An interventional study of GCB-001 in Spinal Muscular Atrophy Type 2, sponsored by Genecombio Ltd.. Enrolling by invitation at 1 site in China. Open to participants aged 2 Years to 12 Years. Per ClinicalTrials.gov, last updated 2026-09-11.
Sponsored by Genecombio Ltd. · Not applicable, Interventional, and Treatment
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.
61 studies on the registry are indexed under Spinal Muscular Atrophies of Childhood; 18 are open to participants now.
This study's planned enrollment of 6 is below the median of 15 across 41 interventional studies indexed under Spinal Muscular Atrophies of Childhood.
Browse Spinal Muscular Atrophies of Childhood studies →Genecombio Ltd. is the lead sponsor of 2 studies on the registry; 2 are open to participants now.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Low dose is the first cohort of the study with a low dose level.
Genetic: GCB-001
High dose is the first cohort of the study with a high dose level.
Genetic: GCB-001
GCB-001 is a self-complementary AAV9 carrying a full length human SMN transgenetic product.
The rate of adverse events from baseline to 12 months after administration Assessed by CTCAE v4.0.
Time frame: 0-12 months
AAV viral load after a single administration.
Detect the AAV viral load by central lab.
Time frame: 0-12 months
AAV viral immunogenicity after a single administration.
Detect the AAV viral immunogenicity by central lab.
Time frame: 0-12 months
AAV viral shedding after a single administration.
Detect the AAV viral shedding by central lab.
Time frame: 0-12 months
The change in total HFMSE (Hammersmith Functional Motor Scale Expanded) score from baseline at 12 months after a single administration
This 66-point scale (66 = very much improved, 0 = very much worse, etc.) is used by the clinician to assess the participant's performance status; higher scores indicate better status.
Time frame: 0-12 months
The change in total RULM (Revised Upper Limb Module) score from baseline at 12 months after a single administration
This 38-point scale (38 = very much improved, 0 = very much worse, etc.) is used by the clinician to assess the participant's performance status of upper limb; higher scores indicate better status.
Time frame: 0-12 months
Plan to share: No
No publications or documents are linked to this record.
Started Jan 2025 (actual). Primary completion is estimated for Dec 2027, 35 months after the start.
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Spinal Muscular Atrophies of Childhood →
Genecombio Ltd.