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Tests treatment safety and results for Spinal Muscular Atrophy Type I
Official title Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
ClinicalTrials.gov ID: NCT07070999
What this study is testing
What is GB221?
GB221 is an investigational medicine, being studied as a potential treatment for spinal muscular atrophy type i.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 12
You may be able to join if
- Symptomatic Participants
- Diagnosis of SMA Type 1 based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and up to 3 copies of SMN2
- Participants must be 2 weeks to \< 12 months of age at the time of dosing with disease onset of during the first 6 months of life.
- Presymptomatic Participants
- At risk of SMA Type 1 based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and up to 2 copies of SMN2
You likely can't join if
- Any suspected or confirmed active viral infection at screening baseline (including HIV, Hepatitis B or C, or human T Cell lymphotropic viruses [HTLV])
- History of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry \<95% saturation.
- Ongoing immunosuppressive therapy or immunosuppressive therapy within 3 months of starting the trial (e.g. corticosteroids, cyclosporine, tacrolimus...
- Participation in a recent SMA treatment clinical trial that, in the opinion of the Investigator, creates unnecessary risks for gene transfer.
- Prior history of gene therapy for any indication, hematopoietic transplant or solid organ transplant
- people with severe scoliosis
See the full eligibility criteria
- Symptomatic Participants
- Diagnosis of SMA Type 1 based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and up to 3 copies of SMN2
- Participants must be 2 weeks to \< 12 months of age at the time of dosing with disease onset of during the first 6 months of life.
- Presymptomatic Participants
- At risk of SMA Type 1 based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and up to 2 copies of SMN2
- Participants must be 2 weeks to \< 5 months (\< 150 days) of age at the time of dosing.
- Any suspected or confirmed active viral infection at screening baseline (including HIV, Hepatitis B or C, or human T Cell lymphotropic viruses [HTLV])
- History of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry \<95% saturation.
- Ongoing immunosuppressive therapy or immunosuppressive therapy within 3 months of starting the trial (e.g. corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab)
- Participation in a recent SMA treatment clinical trial that, in the opinion of the Investigator, creates unnecessary risks for gene transfer.
- Prior history of gene therapy for any indication, hematopoietic transplant or solid organ transplant
- people with severe scoliosis
- Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients.
The study team makes the final eligibility decision.
Where it's taking place
- Porto Alegre, Rio Grande do Sul, Brazil
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 weeks to 12 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Porto Alegre, Rio Grande do Sul, Brazil. Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.