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Tests treatment safety and results for Transplant-Associated Thrombotic Microangiopathy (TA-TMA)
Official title Safety and Efficacy of Eculizumab in High-risk TA-TMA
ClinicalTrials.gov ID: NCT07707687
What this study is testing
What is Eculizumab?
Eculizumab is an investigational medicine, given as an once-weekly, being studied as a potential treatment for transplant-associated thrombotic microangiopathy (ta-tma).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- High-risk, complement-mediated, untreated transplant-associated thrombotic microangiopathy (hrTA-TMA) carries a very poor prognosis due to multiple organ dysfunction syndrome (MODS). The complement C5 inhibitor eculizumab has shown promising efficacy in children with hrTA-TMA, but has not been prospectively studied in adult allogeneic hematopoietic stem cell transplantation (HSCT) recipients.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Age ≥18 years.
- Patients who have undergone hematopoietic stem cell transplantation for any indication within 12 months prior to enrollment.
- Meet the diagnostic criteria for TA-TMA within ≤14 days prior to enrollment (at least 4 of the following 7 criteria present simultaneously):① Lactate...
- Meet the criteria for high-risk TA-TMA (presence of any of the following):① Proteinuria (rUPCR ≥2 mg/mg);②Multiple organ dysfunction syndrome...
- Meet the following condition: TA-TMA has not resolved after ≥72 hours of management of triggering factors/conditions, including: ① Discontinuation or...
You likely can't join if
- Known hypersensitivity to eculizumab.
- Uncontrolled severe infection (including meningococcal infection).
- Prior treatment with complement inhibitors.
- Known hereditary or acquired ADAMTS13 deficiency (activity \<10%).
- Disseminated intravascular coagulation (DIC).
- Respiratory failure (any cause) requiring mechanical ventilation, occurring within 72 hours prior to enrollment.
See the full eligibility criteria
- Age ≥18 years.
- Patients who have undergone hematopoietic stem cell transplantation for any indication within 12 months prior to enrollment.
- Meet the diagnostic criteria for TA-TMA within ≤14 days prior to enrollment (at least 4 of the following 7 criteria present simultaneously):① Lactate dehydrogenase (LDH) above the age-adjusted upper limit of normal;②...
- Meet the criteria for high-risk TA-TMA (presence of any of the following):① Proteinuria (rUPCR ≥2 mg/mg);②Multiple organ dysfunction syndrome (MODS);③ Elevated IL-10 (≥2× upper limit of normal [ULN]).
- Meet the following condition: TA-TMA has not resolved after ≥72 hours of management of triggering factors/conditions, including: ① Discontinuation or dose reduction of inciting medications (e.g., calcineurin inhibitors...
- Provide written informed consent.
- Known hypersensitivity to eculizumab.
- Uncontrolled severe infection (including meningococcal infection).
- Prior treatment with complement inhibitors.
- Known hereditary or acquired ADAMTS13 deficiency (activity \<10%).
- Disseminated intravascular coagulation (DIC).
- Respiratory failure (any cause) requiring mechanical ventilation, occurring within 72 hours prior to enrollment.
- Acute and/or chronic heart failure with ejection fraction ≤40%.
- Expected survival \<48 hours.
- Any subject who, in the investigator's opinion, is not suitable for participation in this study.
The study team makes the final eligibility decision.
Where it's taking place
- Changsha, Hunan, China
- Hangzhou, Zhejiang, China
- Jinhua, Zhejiang, China
- Ningbo, Zhejiang, China
- Wenzhou, Zhejiang, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Changsha, Hunan, China; Hangzhou, Zhejiang, China; Jinhua, Zhejiang, China; Ningbo, Zhejiang, China; Wenzhou, Zhejiang, China. Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.