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

NCT00261391 | Completed Phase 1

• 1 other identifier: 03-04-052R
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

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

Keywords

Arteriovenous Malformation; Lymphatic Malformation; Gorham

Outcome Measures

Primary Outcomes (6)

• History

• Physical Examination

• Laboratory studies

• Vital Signs

• EKG

• Urine studies

Secondary Outcomes (1)

• Individualized. Change in the predetermined measure of the vascular anomaly.

Interventions

Marimastat DRUG

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Boston Children's Hospital: lead

Study Sites (1)

Childrens Hospital

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Vascular Malformations; Arteriovenous Malformations; Lymphatic Abnormalities

Interventions

marimastat

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Vascular Diseases; Lymphatic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Steven J Fishman, M.D.

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: December 2, 2005
• First Posted: December 5, 2005
• Study Start: October 1, 2000
• Study Completion: October 1, 2007
• Last Updated: January 9, 2008

Record last verified: 2005-05

Locations

US (1)