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The Symptomatic Cerebral Cavernous Malformation Trial of REC-994 (SYCAMORE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05085561
Recruitment Status : Active, not recruiting
First Posted : October 20, 2021
Last Update Posted : July 20, 2023
Sponsor:
Information provided by (Responsible Party):
Recursion Pharmaceuticals Inc.

Study Description
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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 subjects with symptomatic cerebral cavernous malformation (CCM).

Condition or disease Intervention/treatment Phase
Cerebral Cavernous Malformation Drug: REC-994 Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 62 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Two-Part Study of REC-994 in the Treatment of Adults With Symptomatic Cerebral Cavernous Malformation (CCM); Part 1: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety, Efficacy and Pharmacokinetics of Two Doses of REC-994; Part 2: A Long-Term Blinded Extension Clinical Trial to Evaluate Long-Term Safety Tolerability and Efficacy of REC-994
Actual Study Start Date : March 17, 2022
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Active Comparator: REC-994 200 mg
REC-994 200 mg po once daily (QD) (1 200 mg REC-994 tablet, 1 matching placebo tablet)
 Drug: REC-994
REC-994 200 mg tablets

Drug: Placebo
Placebo Tablets
Active Comparator: REC-994 400 mg
REC-994 400 mg po QD (2 200 mg REC-994 tablets)
 Drug: REC-994
REC-994 200 mg tablets
Placebo Comparator: Placebo
Matching Placebo po QD (2 matching placebo tablets)
 Drug: Placebo
Placebo Tablets


Outcome Measures
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Primary Outcome Measures :
  1. Incidence and severity of adverse events (AEs) [ Time Frame: 24 months ]
    Safety and tolerability

  2. Incidence of clinically significant changes in physical examinations [ Time Frame: 12 months (Part 2) ]
    Safety and tolerability


Secondary Outcome Measures :
  1. Change in patient reported outcomes (Cerebral Cavernous Malformation Health Index) [ Time Frame: 24 months ]
    Efficacy

  2. Change in patient reported outcomes (Modified Rankin Scale) [ Time Frame: 24 months ]
    Efficacy

  3. Change in patient reported outcomes (SymptoMScreen Score) [ Time Frame: 24 months ]
    Efficacy

  4. Change in disease-associated symptoms (size and number of lesions on MRI) [ Time Frame: 24 months ]
    Efficacy

  5. Change in disease-associated symptoms (number of MRI-confirmed cerebral hemorrhagic events) [ Time Frame: 24 months ]
    Efficacy

  6. Incidence of clinically significant changes in physical examinations [ Time Frame: 12 months (Part 1) ]
    Safety and tolerability


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05085561


Locations
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United States, Arizona
Xenoscience Inc
Phoenix, Arizona, United States, 85004
United States, California
UCLA
Los Angeles, California, United States, 90095
Stanford University
Palo Alto, California, United States, 94304
United States, Florida
University of Florida
Gainesville, Florida, United States, 32608
Lyerly Neurosurgery
Jacksonville, Florida, United States, 32207-8202
Cleveland Clinic Florida
Port Saint Lucie, Florida, United States, 34987
United States, Georgia
Emory
Atlanta, Georgia, United States, 30322
United States, New Jersey
Valley Hospital
Ridgewood, New Jersey, United States, 07450
United States, New York
Columbia University Medical Center
New York, New York, United States, 10027
University of Rochester Medical Center
Rochester, New York, United States, 14642
United States, Pennsylvania
Thomas Jefferson University
Philadelphia, Pennsylvania, United States, 19104
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15260
United States, Texas
The University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390-8855
United States, Virginia
University of Virginia
Charlottesville, Virginia, United States, 22908
Sponsors and Collaborators
Recursion Pharmaceuticals Inc.
More Information
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Responsible Party: Recursion Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT05085561    
Other Study ID Numbers: REC-994-201
First Posted: October 20, 2021    Key Record Dates
Last Update Posted: July 20, 2023
Last Verified: July 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemangioma, Cavernous, Central Nervous System
Congenital Abnormalities
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