Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UCLA
Dates
study started
completion around

Description

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).

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

Keywords

Cerebral Cavernous Malformation, Hemangioma, Cavernous, Central Nervous System, Congenital Abnormalities, REC-994, REC-994 200 mg, REC-994 400 mg

Eligibility

You can join if…

Open to people ages 18 years and up

  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

You CAN'T join if...

  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)

Locations

  • UCLA
    Los Angeles California 90095 United States
  • Stanford University
    Palo Alto California 94304 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Recursion Pharmaceuticals Inc.
ID
NCT05085561
Phase
Phase 2 Cerebral Cavernous Malformations Research Study
Study Type
Interventional
Participants
About 62 people participating
Last Updated