Survival
Primary
· 6 months
Survival at 6 months after the date of TMA diagnosis
| Group | Value | 95% CI |
|---|---|---|
| Eculizumab | 15 |
Last reviewed · How we verify
NCT03518203
Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome -Associated Multiple Organ Dysfunction Syndrome in Hematopoietic Stem Cell Transplant Recipients
Completed
Phase 2
Results posted
Last updated 21 September 2023
What this trial tests
Phase 2 trial testing Eculizumab in Thrombotic Microangiopathies in 23 participants. Completed in 1 June 2022.
Timeline
3 August 2018
Primary endpoint
4 April 2022
4 April 2022
1 June 2022
Quick facts
| Lead sponsor | Children's Hospital Medical Center, Cincinnati |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 23 |
| Start date | 3 August 2018 |
| Primary completion | 4 April 2022 |
| Estimated completion | 1 June 2022 |
| Sites | 3 locations across United States |
Drugs / interventions tested
- Eculizumab (ECULIZUMAB) — full drug profile →
Conditions studied
- Thrombotic Microangiopathies — all drugs for Thrombotic Microangiopathies →
- Atypical Hemolytic Uremic Syndrome — all drugs for Atypical Hemolytic Uremic Syndrome →
- Multiple Organ Dysfunction Syndrome — all drugs for Multiple Organ Dysfunction Syndrome →
Sponsor
Children's Hospital Medical Center, Cincinnati
Who can join
Eligibility, any sex, with Thrombotic Microangiopathies or Atypical Hemolytic Uremic Syndrome. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Organ Dysfunction
Secondary
· 6 months
Number of participants with organ dysfunction at 6 months after TMA diagnosis. Organ dysfunction definitions are listed in the protocol Appendix II that is uploaded to ClinicalTrials.gov site.
| Group | Value | 95% CI |
|---|---|---|
| Eculizumab | 4 |
Number of Participants With Organ Dysfunction
Secondary
· 1 year
Number of participants with organ dysfunction at 1 year after TMA diagnosis. Organ dysfunction definitions are listed in the protocol Appendix II that is uploaded to ClinicalTrials.gov site.
| Group | Value | 95% CI |
|---|---|---|
| Eculizumab | 2 |
Non-relapse Mortality
Secondary
· 1 year
Non-relapse mortality descriptively compared with historical controls at 1 year
| Group | Value | 95% CI |
|---|---|---|
| Eculizumab | 8 |
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Eculizumab
Serious: 19/23 (83%)
Deaths: 9/23
Serious adverse events (42 terms)
| Reaction | System | Eculizumab |
|---|---|---|
| Acute kidney injury | Renal and urinary disorders | — |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | — |
| Sepsis | Infections and infestations | — |
| Fever | General disorders | — |
| Multi organ failure | Infections and infestations | — |
| Possible/Rule Out Sepsis | Infections and infestations | — |
| Seizure | Nervous system disorders | — |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | — |
| Hypotension | Vascular disorders | — |
| Febrile neutropenia | Blood and lymphatic system disorders | — |
| Cardiac arrest | Cardiac disorders | — |
| Vomiting | Gastrointestinal disorders | — |
| Altered mental status | Nervous system disorders | — |
| Renal failure | Renal and urinary disorders | — |
| Anemia | Blood and lymphatic system disorders | — |
| Cardiac Tamponade | Cardiac disorders | — |
| Decreased left ventricular ejection fraction | Cardiac disorders | — |
| Pericardial effusion | Cardiac disorders | — |
| Abdominal pain, right upper quadrant | Gastrointestinal disorders | — |
| Lower gastrointestinal hemorrhage | Gastrointestinal disorders | — |
| Nausea | Gastrointestinal disorders | — |
| Graft versus Host Disease (liver and gastrointestinal) | General disorders | — |
| Sinusoidal obstruction syndrome | Hepatobiliary disorders | — |
| Cellulitis | Infections and infestations | — |
| Platelet count decreased | Investigations | — |
Other adverse events (77 terms — click to expand)
| Reaction | System | Eculizumab |
|---|---|---|
| Hypokalemia | Metabolism and nutrition disorders | — |
| Increased aspartate aminotransferase | Investigations | — |
| Increased alanine aminotransferase | Investigations | — |
| Abdominal pain | Gastrointestinal disorders | — |
| Bacteremia | Infections and infestations | — |
| End stage renal disease (or CKD) | Renal and urinary disorders | — |
| Blood bilirubin increased | Infections and infestations | — |
| CMV viremia reactivation | Infections and infestations | — |
| Hypoalbuminemia | Metabolism and nutrition disorders | — |
| Hyperglycemia | Metabolism and nutrition disorders | — |
| Anemia | Blood and lymphatic system disorders | — |
| Febrile neutropenia | Blood and lymphatic system disorders | — |
