Hemolytic Uremic SyndromePhase 3NCT00004465
Phase III Randomized Study of SYNSORB Pk in Children With E. Coli-Associated Hemolytic Uremic Syndrome
A Phase 3 study testing SYNSORB Pk in people with Hemolytic Uremic Syndrome, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 27 sites in 2 countries.
Trial summary
OBJECTIVES: I. Determine the effect of SYNSORB Pk therapy on mortality and frequency of severe extrarenal complications observed in children with acute stage E. coli-associated hemolytic uremic syndrome. II. Determine the effect of SYNSORB Pk therapy on the need for the duration of dialysis in these patients. III. Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Hemolytic Uremic Syndrome
- What is being tested?
- SYNSORB Pk
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 145 participants
- Where
- 27 sitesin 2 countries
- Study dates
- 27 Jul 1997to 14 Apr 2001
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
- Clinically diagnosed E. coli-associated diarrhea-associated hemolytic uremic syndrome (HUS)
- Diarrheal prodrome within 7 days before onset of disease
- No HUS associated with the following: Hereditary Post-bone marrow transplantation Streptococcus pneumoniae infection
- No prior catastrophic complications
--Patient Characteristics--
- Hematopoietic: Thrombocytopenia less than 140,000/mm3 Fragmented red blood cells
- Renal: Renal involvement (hematuria, proteinuria, or azotemia) No underlying glomerular disease
- Other: HIV negative No pre-existing structural abnormality or dysmotility syndrome of the gastrointestinal tract No inflammatory bowel disease (e.g., Crohn's disease, ulcerative colitis)
Trial locations and contacts
27 sites in 2 countries.
27 sites in 2 countries
United States26 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
Medical College of Georgia Hospital and Clinics
Augusta, Georgia, 30912-3620
Boston Floating Hospital Infants and Children
Boston, Massachusetts, 02111
Maimonides Medical Center
Brooklyn, New York, 11219
Children's Hospital of Buffalo
Buffalo, New York, 14222
University of Virginia
Charlottesville, Virginia, 22908
Ohio State University Children's Hospital
Columbus, Ohio, 43205-2696
Children's Hospital of Denver
Denver, Colorado, 80218
Indiana University Cancer Center
Indianapolis, Indiana, 46202-5265
St. Barnabas Medical Center
Livingston, New Jersey, 07039
University of Wisconsin Children's Hospital
Madison, Wisconsin, 53792-4108
North Shore University Hospital
Manhasset, New York, 11030
West Virginia University Hospitals
Morgantown, West Virginia, 26506-9162
Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08903
Long Island Jewish Medical Center
New Hyde Park, New York, 11040
Weill Medical College of Cornell University
New York, New York, 10021
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
Children's Hospital at St. Joseph's
Paterson, New Jersey, 07503
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
University of Rochester Medical Center
Rochester, New York, 14642
State University of New York Health Sciences Center - Stony Brook
Stony Brook, New York, 11790-7775
Jack D. Weiler Hospital of the Albert Einstein College of Medicine
The Bronx, New York, 10461
New York Medical College
Valhalla, New York, 10595
Alfred I. Dupont Institute
Wilmington, Delaware, 19899
Bowman Gray School of Medicine
Winston-Salem, North Carolina, 27157
About this study
Official title: Phase III Randomized Study of SYNSORB Pk in Children With E. Coli-Associated
OBJECTIVES: I. Determine the effect of SYNSORB Pk therapy on mortality and frequency of severe extrarenal complications observed in children with acute stage E. coli-associated hemolytic uremic syndrome.
II. Determine the effect of SYNSORB Pk therapy on the need for the duration of dialysis in these patients.
III. Determine the effect of SYNSORB Pk therapy on the recovery of renal function and resolution of urinary abnormalities in these patients.
Read the detailed description
PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, multicenter study. Patients are randomized to receive either SYNSORB Pk or placebo.
Patients receive oral SYNSORB Pk or placebo three times daily for 7 days. Patients are followed on days 7, 14, 28, and 60 after discharge from the hospital.
- Conditions
- Hemolytic Uremic Syndrome
- Being tested
- SYNSORB Pk, Placebo
- Keywords
- E. coli infection, bacterial infection, hemolytic uremic syndrome, immunologic disorders and infectious disorders, rare disease, renal and genitourinary disorders
- Medical subject headings
- Hemolytic-Uremic Syndrome, Escherichia coli Infections, Bacterial Infections, Immune System Diseases, Communicable Diseases, Rare Diseases, Urogenital Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SYNSORB PkDrug
Also called Oral Shiga Toxin Binding Agent
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| SYNSORB Pk | Experimental | Oral Shiga toxin-binding agent (500 mg/kg/day) Receives: Drug: SYNSORB Pk |
| Placebo | Placebo comparator | Cornmeal placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to death or serious extrarenal events
Measured: 60 days
Time to dialysis
Measured: 60 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 1999 (estimated)
- Study start27 Jul 1997
- Primary completion14 Apr 2001
- Study completion14 Apr 2001
Why it stopped: Lack of efficacy at review by DSMB using pre-specified monitoring criteria
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Hemolytic Uremic SyndromeStudies of the same condition, open and under way.
