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Could a new drug tame dangerous brain blood vessel clusters?

NCT ID NCT05085561

Disease control Sponsor: Recursion Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This trial tests an experimental drug called REC-994 in adults with symptomatic cerebral cavernous malformation (CCM), a condition where clusters of abnormal blood vessels in the brain can cause bleeding, seizures, and other symptoms. Participants receive either a low or high dose of REC-994 or a placebo for up to a year, with a long-term extension phase. The study measures safety, symptom changes, and bleeding events to see if the drug can help control the disease.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
REC-994
What this could lead to
If successful, REC-994 could become the first drug treatment to reduce symptoms and bleeding events in people with cerebral cavernous malformation.
What could go wrong
This is a small, early-phase trial. The drug may not prove more effective than placebo, and long-term safety is still unknown.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

62 people

The number who actually took part.

Started

Mar 2022

Finished

Jun 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion criteria: 1. 18 years of age or older with anatomic CCM lesions demonstrated by brain MRI 2. Have symptomatic CCM 3. Have provided written informed consent to participate in the study 4. Have not participated in a clinical trial utilizing an investigational agent within 28 days or within 5 half-lives of the investigational drug (whichever is longer) prior to Screening Exclusion Criteria: 1. Symptoms deemed by the study Investigator to be caused exclusively by irreversible neuronal damage from prior stroke or neurosurgical instrumentation 2. History of cranial irradiation or surgical/radiosurgical treatment of the primary symptomatic CCM lesion 3. Pregnant or breast feeding 4. Be unable or unwilling to participate in MRI assessments (e.g., claustrophobia, metal implant or implanted cardiac pacemaker) 5. Liver dysfunction or active liver disease as defined by baseline serum transaminases \>2x upper limit of normal (ULN) 6. Have moderately or severely impaired renal function (estimated glomerular filtration rate \[eGFR\] \<60ml/min) or active renal disease or have previously received a kidney transplant 7. Have had a previous diagnosis of skeletal muscle disorders (myopathy) of any cause or have a baseline creatine kinase level \> 5x ULN 8. History of alcohol or substance abuse within 1 year prior to screening 9. Clinically significant laboratory abnormality 10. Have had an intracerebral hemorrhage within 3 months of screening or any brain surgery within 6 months of screening (not including the primary symptomatic CCM lesion)

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Conditions

The condition(s) this trial relates to.

cerebral cavernous malformation Hemangioma, Cavernous, Central Nervous System

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cerebral cavernous malformation

Contacts and locations

Locations

  • Cleveland Clinic Florida

    Port Saint Lucie, Florida, 34987, United States

  • Columbia University Medical Center

    New York, New York, 10027, United States

  • Emory

    Atlanta, Georgia, 30322, United States

  • Lyerly Neurosurgery

    Jacksonville, Florida, 32207-8202, United States

  • Stanford University

    Palo Alto, California, 94304, United States

  • The University of Texas Southwestern Medical Center

    Dallas, Texas, 75390-8855, United States

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19104, United States

  • UCLA

    Los Angeles, California, 90095, United States

  • University of Florida

    Gainesville, Florida, 32608, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Pittsburgh

    Pittsburgh, Pennsylvania, 15260, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Virginia

    Charlottesville, Virginia, 22908, United States

  • Valley Hospital

    Ridgewood, New Jersey, 07450, United States

  • Xenoscience Inc

    Phoenix, Arizona, 85004, United States