Vascular Ehlers-Danlos Syndrome Clinical Trial
— PREVEntOfficial title:
A Multicenter, Randomized, Double-blind, Placebo-controlled Study of Enzastaurin for the Prevention of Arterial Events in Patients With Vascular Ehlers-Danlos Syndrome (vEDS) Confirmed With COL3A1 Mutations, Followed by an Open Label Extension (OLE)
Verified date | March 2024 |
Source | Aytu BioPharma, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The purpose of this study is to investigate the efficacy of enzastaurin compared to placebo in preventing arterial events (rupture, dissection, pseudoaneurysm, carotid-cavernous sinus fistula, or aneurysm, fatal or not) leading to intervention or mortality attributable to an arterial event in patients with vEDS confirmed with pathogenic heterozygous COL3A1 gene mutations predicted to derive a mutant protein.
Status | Suspended |
Enrollment | 260 |
Est. completion date | March 2027 |
Est. primary completion date | June 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 60 Years |
Eligibility | Inclusion Criteria: 1. Subjects aged 18 - 60 years old at time of initial screening. 2. Adolescent subjects aged 12 - 17 years old, may be considered to enroll later in the clinical trial pending interim analysis. 3. Diagnosis for VEDS (vascular Ehlers Danlos Syndrome), with a confirmed and documented COL3A1 genetic variant. 4. Subject should be stable, having no VEDS-related vascular events within the past 3 months prior to enrollment. 5. Confirmed use of contraception for both male and female participants. Exclusion Criteria: 1. Inability to swallow or receive intact tablets. 2. Currently being treated with CYP3A4 inhibitors within 4 weeks prior to enrollment. 3. Known allergy or hypersensitivity to enzastaurin. 4. Patient currently pregnant or breast feeding. Other criteria will be reviewed at the first study visit to determine if you are able to participate in the study. |
Country | Name | City | State |
---|---|---|---|
United States | Aytu BioPharma | Englewood | Colorado |
Lead Sponsor | Collaborator |
---|---|
Aytu BioPharma, Inc. | Parexel |
United States,
Bowen CJ, Calderon Giadrosic JF, Burger Z, Rykiel G, Davis EC, Helmers MR, Benke K, Gallo MacFarlane E, Dietz HC. Targetable cellular signaling events mediate vascular pathology in vascular Ehlers-Danlos syndrome. J Clin Invest. 2020 Feb 3;130(2):686-698. doi: 10.1172/JCI130730. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Time to intervention for an arterial event (rupture, dissection, pseudoaneurysm, carotid-cavernous sinus fistula, or aneurysm, fatal or not) or mortality attributable to an arterial event. | Time to intervention for an arterial event (rupture, dissection, pseudoaneurysm, carotid-cavernous sinus fistula, or aneurysm, fatal or not) or mortality attributable to an arterial event, as adjudicated by an Event Committee and analyzed for difference in the time-to-composite-event of active vs. placebo treatments, using survival analysis until end of study | 30 months | |
Secondary | Number of and proportion of patients with adverse events, or with abnormal vital signs, physical examinations, ophthalmological examinations, clinical laboratory values, or electrocardiograms (ECGs) medical attention. | An Adverse Event (AE) is defined as any untoward medical occurrence associated with the use of the investigational product in humans, whether or not considered related to investigational product. An AE can be any unfavorable and unintended sign (e.g., an abnormal laboratory finding), symptom, or disease temporally associated with any use of the investigational product, without any judgment about causality and irrespective of route of administration, formulation, or dose, including an overdose. | 30 months | |
Secondary | Number of and proportion of patients who discontinue study drug due to adverse events | Discontinuation or withdrawal from the study | 30 months |
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