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Safety and Tolerability of Intrathecal CMP-002 in Participants with SYNGAP1-Related Disorder

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What is this trial about?

A plain-language summary of the goals, design and what participants do

SYNGAP1-Related Disorder is a rare genetic condition that affects brain development and can cause seizures, learning difficulties, and problems with movement. The study is testing a new medicine called CMP-002, which is a short piece of genetic material designed to block a specific RNA that interferes with normal brain function. The medicine is given intrathecal, meaning it is injected directly into the fluid that surrounds the spinal cord, so it can reach the brain more effectively.

The purpose of the study is to see whether the medicine is safe and can be tolerated when given at increasing dose levels. Participants will be randomly assigned to receive either the study drug or a harmless fluid called placebo, and neither the participants nor the doctors will know which one is given. The study involves several visits over a few months, during which the medication is administered, and simple checks are done to watch for any side effects.

Throughout the trial, doctors will monitor participants for any unwanted reactions, take routine blood and urine samples, and perform basic lab tests to look for changes. These checks help ensure that any problems are spotted early and that the medication is being used safely.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment Confirmation and Baseline Assessments

    After agreeing to join the study, you will complete a series of initial tests that record your current health status, laboratory results, and neurological function.

    These assessments create a reference point for comparing any changes that occur during the trial.

  2. Step 2

    Randomization and Assignment to Study Medication

    You will be randomly placed into one of two groups without knowing which group you are in.

    One group receives the investigational medicine CMP-002, an antisense oligonucleotide designed to target the SYNGAP1 gene; the other group receives a placebo consisting of artificial cerebrospinal fluid (aCSF).

  3. Step 3

    First Intrathecal Administration

    A qualified medical professional will inject the assigned study medication directly into the spinal fluid (intrathecal injection).

    The exact amount of CMP-002 or placebo is defined by the study protocol and is given as a single dose at this visit.

  4. Step 4

    Post‑Injection Monitoring

    You will remain in the clinic for several hours after the injection so that staff can observe you for any immediate reactions.

    Vital signs, neurological status, and any symptoms will be recorded.

  5. Step 5

    Scheduled Follow‑Up Visits and Additional Doses

    You will return to the clinic at regular intervals (for example, every 4 weeks) for additional intrathecal doses as the study tests increasing dose levels.

    Each visit includes the same injection procedure, safety checks, and collection of blood, urine, and spinal fluid samples to measure drug levels.

  6. Step 6

    Safety and Laboratory Assessments

    During each visit, laboratory tests will be performed to monitor organ function and to detect any adverse events.

    The study team will track any side effects, including those that might limit the amount of drug you can receive.

  7. Step 7

    End‑of‑Study Evaluation

    At the final scheduled visit, a comprehensive evaluation will be conducted to compare your health measurements with the baseline data.

    The results will help determine the overall safety and tolerability of the multiple doses of CMP-002.

Who can join the trial?

7 criteria

  • Provide written informed consent (a signed agreement that you understand the study and agree to take part) according to local rules.
  • Be at least 2 years old but younger than 18 years old when the consent is given.
  • Have a doctor‑confirmed diagnosis of SYNGAP1‑related disorder and a genetic test showing a specific type of change called a protein truncating mutation in the gene’s exons 5‑19; acceptable changes are frameshift mutations (which shift the genetic reading frame) or nonsense mutations (which create a premature stop signal).
  • Be able to follow the study schedule and complete the required visits and tests.
  • Show a physical or functional picture (called a phenotype) that matches haploinsufficiency (meaning the body has only one working copy of the SYNGAP1 gene instead of the normal two).
  • Be taking at least one regular anti‑seizure medicine (maintenance ASM) and have kept the dose and how often you take it the same for at least 6 weeks before the study starts, with no plans to change it during the study.
  • During the initial study phase (baseline period), keep all seizure medicines, other seizure treatments, and any cannabis‑derived products stable (small changes for weight are allowed).

Who cannot join the trial?

