TAG: Thalidomide Reduces Arteriovenous Malformation Related Gastrointestinal Bleeding

Sponsor

Northport Veterans Affairs Medical Center (U.S. Fed)

Overall Status

Completed

CT.gov ID

NCT00389935

Collaborator

Augusta University (Other), University of Massachusetts, Worcester (Other)

Enrollment

Locations

Arm

Duration (Months)

4.7

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Arteriovenous Malformation Hereditary Hemorrhagic Telangiectasia Hematochezia Melena	Drug: Thalidomide	Phase 2

Detailed 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.

Study Design

Study Type:

Interventional

Actual Enrollment :

14 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Thalidomide Reduces Arteriovenous Malformation Related Gastrointestinal Bleeding

Study Start Date :

Oct 1, 2006

Actual Primary Completion Date :

Jun 1, 2011

Actual Study Completion Date :

Jul 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Treatment	Drug: Thalidomide Thalidomide 50 - 200 mg once at nightime

Arm

Intervention/Treatment

Experimental: Treatment

Drug: Thalidomide

Thalidomide 50 - 200 mg once at nightime

Outcome Measures

Primary Outcome Measures

Blood Transfusion requirements [6 months]

Secondary Outcome Measures

Hemoglobin overall complication rate Constipation Neuropathy [6 month]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Medical College of Georgia	Augusta	Georgia	United States	30912
2	UMass Memorial Medical Center	Worcester	Massachusetts	United States	01655
3	Northport VAMC	Northport	New York	United States	11768