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Retreatment and Its Efficiency of Thalidomide for Vascular Malformation Patients With Failure of First Course Treatment

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ClinicalTrials.gov Identifier: NCT02301949
Recruitment Status : Withdrawn
First Posted : November 26, 2014
Last Update Posted : April 21, 2016
Sponsor:
Information provided by (Responsible Party):
Zhizheng Ge, Shanghai Jiao Tong University School of Medicine

Brief Summary:

Background: Repeated episodes of bleeding from gastrointestinal vascular malformations refractory to endoscopic or surgical therapy often pose a major therapeutic challenge.

Methods: The investigators will perform a randomized, double blind, placebo controlled study of thalidomide as a retreatment therapy for recurrent gastrointestinal bleeding due to vascular malformation. Patients with failure of first course treatment of thalidomide will be randomly grouped, prescribed a second four-month course regimen of 25 mg of thalidomide or placebo orally four times daily. All patients will be monitored for at least one year. The primary end point is defined as the patients whose rebleeds decrease from baseline by ≥ 50% at 12 months and the cessation of bleeding. Rebleeding is defined based on a positive fecal occult blood test (FOBT) (monoclonal colloidal gold color technology) at any visit after treatment. Secondary outcomes include the participants dependent on blood transfusions and changes from baseline in transfused packed red cell units, bleeding episodes, and hemoglobin levels at 12 months. Statistical significance is defined at P < 0.05.


Condition or disease Intervention/treatment Phase
Gastrointestinal Bleeding of Unknown Origin Gastrointestinal Vascular Malformation Thalidomide Efficiency Drug: Thalidomide Other: Placebo Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: The Study of the Optimal Treatment Strategy for Patients With Gastrointestinal Bleeding Due to Gastrointestinal Vascular Malformation: a Randomized, Double Blind, Placebo Controlled Study
Study Start Date : December 2015
Estimated Primary Completion Date : December 2015
Estimated Study Completion Date : December 2018


Arm Intervention/treatment
Active Comparator: Thalidomide Retreatment Group Drug: Thalidomide
Patients are randomly assigned to receive a second course of four-month treatment of thalidomide (Pharmaceutical Co., Ltd. of ChangZhou, China). Medications are taken orally 25mg four times daily at 6 a.m., 12 noon, 6 p.m., and 10 p.m.
Other Name: Softenon

Placebo Comparator: Placebo Group Other: Placebo
Patients are randomly assigned to receive placebo tablets (Pharmaceutical Co., Ltd. of ChangZhou, China) four times daily at 6 a.m., 12 noon, 6 p.m., and 10 p.m.




Primary Outcome Measures :
  1. The primary end point is defined as the patients whose rebleeds decrease from baseline by ≥ 50% at 12 months [ Time Frame: 12 months ]
    The primary end point is defined as the patients whose rebleeds decrease from baseline by ≥ 50% at 12 months. Reduction of rebleeds = [(total bleeding episode at 12 months - total bleeding episodes at a year before randomization)/total bleeding episodes at a year before randomization(baseline)]*100%. Rebleeding is defined based on a positive fecal occult blood test (FOBT) (monoclonal colloidal gold color technology) at any visit after treatment.


Secondary Outcome Measures :
  1. Change From Baseline in Hemoglobin (Hb) Level at 12 Months [ Time Frame: 12 months ]
    The change from baseline in average hemoglobin (Hb) level(tested every month) at 12 months.

  2. Change From Baseline in Bleeding Episodes at 12 Months [ Time Frame: 12 months ]
    The Change from baseline in bleeding episodes at 12 months

  3. Participants Dependent on Blood Transfusions [ Time Frame: 12 months ]
    Numbers of participants dependent on blood transfusions

  4. Change From Baseline in Total Transfused Red Cell Requirements at 12 Months [ Time Frame: 12 months ]
    Change of total transfused red cell requirements at 12 months after randomization from one year before baseline in transfusion dependent patients

  5. Cessation of Bleeding [ Time Frame: 12 months ]
    The cessation of bleeding is defined as repeated negative faecal occult blood test (FOBT) (monoclonal colloidal gold color technology) during our observation period. Rebleeding is defined based on a positive FOBT at any visit after treatment.



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Ages Eligible for Study:   35 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age between 35-85 years; women are post-menopausal, post-tubal ligation, or on some form of birth control like long-term laying up contraceptive ring or using condom;
  • Patients with failure of first course treatment of thalidomide, which means rebleeds decreased from baseline by< 50% at 12 months follow up ;
  • History of at least six documented gastrointestinal bleeding episodes in the year prior first course thalidomide treatment, which are refractory or inaccessible to endoscopic therapy or surgical ectomy; so, patients should have at least four episodes of gastrointestinal bleeding a year prior our study;
  • Confirmed diagnosis of vascular malformation by esophagogastroduodenoscopy (EGD), capsule endoscope (CE), double-balloon endoscope (DBE), or colonoscopy, but no obvious infectious, neoplastic, or other specific diagnosis;
  • Angiodysplasia at endoscopy characterized by focal or diffused venous/capillary lesions presenting as bright red ectatic vessels or pulsatile red protrusions, with surrounding venous dilatation or patchy erythema with or without oozing;
  • Endoscopic appearance of GAVE (also known as watermelon stomach), indicated by longitudinal antral folds converging on the pylorus, containing visible columns of tortuous red ecstatic vessels.

Exclusion Criteria:

  • Patients are excluded if first course treatment of thalidomide is effective, which means rebleeds decreased from baseline by ≥ 50% at 12 months follow up;
  • if they have cirrhotic or portal hypertension gastropathy; severe co-morbidities of cardiac, pulmonary, renal, liver, hematological, rheumatologic disorders, or uncontrollable diabetes mellitus or hypertension;
  • if they have a history of severe bilateral peripheral neuropathy or seizure activity, thromboembolic disease, known thalidomide allergy;
  • if they have a history of treatment with any dose of systemic or oral topical corticosteroids or aspirin, NSAIDs, anti-platelet drugs, anticoagulants, or Chinese medications (with salicylates), gingko, or Echinacea, or other putative immunomodulators or anti-angiogenic agents;
  • Currently pregnant or lactating or currently undergoing systemic cancer chemotherapy or receiving radiation

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 ClinicalTrials.gov identifier (NCT number): NCT02301949


Locations
China, Shanghai
Department of Gastroenterology, Renji Hospital, Shanghai Institute of Digestive Diseases, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai, China, 200127
Sponsors and Collaborators
Zhizheng Ge
Investigators
Principal Investigator: Zhizheng Ge, MD. Ph.D Shanghai Ren Ji Hospital

Additional Information:
Publications:
Responsible Party: Zhizheng Ge, Shanghai Institute of Digestive Disease,Renji Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai Jiao Tong University School of Medicine
ClinicalTrials.gov Identifier: NCT02301949     History of Changes
Other Study ID Numbers: rjyyxhnj3015
First Posted: November 26, 2014    Key Record Dates
Last Update Posted: April 21, 2016
Last Verified: April 2016

Keywords provided by Zhizheng Ge, Shanghai Jiao Tong University School of Medicine:
gastrointestinal bleeding
vascular malformation
thalidomide retreatment

Additional relevant MeSH terms:
Vascular Malformations
Hemorrhage
Congenital Abnormalities
Gastrointestinal Hemorrhage
Pathologic Processes
Gastrointestinal Diseases
Digestive System Diseases
Cardiovascular Abnormalities
Cardiovascular Diseases
Thalidomide
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents