VEDS Clinical Trial - Celiprolol

Zevra Therapeutics plans to conduct a clinical trial in the U.S. to evaluate the efficacy of a medicine known as celiprolol for the treatment of vascular Ehlers-Danlos Syndrome (vEDS).

Objective

The clinical study intends to enrovl 150 COL3A1 positive vEDS patients. 50 patients will receive placebo and 100 patients will receive celiprolol up to 400 mg/day (200mg bid).

The study will conclude when 46 qualifying events (fatal or non-fatal cardiac event such as rupture of dissection, uterine rupture, colon rupture and/or unexplained sudden death) have been reported.

About Celiprolol

Celiprolol is an adrenoceptor modulator believed to decrease mechanical stress on the vascular wall of large arteries and hollow organs such as the uterus. It is an investigational medication for the treatment of vascular Ehlers Danlos Syndrome (vEDS).

Inclusion Criteria

To pre-qualify for this clinical research study, you must meet the following criteria:

• Must be greater than or equal to 15 years old
• Willing to obtain a magnetic resonance angiogram (MRA) image
• During screening, must have a genetic test performed, via a saliva sample, to confirm the presence of the COL3A1 mutation which will confirm a vEDS diagnosis; you will need a positive test result
• Must be able and willing to discontinue use of beta blockers under the care of your primary physician
• Should not have had an arterial rupture or dissection, uterine rupture, and/or intestinal rupture within the last 6 months
• Must be willing to complete all study procedures

Please note this is a summary of the main criteria to assess pre-eligibility and is not an exhaustive list