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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00261391
Recruitment Status : Completed
First Posted : December 5, 2005
Last Update Posted : January 9, 2008
Information provided by:
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.

Condition or disease Intervention/treatment Phase
Vascular Anomalies Drug: Marimastat Phase 1

Study Type : Interventional  (Clinical Trial)
Enrollment : 9 participants
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
Study Start Date : October 2000
Study Completion Date : October 2007

Primary Outcome Measures :
  1. History
  2. Physical Examination
  3. Laboratory studies
  4. Vital Signs
  5. EKG
  6. Urine studies

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

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): 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 Identifier: NCT00261391     History of Changes
Other Study ID Numbers: 03-04-052R
First Posted: December 5, 2005    Key Record Dates
Last Update Posted: January 9, 2008
Last Verified: May 2005

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