Vascular AnomaliesPhase 1NCT00261391
Phase I Drug Trial for S/E of Marimastat in Disabling Malformations When no Other Options.
A Phase 1 study testing Marimastat in people with Vascular Anomalies, run by Boston Children's Hospital at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Vascular Anomalies
- What is being tested?
- Marimastat
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2000to Oct 2007
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
- Conditions
- Vascular Anomalies
- Being tested
- Marimastat
- Keywords
- Arteriovenous Malformation, Lymphatic Malformation, Gorham
- Medical subject headings
- Vascular Malformations, Arteriovenous Malformations, Lymphatic Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MarimastatDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
History
Physical Examination
Laboratory studies
Vital Signs
EKG
Urine studies
Secondary outcomes (1)
Individualized. Change in the predetermined measure of the vascular anomaly.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2005 (estimated)
- Study startOct 2000
- Study completionOct 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Vascular AnomaliesStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Investigators
- Steven J Fishman, M.D., Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00261391
- Sponsor's protocol ID
- 03-04-052R
- Status last verified
- May 2005
- First submitted
- 2 Dec 2005
- Last updated
- 9 Jan 2008
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00261391
- Acronym / sponsor ID
- 03-04-052R
- Conditions
- Vascular Anomalies
- Keywords
- Arteriovenous Malformation, Lymphatic Malformation, Gorham
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Study start
- Oct 2000
- Study completion
- Oct 2007
- First posted
- 5 Dec 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Marimastat | Drug | – |
Primary outcome
History
Physical Examination
Laboratory studies
Vital Signs
EKG
Urine studies
1 secondary outcome
Individualized. Change in the predetermined measure of the vascular anomaly.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jan 2008.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven J Fishman, M.D. principal investigator
Boston Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Marimastat in people with Vascular Anomalies, run by Boston Children's Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Jan 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
History. The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jan 2008.