Thalidomide Reduces Arteriovenous Malformation Related Gastrointestinal Bleeding

Hereditary Hemorrhagic Telangiectasia Clinical Trial

— TAG  

Official title:

Thalidomide Reduces Arteriovenous Malformation Related Gastrointestinal Bleeding

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00389935
• Other study ID #: 00198
• Status: Completed
• Phase: Phase 2
• First received: October 17, 2006
• Last updated: August 1, 2011
• Start date: October 2006
• Est. completion date: July 2011

Study information

• Verified date: July 2011
• Source: Northport Veterans Affairs Medical Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to determine whether Thalidomide is effective in the treatment of arteriovenous malformations in the gastrointestinal tract.

Description:

Arteriovenous malformations (AVM's) are the commonest vascular abnormalities of the gut. AVM's or Angiodysplasia may be acquired or inherited as in a hereditary hemorrhagic telangiectasia (HHT). Repeated episodes of gastrointestinal bleeding (GIB), especially in the elderly have been attributed to angiodysplasia. Clinically significant GIB may be also seen in up to 40% of HHT patients, usually in the fourth and fifth decades of life. GIB may manifest both as acute major hemorrhage, slow intermittent blood loss or a combination of these findings and patients may be symptomless, present with acute bleeding or iron deficiency anemia. Recurrent hemorrhage and persistent iron-deficiency anemia is common despite supplemental iron therapy and patients require repeated transfusions. Amongst patients with preexisting co-morbidities, repeated bleeding may lead to significant morbidity and mortality. Furthermore, re-bleeding among these patients consumes a disproportionate share of healthcare resources devoted to multiple admissions, repeated endoscopies and blood transfusions. There are no effective treatment options available currently. The purpose of this study is to determine whether Thalidomide is effective in the treatment of arteriovenous malformations in the gastrointestinal tract.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: July 2011
• Est. primary completion date: June 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Patients must be 18 years of age or older

• Patient must have had greater than 2 episodes of overt bleeding over last 2 years requiring > 4 units of PRBC for bleeding 20 AVM. AVM's should have been identified at optical/capsule endoscopy or angiography.

• Patients must have adequate hematologic, renal and liver function (i.e. Platelets = 100,000/mm3, Creatinine = 1.7mg/dl, Total Bilirubin = 2.5mg/dl, Transaminases = 4 times above the upper limits of the institutional norm)

• Patients must be able to provide written informed consent. Patients with the potential for pregnancy or impregnating their partner must agree to follow acceptable birth control methods (described in detail under thalidomide drug information section) to avoid conception. Women of child-bearing potential must have a negative pregnancy test prior to treatment on this protocol. Men taking thalidomide must agree to use latex condoms every time they have sex with women since it has been shown that thalidomide is found in semen. All patients must agree to participate in the S.T.E.P.S.® program (System for Thalidomide Education and Prescribing Safety). All patients must be educated under the requirements of the S.T.E.P.S.® program. Patients are required to complete a S.T.E.P.S.® survey and sign and additional consent form indicating that they understand all information provided to them as part of the S.T.E.P.S.® educational counseling.

• Estimated life expectancy must be greater than 2 months.

Exclusion Criteria:

• Pregnant and/ or lactating female

• Personal history of thromboembolic disease

• History of seizure activity

• History of neoplasm except basal cell carcinoma in-situ

• History of severe neuropathies

• Women of child bearing potential

• Inability to comply with the protocol

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Arteriovenous Malformation
• Arteriovenous Malformations
• Congenital Abnormalities
• Gastrointestinal Hemorrhage
• Hemangioma
• Hematochezia
• Hereditary Hemorrhagic Telangiectasia
• Melena
• Telangiectasia, Hereditary Hemorrhagic
• Telangiectasis

Intervention

Drug:
• Thalidomide
Thalidomide 50 - 200 mg once at nightime

Locations

Country	Name	City	State
United States	Medical College of Georgia	Augusta	Georgia
United States	Northport VAMC	Northport	New York
United States	UMass Memorial Medical Center	Worcester	Massachusetts

Sponsors (3)

Lead Sponsor	Collaborator
Northport Veterans Affairs Medical Center	Georgia Regents University, University of Massachusetts, Worcester

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Blood Transfusion requirements		6 months	No
Secondary	Hemoglobin overall complication rate Constipation Neuropathy		6 month	Yes