Drug could double survival in rare stem cell transplant complication
NCT ID NCT07707687
First seen Jul 16, 2026 · Last updated Jul 17, 2026 · Updated 1 time
Summary
This trial tests the drug eculizumab in adults who develop a severe blood clotting disorder called high-risk transplant-associated thrombotic microangiopathy (TA-TMA) after a stem cell transplant. The condition can cause multiple organ failure and has a poor outlook. The study aims to see if eculizumab can improve survival at 6 months compared to historical outcomes. Participants receive weight-based doses over up to 24 weeks.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- eculizumab, a complement C5 inhibitor
- What this could lead to
- If successful, this could offer a targeted treatment for a life-threatening complication after stem cell transplants, potentially doubling survival rates.
- What could go wrong
- This is a small, early-phase study with only 24 participants and no control group, so results may not be definitive. Eculizumab also carries risks like increased susceptibility to infections.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 24 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Jul 2026
An estimate. Start dates often move.
- Expected to finish
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Jul 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion 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 Presence of schistocytes on peripheral blood smear;③ New-onset thrombocytopenia or requirement for platelet transfusions;④ New-onset anemia or requirement for red blood cell transfusions;⑤ Hypertension;⑥ Random urine protein-to-creatinine ratio (rUPCR) ≥1 mg/mg;⑦ Elevated plasma soluble C5b-9 (sC5b-9) level (≥244 ng/mL). * 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, CNI); ② Treatment of any underlying infection; ③ Treatment of underlying acute graft-versus-host disease (aGVHD). * Provide written informed consent. Exclusion Criteria: * 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
8 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Jinhua Central Hospital
RECRUITINGJinhua, Zhejiang, China
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Sir Run Run Shaw Hospital, Zhejiang University School of Medicine
RECRUITINGHangzhou, Zhejiang, China
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The Affiliated People's Hospital of Ningbo University
RECRUITINGNingbo, Zhejiang, China
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The First Affiliated Hospital of Ningbo University
RECRUITINGNingbo, Zhejiang, China
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The First Affiliated Hospital of Wenzhou Medical University
RECRUITINGWenzhou, Zhejiang, China
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The First Affiliated Hospital, Zhejiang University School of Medicine.
RECRUITINGHangzhou, Zhejiang, China
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The Second Affiliated Hospital of Zhejiang University School of Medicine
RECRUITINGHangzhou, Zhejiang, China
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Xiangya Hospital of Central South University
RECRUITINGChangsha, Hunan, China