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

Phase I Dose Escalation Study to Evaluate the Safety and Preliminary Efficacy of Marimastat in Patients With Disabling Malformations and No Other Treatment 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.

Study Overview

• Status: Completed
• Conditions: Vascular Anomalies
• Intervention / Treatment: Drug: Marimastat

• Study Type: Interventional
• Enrollment: 9
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Childrens Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
History
Physical Examination
Laboratory studies
Vital Signs
EKG
Urine studies

Secondary Outcome Measures

Outcome Measure
Individualized. Change in the predetermined measure of the vascular anomaly.

Collaborators and Investigators

• Sponsor: Boston Children's Hospital
• Investigators: Principal Investigator: Steven J Fishman, M.D., Boston Children's Hospital

Study record dates

Study Major Dates

• Study Start: October 1, 2000
• Primary Completion: December 7, 2022
• Study Completion: October 1, 2007

Study Registration Dates

• First Submitted: December 2, 2005
• First Submitted That Met QC Criteria: December 2, 2005
• First Posted (Estimate): December 5, 2005

Study Record Updates

• Last Update Posted (Estimate): January 9, 2008
• Last Update Submitted That Met QC Criteria: January 8, 2008
• Last Verified: May 1, 2005

More Information

Terms related to this study

• Keywords: Arteriovenous Malformation; Lymphatic Malformation; Gorham
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Cardiovascular Abnormalities; Congenital Abnormalities; Vascular Malformations; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Marimastat
• Other Study ID Numbers: 03-04-052R

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No