The Phase I trial of REC-994, using healthy volunteers, was completed in late 2020. A Phase II trial testing the safety and tolerability of the medicine in patients with CCM enrolled its first patient in March 2022. The Phase 2 trial is currently enrolling and will include 60 patients who will be randomized into three groups: a high-dose group, a low-dose group, and a placebo group. The patient eligibility criteria and sites for the trial are below. Please check clinicaltrials.gov to learn more.
Phase 2 enrollment information
Who can participate? Please scroll down for complete inclusion and exclusion criteria. The Phase 2 trial will enroll 60 adults with symptomatic CCM. Symptoms must be caused by an active brain lesion and can include seizure, headache, neurological deficits, or other issues that are not solely from a prior hemorrhage or surgery. REC-994 is meant to address symptoms that can be reduced.
What is involved? This trial involves 7 visits to the study site over a 15 month period. Three of these visits will include MRI, but gadolinium is not used in this study. In addition to taking the medicine, you will also use an app to record headaches and seizures daily.
Where are the study sites and who should I contact? There will be at least 12 sites around the US once all are up and running. We will list the new sites here when they come online.
CURRENTLY ENROLLING SITES
Xenoscience/21st Century Neurology
Principal Investigator: Dr. Stephen Flitman
Clinical Research Coordinator
(602) 274-9500 ext 406
Principal Investigator: Dr. Ricardo Hanel
University of Virginia
Principal Investigator: Dr. Ryan Kellogg
Judy Beenhakker, MS, CCRC
SITES OPENING SOON
University of Pennsylvania
Principal Investigator: Jan Karl Burkhardt
Contact: Leah Coghlan NCRDNeuroICU@uphs.upenn.edu
The University of Texas Southwestern Medical Center
Principal Investigator: Dr. Jonathan White
Contact: Tash Mupambo Tashinga.Mupambo@UTSouthwestern.edu
ELIGIBILITY CRITERIA FROM Clinicaltrials.gov NCT05085561
Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
- 18 years of age or older with anatomic CCM lesions demonstrated by brain MRI
- Have symptomatic CCM
- Have provided written informed consent to participate in the study
- Have NOT participated in a clinical trial utilizing an investigational agent within 28 days or within 6 half-lives of the investigational drug (whichever is longer) prior to Screening
- Symptoms deemed by the study Investigator to be caused exclusively by irreversible neuronal damage from prior stroke or neurosurgical instrumentation
- History of cranial irradiation or surgical/radio-surgical treatment of the primary symptomatic CCM lesion
- Pregnant or breastfeeding
- Unable or unwilling to participate in MRI assessments (eg, claustrophobia, metal implant, implanted cardiac pacemaker). This trial does not use contrast.
- Liver dysfunction or active liver disease as defined by baseline serum transaminases >2x upper limit of normal (ULN)
- Have severely impaired renal function (eGFR <60ml/min) or active renal disease
- Have had a previous diagnosis of skeletal muscle disorders (myopathy) of any cause or have a baseline creatine kinase level > 5x ULN
- History of alcohol or substance abuse within 1 year prior to screening
- Clinically significant laboratory abnormality
- Have had an intracerebral hemorrhage within 3 months of screening or any brain surgery within 6 months of screening
REC-994 is the first industry-sponsored trial of a medicine developed specifically for CCM. The following webinar offers more information about its unique history.
In this blog post, Recursion’s CEO Chris Gibson shares the origin story of REC-994 beginning in graduate school at the University of Utah.