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

Vascular Anomalies Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00261391
• Other study ID #: 03-04-052R
• Status: Completed
• Phase: Phase 1
• First received: December 2, 2005
• Last updated: January 8, 2008
• Start date: October 2000
• Est. completion date: October 2007

Study information

• Verified date: May 2005
• Source: Children's Hospital Boston
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: October 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years and older

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Congenital Abnormalities
• Vascular Anomalies
• Vascular Malformations

Intervention

Drug:
• Marimastat

Locations

Country	Name	City	State
United States	Childrens Hospital	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital Boston

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	History
Primary	Physical Examination
Primary	Laboratory studies
Primary	Vital Signs
Primary	EKG
Primary	Urine studies
Secondary	Individualized. Change in the predetermined measure of the vascular anomaly.