NCT06772402Enrolling by invitation
GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy
An Open Label, Single Arm IIT Clinical Study Evaluating the Safety, Tolerability, and Preliminary Efficacy of GCB-001 in the Treatment of Patients With Delayed Onset Type 2 SMA Who Can Sit Alone But Cannot Walk.
Source: ClinicalTrials.gov · Last updated 11 Sept 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Enrolling by invitation
- Study type
- Interventional
- Enrollment
- 6 (estimated)
- First submitted
- 3 Jan 2025
- Start date
- 15 Jan 2025
- Primary completion date
- Dec 2027
- Lead sponsor
- Genecombio Ltd.
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT06772402
What you commit to
- Study type: Interventional
- Planned participants: 6
- From 15 Jan 2025 to the primary completion date, Dec 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Researchers believe that gene replacement therapy may cause unnecessary risk of concomitant diseases, such as serious cardiovascular and cerebrovascular diseases, digestive tract diseases, liver and kidney dysfunction diseases, diabetes, known epilepsy, convulsions, convulsions or family history of psychosis;
- Subjects who have participated in AAV gene therapy or have participated in or are currently participating in clinical trials of other SMA drugs;
- Received treatment with risperidone within 15 days prior to administration;
- Subjects who have been treated with β 2 receptor agonists within 30 days prior to treatment (excluding inhaled salbutamol);
Other illness
- Subjects with allergic constitution, including those who are allergic or hypersensitive to prednisolone, other glucocorticoids or their excipients, and allergic to local anesthetics;
Lab values
- Received treatment with Nordenafil Sodium Injection within 4 months prior to administration;
- The serum Anti-AAV9 neutralizing antibody titer is greater than 1:200.
Other
- During the screening period, non-invasive ventilation support should be used for at least 12 hours per day;
Who the study is looking for
- Age ≥ 2 years and ≤ 12 years, gender not limited;
- Meet the clinical diagnostic criteria for type 2 SMA, have an onset age form 6 months to 18 months, are diagnosed with SMN1 double allele pathogenic mutation, have 2-4 copies of SMN2 gene, and meet the clinical diagnostic criteria for SMA 5qSMA;
- Capable of sitting alone but has never acquired the ability to walk independently (according to HFMSE standards, sitting alone: able to maintain a sitting position without hand support and count to 3 or more; walking independently: able to walk 4 or more steps without assistance);
- The guardians of the subjects are able to understand and willing to comply with the requirements and procedures of protocol, voluntarily participate and sign the informed consent form.
Age in the registry: 2 years to 12 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Children's Hospital ZheJiang Univisity School Of Medicine Coordinator of this site? Confirm its status | Hangzhou | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.