- More trials run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 145 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- Long Island Jewish Medical Center
- Responsible party
- Sponsor
- Investigators
- Howard Trachtman, Study chair, Long Island Jewish Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004465
- Sponsor's protocol ID
- 199/13923
- Other IDs
- LIJMC-96-5-186, R01DK052147
- Status last verified
- May 2017
- First submitted
- 18 Oct 1999
- Last updated
- 1 Jun 2017
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Trachtman H, Cnaan A, Christen E, Gibbs K, Zhao S, Acheson DW, Weiss R, Kaskel FJ, Spitzer A, Hirschman GH; Investigators of the HUS-SYNSORB Pk Multicenter Clinical Trial. Effect of an oral Shiga toxin-binding agent on diarrhea-associated hemolytic uremic syndrome in children: a randomized controlled trial. JAMA. 2003 Sep 10;290(10):1337-44. doi: 10.1001/jama.290.10.1337.
PubMed 12966125Imdad A, Nelson JR, Tanner-Smith EE, Huang D, Gomez-Duarte OG. Interventions for preventing diarrhoea-associated haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2025 Apr 25;4(4):CD012997. doi: 10.1002/14651858.CD012997.pub3.
PubMed 40277027Imdad A, Mackoff SP, Urciuoli DM, Syed T, Tanner-Smith EE, Huang D, Gomez-Duarte OG. Interventions for preventing diarrhoea-associated haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2021 Jul 5;7(7):CD012997. doi: 10.1002/14651858.CD012997.pub2.
PubMed 34219224
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase III Randomized Study of SYNSORB Pk in Children With E. Coli-Associated
- ClinicalTrials.gov ID
- NCT00004465
- Acronym / sponsor ID
- 199/13923
- Conditions
- Hemolytic Uremic Syndrome
- Keywords
- E. coli infection, bacterial infection, hemolytic uremic syndrome, immunologic disorders and infectious disorders, rare disease, renal and genitourinary disorders
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of efficacy at review by DSMB using pre-specified monitoring criteria
- Enrolment
- 145 participants (actual)
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)US National Institutes of Health
- Collaborators
- Long Island Jewish Medical Center
- Study start
- 27 Jul 1997 (actual)
- Primary completion
- 14 Apr 2001 (actual)
- Study completion
- 14 Apr 2001 (actual)
- First posted
- 19 Oct 1999 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
OBJECTIVES: I. Determine the effect of SYNSORB Pk therapy on mortality and frequency of severe extrarenal complications observed in children with acute stage E. coli-associated hemolytic uremic syndrome.
II. Determine the effect of SYNSORB Pk therapy on the need for the duration of dialysis in these patients.
III. Determine the effect of SYNSORB Pk therapy on the recovery of renal function and resolution of urinary abnormalities in these patients.
Detailed description
PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, multicenter study. Patients are randomized to receive either SYNSORB Pk or placebo.
Patients receive oral SYNSORB Pk or placebo three times daily for 7 days. Patients are followed on days 7, 14, 28, and 60 after discharge from the hospital.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SYNSORB Pk | Experimental | Drug: SYNSORB PkOral Shiga toxin-binding agent (500 mg/kg/day) |
| Placebo | Placebo comparator | Drug: PlaceboCornmeal placebo |
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SYNSORB PkAlso: Oral Shiga Toxin Binding Agent | Drug | SYNSORB Pk |
| Placebo | Drug | Placebo |
Primary outcome
Time to death or serious extrarenal events
Time frame: 60 days
Time to dialysis
Time frame: 60 days
Results and safety
The study was stopped early (reason given: Lack of efficacy at review by DSMB using pre-specified monitoring criteria). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2017.
- Results publication: PubMed 12966125Trachtman H, Cnaan A, Christen E, Gibbs K, Zhao S, Acheson DW, Weiss R, Kaskel FJ, Spitzer A, Hirschman GH; Investigators of the HUS-SYNSORB Pk Multicenter Clinical Trial. Effect of an oral Shiga toxin-binding agent on diarrhea-associated hemolytic uremic syndrome in children: a randomized controlled trial. JAMA. 2003 Sep 10;290(10):1337-44. doi: 10.1001/jama.290.10.1337.
- Derived from this study: PubMed 40277027Imdad A, Nelson JR, Tanner-Smith EE, Huang D, Gomez-Duarte OG. Interventions for preventing diarrhoea-associated haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2025 Apr 25;4(4):CD012997. doi: 10.1002/14651858.CD012997.pub3.
- Derived from this study: PubMed 34219224Imdad A, Mackoff SP, Urciuoli DM, Syed T, Tanner-Smith EE, Huang D, Gomez-Duarte OG. Interventions for preventing diarrhoea-associated haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2021 Jul 5;7(7):CD012997. doi: 10.1002/14651858.CD012997.pub2.
- Individual participant dataSharing planned: No
Related trials for Hemolytic Uremic Syndrome
Active, not recruitingPhase 3
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Atypical Hemolytic Uremic Syndrome
RecruitingObservational study
Collection of Human Samples to Study Hairy Cell and Other Leukemias, and to Develop Recombinant Immunotoxins for Cancer Treatment
Hairy Cell Leukemia (HCL), Chronic Lymphocytic Leukemia (CLL)
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Active, not recruitingObservational study
A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome Patients
Atypical Hemolytic Uremic Syndrome(aHUS)
Active, not recruitingPhase 3
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Atypical Hemolytic Uremic Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Howard Trachtman study chair
Long Island Jewish Medical Center
27 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing SYNSORB Pk in people with Hemolytic Uremic Syndrome, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 27 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 1 Jun 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Lack of efficacy at review by DSMB using pre-specified monitoring criteria). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to death or serious extrarenal events (time frame: 60 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00004465, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jun 2017.