Congenital Venous MalformationPhase 2NCT00462462
Systemic and Local Diffusion of Ethanol After Administration of Ethanol 96% Formulated in a Gel and Ethanol 98% Solution by the Percutaneous Route, in Patients With Congenital Venous Malformations:Pharmacokinetic, Pharmacodynamic and Clinical Study.
A Phase 2 study testing Ethanol 96% Gel and Ethanol 98% Solution in people with Congenital Venous Malformation, run by Orfagen at 2 sites in 2 countries.
Trial summary
Absolute ethanol has been used "off-label" as an unmodified formulation (solution) in Congenital Venous Malformations (CVM). Despite its effectiveness, absolute ethanol appears difficult to handle because of its high diffusion capacity outside the CVM and in the blood circulation. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Congenital Venous Malformation
- What is being tested?
- Ethanol 96% Gel and Ethanol 98% Solution
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 2 sitesin 2 countries
- Study dates
- May 2007to Jun 2010
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients of both sexes, of at least 12 years of age,
- For women of childbearing potential, negative pregnancy test at baseline,
- Patients with one clinically and radiologically (MRI) documented lesion diagnosed as CVM (pure or predominant),
- Patients for which an embolosclerotherapy by the percutaneous route is indicated as first line therapy of the test lesion, or for which previous treatments (i.e. surgery, embolosclerotherapy, laser) have been unsuccessful or insufficient,
- Patients with CVM lesional size of at least 12 cm3 (maximum craniocaudal dimension X mean dimension of 3 transverse equispaced measurements X mean dimension of 3 deepness equispaced measurements dimension) at MRI,
- Patients with focal or multifocal CVM lesion, i.e. with one or several well-interconnecting venous spaces and well-defined margins,
- Patients or parents able to follow study instructions and attend study visits,
- Written informed consent from the patients or parents.
Who cannot join
- Patients under 12 years of age,
- Pregnant women, nursing mothers and women of childbearing potential with no reliable contraception from more than 2 months,
- Women of childbearing potential with a positive pregnancy test at baseline,
- Patients with CVM of non venous predominance,
- Patients with CVM that are not reachable by the percutaneous route,
- Patients with extensive superficial skin CVM (i.e. with high risk of skin necrosis),
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- Patients with a test lesion adjacent to major nerves (e.g. facial nerve in the parotid region, intramuscular regions adjacent to major nerves),
- Patients with facial CVM or bone involvement,
- Patients with small CVM lesion (<12 cm3 at MRI),
- Patients requiring more than 1 ml/Kg body weight (b.w.) in USA or more than 0.5 ml/Kg b.w. in France, or more than 30 mL of absolute ethanol to infuse,
- Patients with a known allergy to one of the components of the test products,
- Patients with a suspected allergy to iodinate.ed products,
- Patients with abnormal clotting parameters (platelets, partial thromboplastin, prothrombin time),
- Patients with an active inflammatory episode of the test lesion (i.e. acute or subacute swelling of the test lesion),
- Patients with complex malformations (e.g. Klippel-Trenaunay syndrome, Blue Rubber Bled Nevus syndrome, Muco-cutaneous familial venous malformations, Mafucci's syndrome),
- Patients in which a surgery, laser therapy or embolosclerotherapy of the test lesion has been performed within the last 12 weeks prior to study entry,
- Asthmatic patients who require daily medications,
- Patients with a non treated or non stabilized cardiac disease,
- Patients with a suspected right-left shunt,
- Patients with an intercurrent condition or a concomitant treatment which may interfere with a good conduct or the evaluation parameters of the study,
- Patients who participated in a study within the 12 weeks prior to study entry,
- Patients or parents who are not able or willing to follow the study instructions,
- Patients or parents who refuse to give written informed consent.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
Johns Hopkins Medical Institutions
Baltimore, Maryland, 21287
About this study
Absolute ethanol has been used "off-label" as an unmodified formulation (solution) in Congenital Venous Malformations (CVM). Despite its effectiveness, absolute ethanol appears difficult to handle because of its high diffusion capacity outside the CVM and in the blood circulation. A less diffusible ethanol-based product (ethanol gel) has been developed in order to minimize systemic and local diffusion capacities of ethanol. Therefore, the pharmacokinetic parameters and their clinical and paraclinical outcomes between ethanol gel 96% and absolute ethanol need to be carried out.
FDA Office of Orphan Products Development (FDA OOPD) : Funding source.
- Conditions
- Congenital Venous Malformation
- Being tested
- Ethanol 96% Gel, Ethanol 98% Solution
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ethanol 96% GelDrug
Ethanol 98% SolutionDrug
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Ethanol gel Receives: Drug: Ethanol 96% Gel |
| 2 | Active comparator | Ethanol solution Receives: Drug: Ethanol 98% Solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Systemic Exposure to Ethanol With the Two Test Products: Determination of the Maximum Plasma Concentration (Cmax)
Blood samples were performed, just before infusion, then 5 min, 10 min, 20 min, 40 min, 60 min, 90 min, and 120 min after infusion at the first site, then every 60 min onwards until ethanol levels are found under the detection limit. Cmax was estimated directly from experimental data. If all the ethanol concentrations of a patient was below the limit of quantification of the laboratory (LOQ), Cmax was reported as LOQ/2 for this patient.
Measured: Baseline visit (just before and during test product infusion procedure)
Secondary outcomes (3)
Systemic (Cardiopulmonary, Hematological, Metabolic) and Local Outcome of the Two Test Products.
