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

This study has been completed.
Information provided by:
Boston Children’s Hospital Identifier:
First received: December 2, 2005
Last updated: January 8, 2008
Last verified: May 2005
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.

Condition Intervention Phase
Vascular Anomalies Drug: Marimastat Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Dose Escalation Study to Evaluate the Safety and Preliminary Efficacy of Marimastat in Patients With Disabling Malformations and No Other Treatment Options

Resource links provided by NLM:

Further study details as provided by Boston Children’s Hospital:

Primary Outcome Measures:
  • History
  • Physical Examination
  • Laboratory studies
  • Vital Signs
  • EKG
  • Urine studies

Secondary Outcome Measures:
  • Individualized. Change in the predetermined measure of the vascular anomaly.

Estimated Enrollment: 9
Study Start Date: October 2000
Estimated Study Completion Date: October 2007

Ages Eligible for Study:   2 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
  Contacts and Locations
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Please refer to this study by its identifier: NCT00261391

United States, Massachusetts
Childrens Hospital
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Boston Children’s Hospital
Principal Investigator: Steven J Fishman, M.D. Boston Children’s Hospital
  More Information Identifier: NCT00261391     History of Changes
Other Study ID Numbers: 03-04-052R
Study First Received: December 2, 2005
Last Updated: January 8, 2008

Keywords provided by Boston Children’s Hospital:
Arteriovenous Malformation
Lymphatic Malformation

Additional relevant MeSH terms:
Congenital Abnormalities
Vascular Malformations
Cardiovascular Abnormalities
Cardiovascular Diseases
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action processed this record on September 21, 2017