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

Phase 1

Completed

• Conditions: Vascular Anomalies

• Registration Number: NCT00261391
• Lead Sponsor: Boston Children's Hospital

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2000-10
• Status Verified: 2005-05
• Study First Submitted: 2005-12-02
• Study First Submitted QC: 2005-12-02
• Study First Posted: 2005-12-05
• Study Completion: 2007-10
• Last Update Submitted: 2008-01-08
• Last Update Posted: 2008-01-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
History
Physical Examination
Laboratory studies
Vital Signs
EKG
Urine studies

Secondary Outcome Measures

Name	Time	Method
Individualized. Change in the predetermined measure of the vascular anomaly.

Trial Locations

Locations (1)

Childrens Hospital; 🇺🇸 Boston, Massachusetts, United States