Phase III Randomized Study of SYNSORB Pk in Children With E. Coli-Associated Hemolytic Uremic Syndrome

NCT00004465 | Terminated Phase 3 DMC FDA Drug

• 3 other identifiers: 199/13923LIJMC-96-5-186R01DK052147
• Study Type: interventional
• Target: 145
• Locations: 2 countries
• Sites: 27

Brief 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. Determine the effect of SYNSORB Pk therapy on the recovery of renal function and resolution of urinary abnormalities in these patients.

Conditions

Hemolytic Uremic Syndrome

Keywords

E. coli infection; bacterial infection; hemolytic uremic syndrome; immunologic disorders and infectious disorders; rare disease; renal and genitourinary disorders

Outcome Measures

Primary Outcomes (2)

• Time to death or serious extrarenal events

  60 days

• Time to dialysis

  60 days

Study Arms (2)

SYNSORB Pk

EXPERIMENTAL

Oral Shiga toxin-binding agent (500 mg/kg/day)

Drug: SYNSORB Pk

Placebo

PLACEBO COMPARATOR

Cornmeal placebo

Drug: Placebo

Interventions

SYNSORB Pk DRUG

Also known as: Oral Shiga Toxin Binding Agent

SYNSORB Pk

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 6 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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)

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): lead
• Long Island Jewish Medical Center: collaborator

Study Sites (27)

Children's Hospital of Denver

Denver, Colorado, 80218, United States

Location

Alfred I. Dupont Institute

Wilmington, Delaware, 19899, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30322, United States

Location

Medical College of Georgia Hospital and Clinics

Augusta, Georgia, 30912-3620, United States

Location

Indiana University Cancer Center

Indianapolis, Indiana, 46202-5265, United States

Location

Boston Floating Hospital Infants and Children

Boston, Massachusetts, 02111, United States

Location

St. Barnabas Medical Center

Livingston, New Jersey, 07039, United States

Location

Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08903, United States

Location

Children's Hospital at St. Joseph's

Paterson, New Jersey, 07503, United States

Location

Maimonides Medical Center

Brooklyn, New York, 11219, United States

Location

Children's Hospital of Buffalo

Buffalo, New York, 14222, United States

Location

North Shore University Hospital

Manhasset, New York, 11030, United States

Location

Long Island Jewish Medical Center

New Hyde Park, New York, 11040, United States

Location

Weill Medical College of Cornell University

New York, New York, 10021, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

State University of New York Health Sciences Center - Stony Brook

Stony Brook, New York, 11790-7775, United States

Location

Jack D. Weiler Hospital of the Albert Einstein College of Medicine

The Bronx, New York, 10461, United States

Location

New York Medical College

Valhalla, New York, 10595, United States

Location

Bowman Gray School of Medicine

Winston-Salem, North Carolina, 27157, United States

Location

Ohio State University Children's Hospital

Columbus, Ohio, 43205-2696, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Virginia

Charlottesville, Virginia, 22908, United States

Location

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23507, United States

Location

West Virginia University Hospitals

Morgantown, West Virginia, 26506-9162, United States

Location

University of Wisconsin Children's Hospital

Madison, Wisconsin, 53792-4108, United States

Location

University of Alberta

Edmonton, Alberta, T6G 2R7, Canada

Location

Related Publications (3)

• 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.

  PMID: 12966125 RESULT

• Imdad 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.

  PMID: 40277027 DERIVED

• Imdad 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.

  PMID: 34219224 DERIVED

MeSH Terms

Conditions

Hemolytic-Uremic Syndrome; Escherichia coli Infections; Bacterial Infections; Immune System Diseases; Communicable Diseases; Rare Diseases; Urogenital Diseases

Interventions

SYNSORB Pk

Condition Hierarchy (Ancestors)

Uremia; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Male Urogenital Diseases; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Enterobacteriaceae Infections; Gram-Negative Bacterial Infections; Bacterial Infections and Mycoses; Infections; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Howard Trachtman

  Long Island Jewish Medical Center

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 18, 1999
• First Posted: October 19, 1999
• Study Start: July 27, 1997
• Primary Completion: April 14, 2001
• Study Completion: April 14, 2001
• Last Updated: June 1, 2017

Record last verified: 2017-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (26); CA (1)