13 criteria

  • Having certain types of SYNGAP1 gene changes such as exon 1‑4 non‑truncating mutations, missense mutations, or microdeletions that affect the gene.
  • Currently using or planning to use unapproved or experimental medicinal products, including cannabinoid, cannabidiol, or supplements like acetyl‑leucine.
  • Taking blood‑thinning medicines (anticoagulants or antiplatelet agents) and being unable or unwilling to stop them before and after the lumbar puncture procedure.
  • Having any other symptoms of SYNGAP1‑related disorder or any serious, unstable medical condition, especially a central nervous system (CNS) disease (other than epilepsy) or a brain malformation, that could make participation risky, affect the study results, or limit the ability to take part.
  • Known hypersensitivity (allergy) to any of the inactive ingredients (excipients) in the study medication.
  • Prior genetic testing showing a disease‑causing (pathogenic) or likely disease‑causing variant in a gene other than SYNGAP1 (the variant must be present in both copies of the gene – homozygous – for recessive diseases).
  • Genetic testing indicating a SYNGAP1 change of uncertain meaning (variant of unknown significance (VUS)), mosaicism, or any additional genetic diagnoses.
  • History of a central nervous system (CNS) disease (other than epilepsy or SYNGAP1‑related disorder), such as past bacterial or viral meningitis or a brain malformation.
  • Intolerance to intrathecal (IT) administration, meaning an inability to tolerate a lumbar puncture.
  • Having a spinal deformity or any condition that could block the free flow of cerebrospinal fluid (CSF), or having an implanted CSF drainage shunt (note: scoliosis or spinal rods are allowed only if the investigator believes a lumbar puncture can be performed and CSF will flow freely).
  • Laboratory test results at screening that are outside normal ranges and judged clinically important by the investigator for liver function (including GGT, ALT, AST, alkaline phosphatase), kidney function (creatinine, urea), or platelet count.
  • Positive viral serology test for HIV type 1 or 2 antibodies, hepatitis B surface antigen, or hepatitis C antibody.
  • Participation in any other interventional study of gene therapy, or current or recent (within the past 2 months or 5 half‑lives, whichever is longer) involvement in a study using an investigational medicinal product.
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Investigated drugs

CMP-002 is an experimental medicine that uses a short piece of genetic material called an antisense oligonucleotide. This molecule is designed to bind to the regulatory RNA of the SYNGAP1 gene, which is involved in a rare brain disorder known as SYNGAP1‑related disorder. By attaching to this RNA, CMP-002 aims to reduce the production of abnormal protein that contributes to the disorder’s symptoms. The drug is given directly into the fluid surrounding the spinal cord (intrathecal administration) so it can reach the brain more effectively. In the trial, participants receive several increasing doses of CMP-002 to see if it is safe and well‑tolerated, and to explore whether it might improve neurological function in people with SYNGAP1‑related disorder.

What is already known about the treatment

CMP-002 - CMP-002 is given as a sterile solution that is injected directly into the spinal fluid (intrathecal injection). It is an experimental antisense oligonucleotide designed to bind to the regulatory RNA of the SYNGAP1 gene, reducing its harmful activity; this places it in the class of gene‑targeted antisense therapies. The drug is currently in early clinical testing and has not yet been approved for any use, but it is being studied for SYNGAP1‑related disorder, a rare neurological condition. By lowering the amount of abnormal SYNGAP1 protein, it aims to improve brain signaling and reduce symptoms such as seizures and developmental delays.

Investigated diseases

SYNGAP1-related disorder - SYNGAP1-related disorder is a genetic condition that affects brain development. It typically becomes apparent in early childhood with delays in reaching milestones such as sitting, walking, and talking. Children often have difficulties with speech, learning, and behavior, and many experience seizures. Motor skills may be clumsy and coordination can be poor. As the child grows, the pattern of challenges may shift, with ongoing needs for support in education and daily activities. The disorder persists throughout life, with the severity of symptoms varying between individuals.
Trial detailsLast updated 23 Oct 2026
Age0-17PhasePhase I/IITrial ID2026-526220-46-00Protocol codeSYNG-101Estimated enrolment56 patientsSponsorCamp4 Therapeutics Corp.

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