Phase II Pilot Study of Cladribine (2-Chlorodeoxyadenosine; 2-CdA) for Early Stage Primary Sclerosing Cholangitis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00004762
Recruitment Status : Completed
First Posted : February 25, 2000
Last Update Posted : June 24, 2005
Scripps Clinic
Information provided by:
Office of Rare Diseases (ORD)

Brief Summary:


I. Evaluate the effects of cladribine (2-chlorodeoxyadenosine; 2-CdA) on biochemical, radiologic, and histologic parameters in patients with early stage primary sclerosing cholangitis.

Condition or disease Intervention/treatment Phase
Cholangitis, Sclerosing Drug: cladribine Phase 2

Detailed Description:


Patients are treated with subcutaneous injections of cladribine (2-chlorodeoxyadenosine; 2-CdA) for 5 consecutive days every month for 3 months.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Primary Purpose: Treatment
Study Start Date : December 1994

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics--

Stage I-III primary sclerosing cholangitis

Radiologically and pathologically documented

No concomitant liver disease, e.g.:

  • Viral hepatitis
  • Autoimmune hepatitis
  • Primary biliary cirrhosis
  • Cirrhosis
  • Portal hypertension or associated complications
  • Jaundice caused by dominant stricture

--Prior/Concurrent Therapy--

No concurrent immunosuppressives

--Patient Characteristics--


  • Absolute neutrophil count at least 2500/mm3
  • Platelet count at least 100,000/mm3
  • Hemoglobin at least 10 g/dL


  • No active infection
  • No fistula abscess
  • No active inflammatory bowel disease
  • Quiescent disease allowed, including: Chronic ulcerative colitis Crohn's disease
  • No other significant immunologic disorder
  • No active malignancy
  • No active alcohol or drug abuse
  • No pregnant or nursing women
  • Effective contraception required of fertile patients

Endoscopic retrograde cholangiopancreatography within 36 months prior to registration

Liver biopsy within 12 months prior to registration

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00004762

Sponsors and Collaborators
National Center for Research Resources (NCRR)
Scripps Clinic
Study Chair: Paul J. Pockros Scripps Clinic Identifier: NCT00004762     History of Changes
Other Study ID Numbers: 199/11707
First Posted: February 25, 2000    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: December 2001

Keywords provided by Office of Rare Diseases (ORD):
gastrointestinal disorders
primary sclerosing cholangitis
rare disease

Additional relevant MeSH terms:
Cholangitis, Sclerosing
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs