Phase I Drug Trial for S/E of Marimastat in Disabling Malformations When no Other Options.

NCT ID: NCT00261391

Last Updated: 2008-01-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2000-10-31
• Study Completion Date: 2007-10-31

Brief Summary

3 patients were enrolled in each of 3 study cohorts. There three cohorts were given differing, incrementally larger doses of this phase I drug. The same safety measures are being obtained on all patients. Efficacy measures were individualized as enrolllees do not have the same underlying vascular anomaly. The study is structured to include a 24 month drug-phase and a 24 month follow-up phase. The study is now closed to enrollment.

Conditions

Vascular Anomalies

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Interventions

Marimastat

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Patient with vascular malformation causing risk of one or more of the following based on unanimous assessment of designated physicians in the multidisciplinary Vascular Anomalies Team.

• airway/respiratory/visual/auditory/neurologic compromise;
• high output cardiac failure;
• life-threatening or disabling hemorrhage(cutaneous/GI/intracranial/ parenchymal/cavitary);
• skeletal distortion/destruction/erosion;
• life-threatening or disabling soft tissue distortion or destruction
• Patient must be felt to have failed, be unable to significantly benefit from, or be at risk for other available therapies, including surgeries, embolization, and sclerotherapy based on unanimous assessment of designated physicians in the multidisciplinary Vascular Anomalies Team.
• Patient must be felt to have one or more physical, imaging, photographic, physiologic or other measurable features that can be measured on a regular basis for preliminary evaluation of efficacy. The feature(s) must be agreed on by the designated physicians in the multidisciplinary Vascular Anomalies Team).
• Signed Patient informed consent.

Exclusion Criteria

• Pregnancy
• Patient nursing child.
• Female patient of childbearing potential unwilling to receive contraceptive counseling and use reliable contraceptive method.
• Patient enrolled in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team)
• Parent or guardian or child unwilling to provide consent or assent.

• Minimum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Boston Children's Hospital

OTHER

Sponsor Role: lead

Principal Investigators

Steven J Fishman, M.D.

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Locations

Childrens Hospital

Boston, Massachusetts, United States

Countries

United States

Other Identifiers

03-04-052R

Identifier Type: -

Identifier Source: org_study_id