Clinical trial NCT00261391: 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
Overall status: COMPLETED
Phase: PHASE1
Conditions: Vascular Anomalies
Keywords: Arteriovenous Malformation
Lymphatic Malformation
Gorham
Lead sponsor: Boston Children's Hospital
Sponsor class: OTHER
First posted: 2005-12-05
Enrollment: 9
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: History
measure: Physical Examination
measure: Laboratory studies
measure: Vital Signs
measure: EKG
measure: Urine studies
Secondary outcomes:
measure: Individualized. Change in the predetermined measure of the vascular anomaly.
Other outcomes:
Locations:
city: Boston
country: United States
facility: Childrens Hospital
state: Massachusetts
Study officials:
affiliation: Boston Children's Hospital
name: Steven J Fishman, M.D.
role: PRINCIPAL_INVESTIGATOR
References: