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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.
Sponsor:
Information provided by:
Children's Hospital Boston
ClinicalTrials.gov Identifier:
NCT00261391
First received: December 2, 2005
Last updated: January 8, 2008
Last verified: May 2005
  Purpose

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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 Children's Hospital Boston:

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
  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00261391

Locations
United States, Massachusetts
Childrens Hospital
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Children's Hospital Boston
Investigators
Principal Investigator: Steven J Fishman, M.D. Children's Hospital Boston
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00261391     History of Changes
Other Study ID Numbers: 03-04-052R
Study First Received: December 2, 2005
Last Updated: January 8, 2008
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospital Boston:
Arteriovenous Malformation
Lymphatic Malformation
Gorham

Additional relevant MeSH terms:
Congenital Abnormalities
Vascular Malformations
Cardiovascular Abnormalities
Cardiovascular Diseases
Marimastat
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on November 27, 2014