Study of CMP-002 in Participants With SYNGAP1-Related Disorder (ASCEND)

November 6, 2026 updated by: CAMP4 Therapeutics Corporation

A Double-blind, Randomized, Controlled, Multiple Ascending Dose Study of Intrathecally Administered CMP-002 in Participants With SYNGAP1-Related Disorder

The primary purpose of the study is to evaluate the safety, tolerability, and pharmacokinetics of CMP-002 in participants with SYNGAP1-related disorder.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Not yet recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

ASCEND is a global, Phase 1/2, multicenter, randomized, double-blind (Sponsor-open), controlled, multiple-ascending dose (MAD) study to assess the safety, tolerability, and pharmacokinetics, of CMP-002 in participants with SYNGAP1-related disorder (SRD). The study consists of a Baseline Period in which participants will undergo screening assessments to establish the baseline for clinical study assessments. The Baseline Period is followed by a Treatment Period consisting of 3 planned dose level cohorts wherein each participant will receive 3 doses of CMP-002 administered intrathecally (IT) or a control procedure on Days 1, 29, and 85. Each dose level cohort includes a minimum of 8 participants randomized in a 3:1 ratio (CMP-002: control). Following the completion of Dose 3, participants will be followed for an additional 24 weeks in the Follow-up Period.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
80

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: CAMP4 Therapeutics Clinical Trials Information
  • Phone Number: 617-651-8867
  • Email: medinfo@camp4tx.com

Study Locations

  • Argentina
      • Buenos Aires, Argentina
        • Hospital Alemán

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

2 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 confirmation (specifically, of protein truncating mutations in SYNGAP1 exons 5-19) is required. The following mutations in SYNGAP1 exons 5-19 are acceptable:

    1. Frameshift mutations
    2. Nonsense mutations

  • Ability to comply with the protocol and trial procedures detailed in the Schedule of Assessments (SoA)

  • Participant has phenotype consistent with haploinsufficiency

  • Participants must be taking at least one maintenance ASM, have not made changes to ASMs dose or frequency for 6 weeks prior to entering the Baseline Period, and not be planning to change their anti-seizure regimen throughout the study

  • For entry into Treatment Period: All maintenance ASMs and interventions for seizures, as well as any cannabinoid-based products, must have been stable (weight-related adjustments are allowed) during the Baseline Period

Exclusion Criteria:

  • Participant has an exon 1-4, non-truncating mutation, missense mutations, and microdeletions that include the SYNGAP1 gene
  • Participant's prior genetic test indicates a pathogenic or likely pathogenic variant in a gene other than SYNGAP1 (the pathogenic or likely pathogenic variant must be homozygous in cases of known recessive disease)
  • Participant's genetic test indicates SYNGAP1 variant of unknown significance (VUS), mosaicism or additional genetic diagnoses
  • Participant has a history of central nervous system (CNS) disease (other than epilepsy or SRD), including history of bacterial or viral meningitis or brain malformation
  • History of intolerance of IT administration, including intolerance to lumbar puncture
  • For entry into the Treatment Period: Participant has a spinal deformity or other condition that may alter the free flow of CSF or has an implanted CSF drainage shunt. Note: Participants with scoliosis or spinal rods are not excluded if, in the judgement of the Investigator, lumbar puncture can be performed and there is free flow of CSF.
  • Participant has laboratory results outside of the normal ranges at screening and judged as clinically relevant by the Investigator for liver function (including GGT, alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase), kidney function (creatinine, urea), and platelets
  • Positive viral serology test results for HIV type 1 or 2 antibodies, hepatitis B surface antigen or hepatitis C virus (HCV) antibody
  • Participant has participated in any other interventional clinical study of an investigational gene therapy, or participant is currently participating in or has participated in a clinical study involving an investigational medicinal product within 2 months (or 5 half-lives, whichever is longer) prior to Screening
  • Participant is currently using or plans to use unapproved or experimental medicinal products or cannabinoid or cannabidiol or supplemental products, including acetyl-leucine related products
  • Participant is using anticoagulants and antiplatelet agents; and is unwilling or unable to discontinue use per standard of care prior to and after the lumbar puncture dosing Day 1
  • Participant has any other symptomatology of SRD disease or any disease or disorder, including a clinically significant unstable medical condition, CNS disease (other than epilepsy), or brain malformation, which in the opinion of the Investigator, may either put the participant at risk because of participation in the study including consideration of sedation procedures, may influence the results of the study, or may affect the participant's ability to participate in the study.
  • Participant has known hypersensitivity to the excipients of the investigational medicinal product.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: CMP-002 Multiple Ascending Dose (MAD)

Participants 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.
Drug: CMP-002

CMP-002 is an antisense oligonucleotide that will be administered by IT injection.
Sham Comparator: Sham-prick

Participants will receive the sham-prick procedure on Days 1, 29, and 85, and will be followed for 24 weeks after the last procedure.
Other: Sham-prick

The sham-prick procedure involves no injection/puncture or penetration of the dura, but only a skin prick with a needle.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Safety and tolerability of multiple doses of CMP-002: incidence of adverse events and changes from baseline in clinical laboratory results
Time Frame: Screening to Week 36
Screening to Week 36

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
CSF concentrations of the active drug substance of CMP-002 after multiple doses
Time Frame: Day 1 to Week 36
Day 1 to Week 36
Plasma PK of the active drug substance of CMP-002 after multiple doses: Cmax
Time Frame: Day 1 to Day 85
Day 1 to Day 85
Plasma PK of the active drug substance of CMP-002 after multiple doses: AUC(0-24h)
Time Frame: Day 1 to Day 85
Day 1 to Day 85
Urine PK of the active drug substance of CMP-002: Cmax
Time Frame: Day 1
Day 1
Urine PK of the active drug substance of CMP-002: AUC(0-24hr)
Time Frame: Day 1
Day 1

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
CAMP4 Therapeutics Corporation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 1, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 1, 2029

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 1, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 30, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 6, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 13, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 13, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 6, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • SYNG-101
  • 2026-526220-46-00 (Ctis)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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