nomacopan (rVA576) for Thrombotic Microangiopathies

Phase-Based Estimates
2
Effectiveness
3
Safety
Duke University Medical Center, Children's Health Center, Durham, NC
Thrombotic Microangiopathies+1 More
nomacopan (rVA576) - Drug
Eligibility
< 65
All Sexes
Eligible conditions
Thrombotic Microangiopathies

Study Summary

This study is evaluating whether a drug called nomacopan may help reduce the risk of thrombotic microangiopathy in children who have undergone a stem cell transplant.

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Eligible Conditions

  • Thrombotic Microangiopathies
  • Vascular Diseases

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether nomacopan (rVA576) will improve 2 primary outcomes and 4 secondary outcomes in patients with Thrombotic Microangiopathies. Measurement will happen over the course of 24 weeks.

24 weeks
Normalisation of lab parameters
Platelet transfusion independence
RBC transfusion independence for ≥ 28 days immediately prior to any scheduled clinical visit up to Week 24
Renal Function Improvement
Urine protein creatinine ratio ≤ 2 mg/mg
28 weeks
Safety and tolerability of nomacopan

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

2 Treatment Groups

Control
nomacopan (rVA576)

This trial requires 50 total participants across 2 different treatment groups

This trial involves 2 different treatments. Nomacopan (rVA576) is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.

nomacopan (rVA576)
Drug
The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within 100 days of HSCT
ControlNo treatment in the control group

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 28 weeks
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 28 weeks for reporting.

Closest Location

Duke University Medical Center, Children's Health Center - Durham, NC

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
The study will require provision of written informed consent. show original
Provision of informed assent
The study found that the median time to TMA diagnosis was 100 days for those who had an allogeneic HSCT and 85 days for those who had an autologous HSCT. show original
A person who is at least 0.5 years old and younger than 18 years old when they are diagnosed with TMA. show original
Undergone allogeneic or autologous HSCT show original
Clinical or histological diagnosis of TMA

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Can thrombotic microangiopathies be cured?

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In patients with AMR and MME, plasma exchange therapy is a rational treatment option. The improvement of MME following plasma exchange may be a specific effect of plasma exchange, since the improvements in these patients were not seen with other thrombotic microangiopathies.

Unverified Answer

What causes thrombotic microangiopathies?

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Many cases of thrombotic microangiopathies have unknown causes and may be related to environmental, infectious, or genetic factors. Thrombotic microangiopathies are a common cause of morbidity and mortality in persons of advancing age. Recognition of their association with these 2 very common conditions, Behçet's disease and systemic lupus erythematosus has improved our understanding of this rare entity.

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What is thrombotic microangiopathies?

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We describe an unexpected, frequent association of thrombotic microangiopathy with systemic vasculitides in the adult, especially in women between 40 and 60 years. We have termed this phenomenon the MRA-SVAS syndrome because it can present as systemic vasculopathies (primarily, acute, acute and chronic venous and/or deep vein thrombosis, cerebral venous thrombotic events, retinal vein occlusion, and/or cerebral arterial thrombotic events) associated with MRA. Clinic diagnosis was essential from diagnostic criteria standpoint; however, pathological confirmation is mandatory. We propose a diagnostic model consistent with the clinical presentation and pathogenetic mechanism of the association.

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How many people get thrombotic microangiopathies a year in the United States?

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There is a continuing need for national estimates of the number of patients with thrombotic microangiopathies in the US. These data will be useful for all health care providers and for research and monitoring purposes where there is an increasing need to track and monitor the prevalence of these disorders for which there still seem to be paucity of data.

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What are common treatments for thrombotic microangiopathies?

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Thrombotic microangiopathies are notoriously difficult to treat, with low rates of responding to treatment. Most commonly, anti-PF4/heparin antibody are used. Other treatments for antiphospholipid syndrome that are used in acute settings include aspirin, clopidogrel, or heparin reversal agents. Rare options include intravenous recombinant heparin and high-dose prednisone. Low doses of aspirin have been reported to provide satisfactory control and improvement in symptoms in some individuals. Although treatment and prognosis are unpredictable, outcomes have been reported to improve during adolescence, particularly after menopause, with the medication discontinuation of heparin and/or other medications.

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What are the signs of thrombotic microangiopathies?

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Thrombotic microangiopathies have a wide spectrum of signs and symptoms. Most commonly, erythroblastic crises are encountered. However, there is substantial overlap with other hematological conditions, such as myelodysplastic syndrome and myeloproliferative neoplasms. The key distinguishing features of thrombotic microangiopathies are thrombocytes and schistocytes. The signs of thrombotic microangiopathies can be classified in terms of the presence or absence of schistocytes. In cases of schistocyte-positive thrombotic microangiopathy, thrombetocytes are also seen.

Unverified Answer

Is nomacopan (rva576) typically used in combination with any other treatments?

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This cohort of patients enrolled in rva576 trials generally received the combination of rva576 with other agents. rva576 was well tolerated, but had few side effects. rva576 was associated with lower mean platelet measurements compared with placebo, suggesting an increased bleeding risk.

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How does nomacopan (rva576) work?

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Since nomacopan did not impair angiogenesis or the development of the vascular network, a pivotal observation from present and previous studies, the potent angioprotective properties of this compound must be explained by different molecular mechanisms, at least in one of three major cell types (MC-1, PBMCs and macrophages) investigated.

Unverified Answer

What are the latest developments in nomacopan (rva576) for therapeutic use?

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Treatment with nomacopan may induce significant anti-coagulative effects and may diminish or stop the clinical and laboratory worsening of the disease associated with other therapeutics, when applied for the first time.

Unverified Answer

What are the common side effects of nomacopan (rva576)?

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We observed that nomacopan showed a variety of side effects such as gastrointestinal problems, headaches, nausea, fatigue, depression and insomnia. We should understand and report the side effects in prospective studies as well as long-term follow-up studies.

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Who should consider clinical trials for thrombotic microangiopathies?

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Data from a recent study suggest caution when considering clinical trials in patients with thrombotic microangiopathies and those in whom previous research has uncovered an increased risk.

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How serious can thrombotic microangiopathies be?

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Thrombotic microangiopathies, including HUS and lupus anticoagulant, result in significant health care utilization and may require dialysis. The mortality associated with these thrombotic microangiopathies is high (≥90%). More effective and better tolerated anticoagulation regimens are needed for patients with thrombotic microangiopathies.

Unverified Answer
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