Vascular Anomalies
Conditions
Keywords
Arteriovenous Malformation, Lymphatic Malformation, Gorham
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.
Interventions
DRUGMarimastat
Sponsors
Boston Children's Hospital
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Laboratory studies | — |
| History | — |
| Physical Examination | — |
| Urine studies | — |
| Vital Signs | — |
| EKG | — |
Secondary
| Measure | Time frame |
|---|---|
| Individualized. Change in the predetermined measure of the vascular anomaly. | — |
Countries
United States
Outcome results
None listed