REC-994 for Cerebral Cavernous Malformations

(SYCAMORE Trial)

Recruiting at 15 trial locations

Overseen By Recursion Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Recursion Pharmaceuticals Inc.

Disqualifiers: Pregnancy, Liver disease, Renal impairment, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

62 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a medication called REC-994 at two different doses to see if it can help people with a brain condition called cerebral cavernous malformation (CCM). The condition causes abnormal blood vessels in the brain, leading to symptoms like headaches and seizures. The medication might work by making these blood vessels more stable and less likely to cause problems.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

How does the drug REC-994 differ from other treatments for cerebral cavernous malformations?

REC-994 is a novel treatment option for cerebral cavernous malformations, a condition with limited standard treatments. Unlike aspirin and statin therapies, which are used to reduce the risk of hemorrhage, REC-994 is being specifically investigated for its potential to address the underlying condition, offering a new approach to managing this disorder. ^{1 2 3 4 5}

Eligibility Criteria

This trial is for adults with symptomatic cerebral cavernous malformation (CCM) confirmed by MRI. Participants must consent to the study and not have been in another drug trial within the past 28 days or 5 half-lives of any investigational drug. Exclusions include pregnancy, breastfeeding, inability to undergo MRI, liver or severe kidney issues, recent hemorrhage or brain surgery, history of muscle disorders or substance abuse.

Inclusion Criteria

I am over 18 and have CCM lesions in my brain shown by MRI.

Have provided written informed consent to participate in the study

I have symptoms from cerebral cavernous malformations.

Exclusion Criteria

My liver tests are more than twice the normal limit.

I cannot or do not want to undergo MRI scans due to claustrophobia or having a metal implant/pacemaker.

My symptoms are due to permanent nerve damage from a past stroke or brain surgery.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive REC-994 or placebo in a double-blind manner to evaluate safety, efficacy, and pharmacokinetics

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-Term Blinded Extension

Participants continue to receive REC-994 or placebo to evaluate long-term safety, tolerability, and efficacy

Long-term

Treatment Details

Interventions

• Placebo
• REC-994

Trial Overview The study tests REC-994 at two doses (200 mg and 400 mg) against a placebo in people with CCM. It's randomized and double-blind, meaning participants are put into groups by chance and neither they nor the researchers know who gets the real treatment versus placebo.

Participant Groups

Group I: REC-994 200 mg Active Control 2 Interventions

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

Group II: REC-994 400 mg Active Control 1 Intervention

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

Group III: Placebo Placebo Group 1 Intervention

Matching Placebo po QD (2 matching placebo tablets)

Who Is Running the Clinical Trial?

Recursion Pharmaceuticals Inc.

Lead Sponsor

Trials

6

Recruited

450+

References

1. United States pubmed.ncbi.nlm.nih.gov

Solitary Sporadic Cerebral Cavernous Malformations: Risk Factors of First or Recurrent Symptomatic Hemorrhage and Associated Functional Impairment. [2017]

2. United States pubmed.ncbi.nlm.nih.gov

Baseline Characteristics of Patients With Cavernous Angiomas With Symptomatic Hemorrhage in Multisite Trial Readiness Project. [2022]

3. Scotland pubmed.ncbi.nlm.nih.gov

Role of aspirin and statin therapy in patients with cerebral cavernous malformations. [2020]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Pediatric cavernous malformation in the central nervous system: report of 66 cases. [2009]

5. United States pubmed.ncbi.nlm.nih.gov

Clinical outcomes of pediatric cerebral cavernous malformation: an analysis of 124 consecutive cases. [2022]