What side effects are associated with this type of intervention?

The most common treatments for Cerebral Cavernous Malformations (CCM) can include medications and surgical interventions. For medications like REC-994, which is currently under investigation, potential side effects may include headache, dizziness, and gastrointestinal issues. Surgical treatments, although not specified in the context of REC-994, generally carry risks such as infection, bleeding, and neurological deficits. It is important to discuss with a healthcare provider to understand the specific risks associated with any treatment plan.

What prior FDA approvals exist?

Currently, there are no specific FDA-approved treatments for Cerebral Cavernous Malformations (CCM). The management of CCM typically involves symptomatic treatment and surgical interventions when necessary. The REC-994 trial is investigating a new potential treatment, but its mechanism of action is not specified in the provided context. Further research and clinical trials are needed to establish effective pharmacological treatments for CCM.

What other types of research exist?

Promising novel alternatives to REC-994 for Cerebral Cavernous Malformations include: 1) Bevacizumab, an anti-angiogenic agent that has shown efficacy in various neurological conditions, including optic pathway gliomas and vestibular schwannomas. 2) mTORC1 inhibitors like everolimus, which have been effective in treating neurofibromatosis type 2 and meningiomas. 3) Engineered Wnt ligands, which have demonstrated potential in repairing the blood-brain barrier and mitigating neurological disorders.

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REC-994 for Cerebral Cavernous Malformations (SYCAMORE Trial)

Phase 2

Waitlist Available

Research Sponsored by Recursion Pharmaceuticals Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

18 years of age or older with anatomic CCM lesions demonstrated by brain MRI

Have symptomatic CCM

Must not have

Liver dysfunction or active liver disease as defined by baseline serum transaminases >2x upper limit of normal (ULN)

Be unable or unwilling to participate in MRI assessments (e.g., claustrophobia, metal implant or implanted cardiac pacemaker)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months (part 1)

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects for REC-994 aren't listed here, common ones in trials may include headaches, nausea, allergic reactions or other symptoms depending on how it affects individuals differently.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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I have symptoms from cerebral cavernous malformations.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

My liver tests are more than twice the normal limit.

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

Select...

My kidney function is low, I have kidney disease, or I've had a kidney transplant.

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I have had brain radiation or surgery for a primary CCM lesion.

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I have a muscle disorder or my muscle enzyme levels are very high.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months (part 1)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months (part 1)

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months (part 1) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence and severity of adverse events (AEs)

Secondary study objectives

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

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

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

+3 more

Trial Design

3Treatment groups

Active Control

Placebo Group

Group I: REC-994 200 mgActive Control2 Interventions

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

Group II: REC-994 400 mgActive Control1 Intervention

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

Group III: PlaceboPlacebo Group1 Intervention

Matching Placebo po QD (2 matching placebo tablets)

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Cerebral Cavernous Malformations (CCM) include surgical resection, which physically removes the malformations to prevent hemorrhages, and medications to manage symptoms such as seizures or headaches. Investigational drugs, like REC-994, are being studied for their potential to stabilize or reduce the size of the malformations by targeting the underlying molecular pathways involved in CCM formation and progression. Understanding these mechanisms is crucial for CCM patients as it helps in developing targeted therapies that can effectively manage or potentially cure the condition, reducing the risk of complications and improving quality of life.