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GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy

NCT06772402 · Status: ENROLLING_BY_INVITATION · Phase: NA · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2025-03-07

No results posted yet for this study

Summary

This study explored dose escalation of single-arm, open, single intrathecal injection in patients with delayed onset type 2 SMA. The investigator plans to conduct 2 cohorts. It is expected that each dose will be enrolled 3 subjects, with a total of 6 subjects aged from 2-12 years old.

For safety reasons, first subject of each dose cohort needs to complete a 30-day safety observation. After the researcher determines that the dosing is safe and tolerable, the next two subjects can be enrolled in the cohort; The follow-up dose cohort adopts a sentinel test design, with the first subject of each dose group being a sentinel.

During the DLT observation period, if the subject does not observe DLT and the researcher believes that continuing treatment can bring clinical benefits to the subject, the subject will continue to receive treatment; During the DLT observation period, if there is no occurrence of DLT or ≥ grade 2 adverse events related to the investigational drug, it will be escalated to the next dose. If the subject experiences grade ≥ 2 adverse events related to the study drug, the dose will be expanded to 3 subjects for further safety observation. Each subject in each dose cohort will be enrolled on a case by case basis.

Conditions

  • Spinal Muscular Atrophy Type 2

Interventions

GENETIC

GCB-001

GCB-001 is a self-complementary AAV9 carrying a full length human SMN transgenetic product.

Sponsors & Collaborators

  • The Children's Hospital of Zhejiang University School of Medicine

    collaborator OTHER

  • Genecombio Ltd.

    lead OTHER

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Max Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-01-15
Primary Completion
2026-12-31
Completion
2030-12-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06772402 on ClinicalTrials.gov