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

Study Overview
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Brief Summary
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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.

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

Conditions
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Vascular Anomalies

Intervention / Treatment
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* Drug: Marimastat


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 2 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Marimastat|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| History |  |  | 
| Physical Examination |  |  | 
| Laboratory studies |  |  | 
| Vital Signs |  |  | 
| EKG |  |  | 
| Urine studies |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Individualized. Change in the predetermined measure of the vascular anomaly. |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Arteriovenous Malformation, Lymphatic Malformation, Gorham