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CMP-002

Phase 1/2

Nervous System Disease | RNA-Based Therapies | Neurology |CAMP4 Therapeutics Corporation|Trials Updated: Nov 13, 2026

CMP-002 development status

Highest phase Phase 1/2
Registered trials 1 across 1 sponsor since Nov 2026

CMP-002 target and mechanism

Molecular targetSYNGAP1
Target classGene
ModalityRNA-Based Therapies

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindSHAM_CONTROLLED
Total Trials1
Total Enrollment80

FDA Designations

No designations recorded

CMP-002 clinical trials

CMP-002 · 1 trial · 1 indication

Phase 1/2 1
NCT07935135Study of CMP-002 in Participants With SYNGAP1-Related DisorderNervous System Disease
NOT YET_RECRUITING80 Analytics
PHASE1/PHASE2NOT YET_RECRUITING
Study of CMP-002 in Participants With SYNGAP1-Related Disorder
Nervous System Disease
NCT0793513580
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Study Endpoints

Primary Endpoints

Safety and tolerability of multiple doses of CMP-002: incidence of adverse events and changes from baseline in clinical laboratory results
Screening to Week 36

Secondary Endpoints

CSF concentrations of the active drug substance of CMP-002 after multiple doses
Day 1 to Week 36
Plasma PK of the active drug substance of CMP-002 after multiple doses: Cmax
Day 1 to Day 85
Plasma PK of the active drug substance of CMP-002 after multiple doses: AUC(0-24h)
Day 1 to Day 85
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
CMP-002 Multiple Ascending Dose (MAD)EXPERIMENTALParticipants will be administered CMP-002 on Days 1, 29, and 85, and will be followed for 24 weeks after the last dose. Three dose levels will be evaluated.
Sham-prickSHAM_COMPARATORParticipants will receive the sham-prick procedure on Days 1, 29, and 85, and will be followed for 24 weeks after the last procedure.

Interventions

NameTypeDescription
CMP-002DRUGCMP-002 is an antisense oligonucleotide that will be administered by IT injection.
Sham-prickOTHERThe sham-prick procedure involves no injection/puncture or penetration of the dura, but only a skin prick with a needle.
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Eligibility Criteria

Age Range2 Years to 18 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Written informed consent and/or assent, according to local guidelines * Participant is ≥2 and \<18 years of age at the time of informed consent/assent * Participants must have a clinical diagnosis of a SYNGAP1-related disorder. For entry into the treatment period, genetic conf...

Countries:Argentina
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Recent Changes (Last 90 Days)

LOWNov 13, 2026NCT07935135NEW_TRIAL: changed
LOWNov 13, 2026NCT07935135NEW_TRIAL: changed

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