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