search
Back to results

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

Primary Purpose

Vascular Anomalies

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Marimastat
Sponsored by
Boston Children's Hospital
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Vascular Anomalies focused on measuring Arteriovenous Malformation, Lymphatic Malformation, Gorham

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Childrens Hospital

Outcomes

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.

Full Information

First Posted
December 2, 2005
Last Updated
January 8, 2008
Sponsor
Boston Children's Hospital
search

1. Study Identification

Unique Protocol Identification Number
NCT00261391
Brief Title
Phase I Drug Trial for S/E of Marimastat in Disabling Malformations When no Other Options.
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 Type
Interventional

2. Study Status

Record Verification Date
May 2005
Overall Recruitment Status
Completed
Study Start Date
October 2000 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
October 2007 (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Boston Children's Hospital

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Vascular Anomalies
Keywords
Arteriovenous Malformation, Lymphatic Malformation, Gorham

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
9 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Marimastat
Primary Outcome Measure Information:
Title
History
Title
Physical Examination
Title
Laboratory studies
Title
Vital Signs
Title
EKG
Title
Urine studies
Secondary Outcome Measure Information:
Title
Individualized. Change in the predetermined measure of the vascular anomaly.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Steven J Fishman, M.D.
Organizational Affiliation
Boston Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Childrens Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs