The Symptomatic Cerebral Cavernous Malformation Trial of REC-994

NCT05085561 | Completed Phase 2 FDA Drug

• 1 other identifier: REC-994-201
• Study Type: interventional
• Target: 62
• Locations: 1 country
• Sites: 15

Brief Summary

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).

Conditions

Cerebral Cavernous Malformation

Keywords

Cerebral Cavernous Malformation; CCM

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of adverse events (AEs)

  Up to 24 months

Secondary Outcomes (5)

• Change in patient reported outcomes (Cerebral Cavernous Malformation Health Index)

  Up to 24 months

• Change in patient reported outcomes (Modified Rankin Scale)

  Up to 24 months

• Change in patient reported outcomes (SymptoMScreen Score)

  Up to 24 months

• Change in disease-associated symptoms (number of MRI-confirmed cerebral hemorrhagic events)

  Up to 24 months

• Plasma concentrations of REC-994

  Up to 12 months

Other Outcomes (1)

• Change in disease-associated symptoms (size and number of lesions on MRI)

  Up to 24 months

Study Arms (3)

REC-994 200 mg

ACTIVE COMPARATOR

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

Drug: REC-994; Drug: Placebo

REC-994 400 mg

ACTIVE COMPARATOR

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

Drug: REC-994

Placebo

PLACEBO COMPARATOR

Matching Placebo po QD (2 matching placebo tablets)

Drug: Placebo

Interventions

REC-994 DRUG

REC-994 200 mg tablets

REC-994 200 mg; REC-994 400 mg

Placebo DRUG

Placebo Tablets

Placebo; REC-994 200 mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 5 half-lives of the investigational drug (whichever is longer) prior to Screening

You may not qualify if:

• 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/radiosurgical treatment of the primary symptomatic CCM lesion
• Pregnant or breast feeding
• Be unable or unwilling to participate in MRI assessments (e.g., claustrophobia, metal implant or implanted cardiac pacemaker)
• Liver dysfunction or active liver disease as defined by baseline serum transaminases \>2x upper limit of normal (ULN)
• 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
• 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 (not including the primary symptomatic CCM lesion)

Sponsors & Collaborators

• Recursion Pharmaceuticals Inc.: lead

Study Sites (15)

Xenoscience Inc

Phoenix, Arizona, 85004, United States

Location

UCLA

Los Angeles, California, 90095, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

University of Florida

Gainesville, Florida, 32608, United States

Location

Lyerly Neurosurgery

Jacksonville, Florida, 32207-8202, United States

Location

Cleveland Clinic Florida

Port Saint Lucie, Florida, 34987, United States

Location

Emory

Atlanta, Georgia, 30322, United States

Location

Valley Hospital

Ridgewood, New Jersey, 07450, United States

Location

Columbia University Medical Center

New York, New York, 10027, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15260, United States

Location

The University of Texas Southwestern Medical Center

Dallas, Texas, 75390-8855, United States

Location

University of Virginia

Charlottesville, Virginia, 22908, United States

Location

MeSH Terms

Conditions

Hemangioma, Cavernous, Central Nervous System

Condition Hierarchy (Ancestors)

Hemangioma, Cavernous; Hemangioma; Neoplasms, Vascular Tissue; Neoplasms by Histologic Type; Neoplasms; Cavernous Sinus Syndromes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Central Nervous System Vascular Malformations; Nervous System Malformations; Vascular Malformations; Cardiovascular Abnormalities; Cardiovascular Diseases; Hemostatic Disorders; Vascular Diseases; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 8, 2021
• First Posted: October 20, 2021
• Study Start: March 17, 2022
• Primary Completion: June 30, 2025
• Study Completion: June 30, 2025
• Last Updated: August 6, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (15)