Phase I Drug Trial for S/E of Marimastat in Disabling Malformations When no Other Options.
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.
|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|
- Physical Examination
- Laboratory studies
- Vital Signs
- Urine studies
- Individualized. Change in the predetermined measure of the vascular anomaly.
|Study Start Date:||October 2000|
|Estimated Study Completion Date:||October 2007|
|United States, Massachusetts|
|Boston, Massachusetts, United States, 02115|
|Principal Investigator:||Steven J Fishman, M.D.||Children's Hospital Boston|