Clinical trial · Interventional
The Symptomatic Cerebral Cavernous Malformation Trial of REC-994
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
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Cerebral Cavernous Malformation | — | UNRESOLVED | — |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Placebo | Drug | — | UNRESOLVED |
| REC-994 | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (3)
- type
- ACTIVE_COMPARATOR
- label
- REC-994 200 mg
- description
- REC-994 200 mg po once daily (QD) (1 200 mg REC-994 tablet, 1 matching placebo tablet)
- interventionNames
- Drug: REC-994
- Drug: Placebo
- type
- ACTIVE_COMPARATOR
- label
- REC-994 400 mg
- description
- REC-994 400 mg po QD (2 200 mg REC-994 tablets)
- interventionNames
- Drug: REC-994
- type
- PLACEBO_COMPARATOR
- label
- Placebo
- description
- Matching Placebo po QD (2 matching placebo tablets)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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)
References
Publications (0)
Data not yet available