Measured: Study end
Change in Volume of Congenital Venous Malformation (CVM) From Screening to Study End (Day 112 Visit).
Measured: Screening and study end (Day 112)
Patient Benefit
Measured: study end
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2007 (estimated)
- Study startMay 2007
- Primary completionJun 2010
- Study completionJun 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Congenital Venous MalformationStudies of the same condition, open and under way.
- 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 2
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Orfagen
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- FDA Office of Orphan Products Development
- Responsible party
- Sponsor
- Investigators
- PATRICK DUPUY, MD, Study director, ORFAGEN LABORATORIES (France)SALLY E MITCHELL, MD, Principal investigator, Johns Hopkins Medical Institution (Baltimore, USA)Denis HERBRETEAU, MD, Principal investigator, Hôpital Bretonneau (Tours, France)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00462462
- Sponsor's protocol ID
- L00122 GI 201 (ORF)
- Status last verified
- Jan 2015
- First submitted
- 16 Apr 2007
- Last updated
- 15 Jan 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jan 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Systemic and Local Diffusion of Ethanol After Administration of Ethanol 96% Formulated in a Gel and Ethanol 98% Solution by the Percutaneous Route, in Patients With Congenital Venous Malformations:Pharmacokinetic, Pharmacodynamic and Clinical Study.
- ClinicalTrials.gov ID
- NCT00462462
- Acronym / sponsor ID
- L00122 GI 201 (ORF)
- Conditions
- Congenital Venous Malformation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- OrfagenIndustry (pharma, biotech, devices)
- Collaborators
- FDA Office of Orphan Products Development
- Study start
- May 2007
- Primary completion
- Jun 2010 (actual)
- Study completion
- Jun 2010 (actual)
- First posted
- 19 Apr 2007 (estimated)
- Results
- Posted 12 Dec 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Absolute ethanol has been used "off-label" as an unmodified formulation (solution) in Congenital Venous Malformations (CVM). Despite its effectiveness, absolute ethanol appears difficult to handle because of its high diffusion capacity outside the CVM and in the blood circulation. A less diffusible ethanol-based product (ethanol gel) has been developed in order to minimize systemic and local diffusion capacities of ethanol. Therefore, the pharmacokinetic parameters and their clinical and paraclinical outcomes between ethanol gel 96% and absolute ethanol need to be carried out.
FDA Office of Orphan Products Development (FDA OOPD) : Funding source.
Study design
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Ethanol 96% GelEthanol gel |
| 2 | Active comparator | Drug: Ethanol 98% SolutionEthanol solution |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Patients of both sexes, of at least 12 years of age,
- For women of childbearing potential, negative pregnancy test at baseline,
- Patients with one clinically and radiologically (MRI) documented lesion diagnosed as CVM (pure or predominant),
- Patients for which an embolosclerotherapy by the percutaneous route is indicated as first line therapy of the test lesion, or for which previous treatments (i.e. surgery, embolosclerotherapy, laser) have been unsuccessful or insufficient,
- Patients with CVM lesional size of at least 12 cm3 (maximum craniocaudal dimension X mean dimension of 3 transverse equispaced measurements X mean dimension of 3 deepness equispaced measurements dimension) at MRI,
Key exclusion criteria (23 listed)
- Patients under 12 years of age,
- Pregnant women, nursing mothers and women of childbearing potential with no reliable contraception from more than 2 months,
- Women of childbearing potential with a positive pregnancy test at baseline,
- Patients with CVM of non venous predominance,
- Patients with CVM that are not reachable by the percutaneous route,
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 |
|---|---|---|
| Ethanol 96% Gel | Drug | 1 |
| Ethanol 98% Solution | Drug | 2 |
Primary outcome
Systemic Exposure to Ethanol With the Two Test Products: Determination of the Maximum Plasma Concentration (Cmax)
Time frame: Baseline visit (just before and during test product infusion procedure)
Blood samples were performed, just before infusion, then 5 min, 10 min, 20 min, 40 min, 60 min, 90 min, and 120 min after infusion at the first site, then every 60 min onwards until ethanol levels are found under the detection limit. Cmax was estimated directly from experimental data. If all the ethanol concentrations of a patient was below the limit of quantification of the laboratory (LOQ), Cmax was reported as LOQ/2 for this patient.
3 secondary outcomes
Systemic (Cardiopulmonary, Hematological, Metabolic) and Local Outcome of the Two Test Products.
Time frame: Study end
Change in Volume of Congenital Venous Malformation (CVM) From Screening to Study End (Day 112 Visit).
Time frame: Screening and study end (Day 112)
Patient Benefit
Time frame: study end
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
PATRICK DUPUY, MD study director
ORFAGEN LABORATORIES (France)
SALLY E MITCHELL, MD principal investigator
Johns Hopkins Medical Institution (Baltimore, USA)
Denis HERBRETEAU, MD principal investigator
Hôpital Bretonneau (Tours, France)
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ethanol 96% Gel and Ethanol 98% Solution in people with Congenital Venous Malformation, run by Orfagen at 2 sites in 2 countries.
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 8 inclusion and 23 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 15 Jan 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, France. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Dec 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Systemic Exposure to Ethanol With the Two Test Products: Determination of the Maximum Plasma Concentration (Cmax) (time frame: Baseline visit (just before and during test product infusion procedure)). 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 15 Jan 2015.