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Gene therapy hope for babies with fatal muscle disease

NCT ID NCT07554924

Disease control Sponsor: Lanyue Biotech (Hangzhou) Co., Ltd. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Aug 27, 2026 · Updated 3 times

Summary

This early-phase trial tests a gene therapy called SKG0201 in 11 infants with spinal muscular atrophy (SMA) type I, a severe muscle-weakening disease. The therapy uses a harmless virus to deliver a working copy of the missing SMN1 gene. The study aims to see if it is safe and can improve survival and motor skills.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
SKG0201 (gene therapy using a harmless virus to deliver a working SMN1 gene)
What this could lead to
If successful, this could provide a one-time treatment that improves motor function and survival in infants with SMA type I.
What could go wrong
This is an early, small trial (11 infants) testing safety and dosing. Gene therapy carries risks like immune reactions or liver issues, and it may not work for all.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 11 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2026

Expected to finish

May 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 180 days

Sex

Anyone

Healthy volunteers

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

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 I SMA, defined by bi-allelic mutations in the SMN1 gene. 2. Clinical history and signs are consistent with type I SMA, such as hypotonia, delayed motor function development, and poor head control. 3. On the day of administration, the age of the subjects do not exceed 180 days after birth. 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. 3. Active viral infection. 4. In the presence of other severe infections or diseases that require systemic anti-infection treatment. 5. Known allergy to prednisolone, other glucocorticoids, or SKG0201's excipients. 6. Clinically significant abnormal laboratory values prior to administration. 7. Previously used Zolgensma or other SMA gene therapy drugs, or currently participating in other SMA clinical studies on therapeutic drugs. 8. Having previously undergone major surgery or expected to undergo major surgery during the study assessment period.

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Conditions

The condition(s) this trial relates to.

Spinal Muscular Atrophies of Childhood spinal muscular atrophy, type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Spinal muscular atrophy 1

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    3 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Children's Hospital of Fudan University

    RECRUITING

    Shanghai, Shanghai Municipality, China

  • Children's Hospital, Zhejiang University School of Medicine

    RECRUITING

    Hangzhou, Zhejiang, China

  • Peking University First Hospital

    RECRUITING

    Beijing, Beijing Municipality, China

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