Enrolling | Clinical Trial | Single-Decentralized Trial | Zevra Therapeutics
A Phase 3 Randomized, Double-Blind, Decentralized Clinical Trial To Compare The Efficacy Of Celiprolol To Placebo In The Treatment Of Patients With Col3a1-Positive Vascular Ehlers-Danlos Syndrome
Duration of treatment:
The duration of the study will be based upon reaching the required number of vEDS-related events requiring medical attention. An interim analysis will occur after 28 confirmed vEDS-related events have accrued, which based on the BBEST study (Ong 2010) is expected to occur around Month 24. If statistical significance is reached, as determined by crossing the interim group sequential efficacy boundary, the DB portion of the study will be completed and final study visits will be scheduled.
Primary Objective:
To determine whether celiprolol reduces the occurrence of vascular Ehlers-Danlos Syndrome(vEDS)-related clinical events requiring medical attention (fatal/nonfatal cardiac or arterial events[including dissection or rupture], uterine rupture, intestinal rupture, and/or unexplained sudden death), relative to placebo as measured by time to event.
Recruitment is open to patients within the US– patients must meet the following inclusion criteria to be eligible for study participation:
1. Written Informed Consent or econsent must be signed voluntarily before any study-related procedure is performed, indicating that the patient is able to understand and comply with the requirements of the study. For patients <18 years of age a parental consent will be required.
2. Willingness to obtain magnetic resonance angiogram (MRA) image at local imaging facility.
3. A genetic test confirming the presence of a pathogenic COL3A1 variant (classified as likely pathogenic or pathogenic according to ACMG/AMP Guidelines [Richards 2015]).
4. Patients must be ≥ 15 years of age at the time of randomization.
5. Able and willing to discontinue use of β-blockers prior to randomization. Be considered medically fit by the treating physician for study participation and be off the β-blocker for a minimum of 10 days.
Sponsor: Zevra Therapeutics
PI name(s): Sheila Woodhouse, M.D.
