its impact on disease (PD), effectiveness in treating symptoms of TMA and overall safety.

What are the potential side effects?

Possible side effects of Pegcetacoplan include allergic reactions to ingredients, potential increased risk of infections due to immune system changes, and other unspecified impacts that will be monitored throughout the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My tests show I have thrombotic microangiopathy.

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My TA-TMA condition persists despite initial treatments.

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I am not pregnant and agree to use birth control as required by the study.

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I have received a stem cell transplant from a donor.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have or might have hereditary fructose intolerance.

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I am not currently experiencing any active bleeding from my stomach or intestines.

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I have a known ADAMTS13 deficiency.

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I have been treated with a drug for my immune system.

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I have been diagnosed with veno-occlusive disease.

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My body weight is either below 30 kg or above 100 kg.

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I have been diagnosed with a condition related to Shiga toxin affecting my kidneys.

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I have been diagnosed with bone marrow failure or a failed bone marrow transplant.

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I have been diagnosed with a blood clotting disorder.

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I have chronic inactive hepatitis B with a high viral load.

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I do not have an uncontrolled infection or sepsis.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from treatment start to end of study, an average of 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from treatment start to end of study, an average of 6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and from treatment start to end of study, an average of 6 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Pegcetacoplan PK parameter AUC0-tau

Pegcetacoplan PK parameter Cmax

Pegcetacoplan PK parameter Ctrough

+1 more

Secondary study objectives

Absolute levels, change from baseline, and percent change in sC5b-9

Duration of TMA response.

Duration of clinical response.

+8 more

Other study objectives

Number of participants with antibodies to polyethylene glycol (PEG) and pegcetacoplan throughout treatment and follow-up periods.

Number of participants with clinically significant changes in abnormal electrocardiogram findings.

Number of participants with clinically significant changes in vital signs.

+1 more

Side effects data

From 2020 Phase 3 trial • 80 Patients • NCT03500549

41%

Injection site erythema

15%

Injection site pruritus

13%

Headache

13%

Injection site swelling

13%

Diarrhoea

10%

Injection site reaction

Nausea

Pyrexia

Injection site pain

Fatigue

Injection site induration

Dyspnoea

Nasopharyngitis

Contusion

Dizziness

Chromaturia

Vaccination site pain

Pain in extremity

Back pain

Myalgia

Vaccination complication

Anxiety

Thrombocytopenia

Cough

Abdominal pain

Abdominal discomfort

Arthralgia

Injection site bruising

Sepsis

Haemolysis

Sinusitis

Vomiting

Oral herpes

Anaemia

Asthenia

Palpitations

Abdominal distension

Constipation

Urinary tract infection

100%

80%

60%

40%

20%

Study treatment Arm

Run-in Period: Pegcetacoplan + Eculizumab

Open-label Period: Pegcetacoplan

RCP: Eculizumab

RCP: Pegcetacoplan

Trial Design

1Treatment groups

Experimental Treatment

Group I: PegcetacoplanExperimental Treatment1 Intervention

sterile solution in stoppered glass vial given as 1080 mg infusion 3 times weekly for 12 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pegcetacoplan

2015

Completed Phase 3

~380

0 / 15Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Thrombotic Microangiopathy (TMA) is often treated with complement inhibitors, such as Pegcetacoplan, which target the complement system, a part of the immune system that contributes to inflammation and cell damage. Pegcetacoplan specifically inhibits C3, a central component of the complement cascade, preventing the formation of downstream inflammatory mediators and membrane attack complexes that can damage blood vessels and organs. This mechanism is crucial for TMA patients as it helps to reduce the underlying inflammation and thrombosis, thereby improving clinical outcomes and reducing organ damage.

Eculizumab in the treatment of membranoproliferative glomerulonephritis.