Gene therapy hope for babies with fatal muscle disease
NCT ID NCT06191354
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new gene therapy called SKG0201 in 12 infants with spinal muscular atrophy type 1, a severe muscle-weakening disease. The treatment aims to improve survival and motor skills by delivering a working gene. Researchers are checking safety and how well the therapy works.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Not a phased trial
Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.
- Participants
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About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jun 2023
- Expected to finish
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Dec 2025
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Up to 180 days
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Type 1 SMA, defined by bi-allelic mutations in the SMN1 gene. 2. Age 180 days or younger at day of infusion. 3. Clinical history and signs are consistent with type I SMA, that is hypotonia on clinical examination, with delay in motor skills, poor head control, rounded shoulder posture, and joint hypermobility. 4. The legal guardian of the subject understands the purpose of the study, the possible risks and rights of the study, agrees that the subject can participate in the study, complete all research steps, tests and visits, and sign the ICF voluntarily. 5. During the study period, according to the change of the subject's condition, the subject's legal guardian is willing to perform standard treatment requirements as suggested by the researcher. Exclusion Criteria: 1. Pulse oximetry \< 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support. 2. Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards (WHO 2006). 3. Active viral infection with significant signs or symptoms and require systematic hospitalization. 4. In the presence of other severe infections or diseases. 5. Known allergy to prednisolone, other glucocorticoids, or their excipients. 6. Clinically significant abnormal laboratory values prior to administration. 7. Previously used other SMA drugs (such as Spinraza, Evrysdi, Zolgensma, etc.) or participated in clinical studies of other SMA drugs. 8. Had received previous or anticipated major surgical procedures during the study assessment period.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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National Children's Medical Center, Children's Hospital of Fudan University
Shanghai, China
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West China Sencond Hospital, Sichuan University / West China women's and children's Hospital
Chengdu, Sichuan, China
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Xinhua Hospital Affiliated To Shanghai Jiao Tong University School Of Medicine
Shanghai, 200092, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.