A Phase 2 interventional study of Eculizumab in Thrombotic Microangiopathies, Atypical Hemolytic Uremic Syndrome and Multiple Organ Dysfunction Syndrome, sponsored by Children's Hospital Medical Center, Cincinnati. Completed at 3 sites in United States. Per ClinicalTrials.gov, last updated 2023-09-21.
Sponsored by Children's Hospital Medical Center, Cincinnati · Phase 2, Interventional, and Treatment
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.
This clinical trial is a prospective single arm multi-institution study in children and young adults undergoing allogeneic or autologous hematopoietic stem cell transplantation who will receive early therapy with eculizumab to prevent TMA-associated MODS after transplantation. The purpose of this research study is to examine efficacy of complement blocker eculizumab in HCT recipients with high risk TMA and to determine optimal eculizumab dosing regimen for HCT recipients with TMA using PK/PD studies. All patients will receive therapy based on their weight for 24 weeks. Survival will be assessed at 6 months from TMA diagnosis.
45 studies on the registry are indexed under Atypical Hemolytic Uremic Syndrome; 14 are open to participants now.
This study's enrollment of 23 is below the median of 34 across 26 interventional studies indexed under Atypical Hemolytic Uremic Syndrome.
Browse Atypical Hemolytic Uremic Syndrome studies →Children's Hospital Medical Center, Cincinnati is the lead sponsor of 673 studies on the registry; 130 are open to participants now.
Of its 57 completed or terminated interventional studies of FDA-regulated products, 31 (54%) have results posted.
Compared within Atypical Hemolytic Uremic Syndrome, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Thrombotic Microangiopathies. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
All patients will receive eculizumab based on their weight for 24 weeks.
Drug: Eculizumab
Eculizumab will be administered as intravenous infusion (IV) over 60 minutes. The dosage form will be 300 mg single-use vials each containing 30 mL of 10 mg/mL sterile, preservative-free solution.
Also known as: Soliris
Survival
Survival at 6 months after the date of TMA diagnosis
Time frame: 6 months
Number of Participants With Organ Dysfunction
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.
Time frame: 6 months
Number of Participants With Organ Dysfunction
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.
Time frame: 1 year
Non-relapse Mortality
Non-relapse mortality descriptively compared with historical controls at 1 year
Time frame: 1 year
| Milestone | Eculizumab |
|---|---|
| Started | 23 |
| Completed | 21 |
| Not completed | 2 |
| Withdrew: The participant died on study prior to receiving 4 doses and was not evaluable. | 1 |
| Withdrew: Relapsed with primary malignancy within 6 months of tma diagnosis making participant unevaluable. | 1 |
Survival at 6 months after the date of TMA diagnosis
| Participants | Eculizumab |
|---|---|
| Survival | 15 |
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.
| Participants | Eculizumab |
|---|---|
| Number of Participants With Organ Dysfunction | 4 |
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.
| Participants | Eculizumab |
|---|---|
| Number of Participants With Organ Dysfunction | 2 |
Non-relapse mortality descriptively compared with historical controls at 1 year
| Participants | Eculizumab |
|---|---|
| Non-relapse Mortality | 8 |
Collected over 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.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Eculizumab | 9/23 (39.1%) | 19/23 (82.6%) | 22/23 (95.7%) |
| Event | Eculizumab |
|---|---|
| Acute kidney injuryRenal and urinary disorders | 8/23 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 8/23 |
| SepsisInfections and infestations | 5/23 |
| FeverGeneral disorders | 3/23 |
| Multi organ failureInfections and infestations | 3/23 |
| Possible/Rule Out SepsisInfections and infestations | 3/23 |
| SeizureNervous system disorders | 3/23 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 3/23 |
| HypotensionVascular disorders | 3/23 |
| Febrile neutropeniaBlood and lymphatic system disorders | 2/23 |
| Event | Eculizumab |
|---|---|
| HypokalemiaMetabolism and nutrition disorders | 14/23 |
| Increased aspartate aminotransferaseInvestigations | 9/23 |
| Increased alanine aminotransferaseInvestigations | 8/23 |
| Abdominal painGastrointestinal disorders | 4/23 |
| BacteremiaInfections and infestations | 4/23 |
| End stage renal disease (or CKD)Renal and urinary disorders | 4/23 |
| Blood bilirubin increasedInfections and infestations | 3/23 |
| CMV viremia reactivationInfections and infestations | 3/23 |
| HypoalbuminemiaMetabolism and nutrition disorders | 3/23 |
| HyperglycemiaMetabolism and nutrition disorders | 3/23 |
| Age, Categorical(Participants) | Eculizumab |
|---|---|
| <=18 years | 20 |
| Between 18 and 65 years | 3 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | Eculizumab |
|---|---|
| Female | 17 |
| Male | 6 |
| Race (NIH/OMB)(Participants) | Eculizumab |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 2 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 3 |
| White | 17 |
| More than one race | 1 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Eculizumab |
|---|---|
| United States | 23 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Aug 2018 to primary completion in Apr 2022, 44 months.
Results were first posted in Sep 2023, 1 year 5 months after primary completion.
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Studies of the drug tested here: Eculizumab
Atypical Hemolytic Uremic Syndrome →
Children's Hospital Medical Center, Cincinnati →