A Phase 3 interventional study of GC101 adeno-associated virus injection in SMA - Spinal Muscular Atrophy, sponsored by GeneCradle Inc. Recruiting at 7 sites in China. Open to participants aged 2 Years to 12 Years. Per ClinicalTrials.gov, last updated 2025-06-05.
Sponsored by GeneCradle Inc · Phase 3, Interventional, and Treatment
Among 23 studies in Muscular Atrophy, Spinal with a site in China — 8 open to participants now, 13 under way, 4 of them phase 3. Muscular Atrophy, Spinal · China.
Among the 23 studies in Muscular Atrophy, Spinal with a site in China, this one is the 7th largest by planned enrollment of the 16 interventional ones that give a count, is one of 4 that started in 2025, is one of 7 in phase 3, reaches primary completion later than 1 of the 12 other live ones, and was the 19th registered.
282 studies on the registry are indexed under Muscular Atrophy, Spinal; 73 are open to participants now.
Browse Muscular Atrophy, Spinal studies →8 of the 23 studies in Muscular Atrophy, Spinal with a site in China are open now. Recruiting first, the newest first:
Peking University Third Hospital · Sites in China
Ages 6–12
Biogen · Sites in China, United States
Ages 15–60
Biogen · Sites in China, United States, Australia and 4 more countries
Ages 0 days–42 days
GeneCradle Inc · Sites in China
Ages 0 mo–6 mo
Wan-Jin Chen · Sites in China
Ages 1 wk–70 yrs · Healthy volunteers
A study can be open while its sites in China have not opened yet or have stopped. Only those recruiting at a site in China →
Of the 58 Muscular Atrophy, Spinal studies recruiting now, 28 (48%) set no upper age limit and 27 (47%) stop at 75 or younger; 46 take children under 18; 11 take healthy volunteers.
This study: 2 Years to 12 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
23 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 3 · Phase 2 4 · Phase 3 7
First in 2016; this study’s year is marked. 4 started in 2025.
This study's planned enrollment of 50 is above the median of 29 across 156 interventional studies indexed under Muscular Atrophy, Spinal.
Median enrollment 35 across the 16 interventional studies, 200 across the 7 observational. Of the 8 that completed or were terminated, 4 (50%) have results posted.
This tab as a report to print or save as PDF → With an account.
17 of 23 have an industry lead sponsor. The 10 most frequent:
GeneCradle Inc is the lead sponsor of 8 studies on the registry; 6 are open to participants now.
GeneCradle Inc has 2 studies filed under Muscular Atrophy, Spinal — fewer than ten, so there is no comparison to draw.
5 of the studies in Muscular Atrophy, Spinal with a site in China that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06971094Phase 3GeneCradle IncSafety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III | 2026-12-31 |
| NCT05824169GeneCradle IncEvaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients | 2026-12 |
| NCT07822711Peking University Third HospitalEffects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMA | 2027-01-15 |
| NCT05042921BiogenPediatric Spinal Muscular Atrophy (SMA) China Registry | 2027-09-02 |
| NCT06288230Lantu BiopharmaAn Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy | 2027-10-30 |
Add all 5 to your calendar (.ics) → With an account.
Among the 23 studies in Muscular Atrophy, Spinal with a site in China, from their registry updates:
This tab as a report to print or save as PDF → With an account.
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen. The primary objective of the trial is to evaluate the efficacy of GC101 in treating Type 2 SMA. The secondary objectives are to assess the efficacy, safety, and pharmacokinetic (PK) profile of GC101 in treating Type 2 SMA.
The trial is divided into two groups: one group will receive a single intrathecal injection of GC101 at a dose of 1.2E+14 vg per person and discontinue their previous standard-of-care treatment with nusinersen; the other group will continue their previous standard-of-care treatment with nusinersen. Participants will be randomly assigned to the trial group or the control group in a 1:1 ratio.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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.
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.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
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.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
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.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
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.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
single dose of GC101 at dosage of 1.2E+14 vg per person via intrathecal injection
Genetic: GC101 adeno-associated virus injection
Participants will continue to receive treatment according to their previous standard-of-care regimen with nusinersen.
Self-complementary recombinant adeno-associated viral vector (scAAV) containing a single-stranded transgene encoding a codon-optimized human SMN1 gene
HFMSE score change from baseline
HFMSE Motor Function Assessment (Hammersmith Functional Motor Scale Expanded) is a standardized tool specifically designed to evaluate motor function in patients with spinal muscular atrophy (SMA). It includes 33 test items covering actions such as head control, sitting, standing, walking, stair climbing, jumping, as well as balance and coordination, providing a comprehensive assessment of motor function in the trunk and limbs. The maximum total score is 66 points, with higher scores indicating better motor capabilities.
Time frame: 52 weeks
HFMSE score change from baseline
Time frame: 26 weeks
The proportion of participants with an increase in HFMSE score of ≥3 points from baseline.
Minimal clinically important differences (MCID) were defined as an HFMSE score improvement of ≥3 points.
Time frame: 26 and 52 weeks
Changes in WHO-MGRS motor milestones from baseline.
WHO-MGRS (World Health Organization Multicenter Growth Reference Study) motor development milestones include: Sitting without support Standing with support Crawling on hands and knees Walking with support Standing alone Walking alone
Time frame: 26 and 52 weeks
RULM score change from baseline.
RULM Motor Function Assessment (Revised Upper Limb Module) is a standardized tool designed to evaluate upper limb function in patients with spinal muscular atrophy (SMA). A maximum total score is 37 points. Higher scores indicate better upper limb performance.
Time frame: 26 and 52 weeks
SMAIS score change from baseline.
Spinal Muscular Atrophy Independence Scale （SMAIS）is a patient- and caregiver-reported outcome measure designed to assess the level of assistance required by individuals with Type 2 and non-ambulant Type 3 spinal muscular atrophy (SMA) to perform activities of daily living. Higher scores indicate greater independence, with a range from 0 to 44
Time frame: 26 and 52 weeks
The incidence of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
Time frame: 52 weeks
Plan to share: Undecided
No publications or documents are linked to this record.
Started May 2025 (actual). Primary completion is estimated for Dec 2026, 19 months after the start.
Of 18 phase 3 interventional studies under Muscular Atrophy, Spinal, half reached primary completion within 33 months of starting (the median), counting those still running; a quarter within 24 and three quarters within 63. This study's planned 19 months is below that.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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GeneCradle Inc