The Symptomatic Cerebral Cavernous Malformation Trial of REC-994

NCT ID: NCT05085561

Last Updated: 2025-08-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

62 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-03-17

Study Completion Date

2025-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This is a two-part, multi-center, randomized, double-blind, placebo-controlled study to investigate the safety, efficacy and pharmacokinetics of REC-994 (200 mg and 400 mg) compared to placebo in participants with symptomatic cerebral cavernous malformation (CCM).

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Part 1: Participants will receive treatment over a period of 12 months. Part 2: Optional long-term extension (LTE) for participants completing Part 1 and who are eligible for extended treatment with REC-994.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cerebral Cavernous Malformation

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

REC-994 200 mg

REC-994 200 mg po once daily (QD) (1 200 mg REC-994 tablet, 1 matching placebo tablet)

Group Type ACTIVE_COMPARATOR

REC-994

Intervention Type DRUG

REC-994 200 mg tablets

Placebo

Intervention Type DRUG

Placebo Tablets

REC-994 400 mg

REC-994 400 mg po QD (2 200 mg REC-994 tablets)

Group Type ACTIVE_COMPARATOR

REC-994

Intervention Type DRUG

REC-994 200 mg tablets

Placebo

Matching Placebo po QD (2 matching placebo tablets)

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo Tablets

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

REC-994

REC-994 200 mg tablets

Intervention Type DRUG

Placebo

Placebo Tablets

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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)
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Recursion Pharmaceuticals Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Xenoscience Inc

Phoenix, Arizona, United States

Site Status

UCLA

Los Angeles, California, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

Lyerly Neurosurgery

Jacksonville, Florida, United States

Site Status

Cleveland Clinic Florida

Port Saint Lucie, Florida, United States

Site Status

Emory

Atlanta, Georgia, United States

Site Status

Valley Hospital

Ridgewood, New Jersey, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

University of Rochester Medical Center

Rochester, New York, United States

Site Status

Thomas Jefferson University

Philadelphia, Pennsylvania, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

The University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

University of Virginia

Charlottesville, Virginia, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

REC-994-201

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A
NCT03520751 NOT_YET_RECRUITING PHASE1/PHASE2
Miglustat in Niemann-Pick Type C Disease
NCT00517153 COMPLETED PHASE2