| Diarrhea | Gastrointestinal disorders | — |
| Graft versus host disease | Gastrointestinal disorders | — |
| Lower GI hemorrhage | Gastrointestinal disorders | — |
| Nausea | Gastrointestinal disorders | — |
| Vomiting | Gastrointestinal disorders | — |
| Fever | General disorders | — |
| Adenoviremia | Infections and infestations | — |
| EBV reactivation | Infections and infestations | — |
| Fungemia | Infections and infestations | — |
| Sinusitis | Infections and infestations | — |
| Neutrophil count decreased | Investigations | — |
| Platelet count decreased | Investigations | — |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | — |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | — |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | — |
| Pneumonia | Respiratory, thoracic and mediastinal disorders | — |
| Productive cough | Respiratory, thoracic and mediastinal disorders | — |
| Skin graft versus host disease | Skin and subcutaneous tissue disorders | — |
| Hypertension | Vascular disorders | — |
| Increased cardiac troponin | Cardiac disorders | — |
| Biventricular dysfunction | Cardiac disorders | — |
| Pericardial effusion | Cardiac disorders | — |
| Ocular graft versus host disease | Eye disorders | — |
| Gastric hemorrhage | Gastrointestinal disorders | — |
| Ileus | Gastrointestinal disorders | — |
| Generalized edema | General disorders | — |
| TMA reactivation unresponsive to treatment | General disorders | — |
| BK viremia | Infections and infestations | — |
Most-reported serious reactions: Acute kidney injury, Respiratory failure, Sepsis, Fever, Multi organ failure, Possible/Rule Out Sepsis, Seizure, Hypoxia.
Data from ClinicalTrials.gov NCT03518203 adverse events section.
Sponsor's own description
Hematopoietic stem cell transplantation (HCT)-associated thrombotic microangiopathy (TMA) is an understudied complication of HCT that significantly affects transplant related morbidity and mortality. The investigators hypothesize that early intervention with complement blocker eculizumab will double survival in HCT recipients with high risk TMA, as compared to historical untreated controls. An optimal eculizumab dosing schedule can be determined for this population through eculizumab pharmacokinetic/pharmacodynamic (PK/PD) testing.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
-
Clinical promise of next-generation complement therapeutics.
Mastellos DC, Ricklin D, Lambris JD. · · 2019 · cited 255× · PMID 31324874 · DOI 10.1038/s41573-019-0031-6 -
A prospective multi-institutional study of eculizumab to treat high-risk stem cell transplantation-associated TMA.
Jodele S, Dandoy CE, Aguayo-Hiraldo P, Lane A, et al · · 2024 · cited 35× · PMID 37946262 · DOI 10.1182/blood.2023022526 -
Treatment of Rare Inflammatory Kidney Diseases: Drugs Targeting the Terminal Complement Pathway.
Anliker-Ort M, Dingemanse J, van den Anker J, Kaufmann P. · · 2020 · cited 33× · PMID 33362783 · DOI 10.3389/fimmu.2020.599417 -
Emerging Role of C5 Complement Pathway in Peripheral Neuropathies: Current Treatments and Future Perspectives.
Giorgio C, Zippoli M, Cocchiaro P, Castelli V, et al · · 2021 · cited 29× · PMID 33917266 · DOI 10.3390/biomedicines9040399 -
Role of the lectin pathway of complement in hematopoietic stem cell transplantation-associated endothelial injury and thrombotic microangiopathy.
Gavriilaki E, Ho VT, Schwaeble W, Dudler T, et al · · 2021 · cited 28× · PMID 34924021 · DOI 10.1186/s40164-021-00249-8 -
The Syndromes of Thrombotic Microangiopathy: A Critical Appraisal on Complement Dysregulation.
Timmermans SAMEG, van Paassen P. · · 2021 · cited 25× · PMID 34300201 · DOI 10.3390/jcm10143034 -
Complement Mediated Endothelial Damage in Thrombotic Microangiopathies.
Blasco M, Guillén-Olmos E, Diaz-Ricart M, Palomo M. · · 2022 · cited 22× · PMID 35547236 · DOI 10.3389/fmed.2022.811504 -
Hematopoietic stem cell transplantation-associated thrombotic microangiopathy and the role of advanced practice providers and pharmacists.
Mahmoudjafari Z, Alencar MC, Alexander MD, Johnson DJ, et al · · 2023 · cited 12× · PMID 37059738 · DOI 10.1038/s41409-023-01951-3
Verify or expand the search:
- PubMed search for NCT03518203
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
Data sources for this page
- Trial protocol + status: ClinicalTrials.gov NCT03518203 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 9 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Children's Hospital Medical Center, Cincinnati
- Last refreshed: 21 September 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03518203.
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