Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

Phase 2Not Yet RecruitingNCT07070999

Gemma Biotherapeutics22 enrolled

Overview

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. participants aged from 2 weeks to younger than 12 months presenting with symptoms of SMA Type 1 who have never received a treatment OR are receiving the drug risdiplam 2. participants aged from 2 weeks to younger than 5 months who are at risk of developing SMA Type 1 (presymptomatic) and have never received treatment OR are receiving the drug risdiplam.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Spinal Muscular Atrophy Type I

Interventions

Eligibility

Sex: ALLMin age: 2 WeeksMax age: 12 Months

Medical Language ↔ Plain English

Inclusion Criteria: * Symptomatic Participants 1. 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 2. 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 1. 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 2. Participants must be 2 weeks to \< 5 months (\< 150 days) of age at the time of dosing. Exclusion Criteria: 1. Any suspected or confirmed active viral infection at screening baseline (including HIV, Hepatitis B or C, or human T Cell lymphotropic viruses \[HTLV\]) 2. History of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry \<95% saturation. 3. Ongoing immunosuppressive therapy or immunosuppressive therapy within 3 months of starting the trial (e.g. corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab) 4. Participation in a recent SMA treatment clinical trial that, in the opinion of the Investigator, creates unnecessary risks for gene transfer. 5. Prior history of gene therapy for any indication, hematopoietic transplant or solid organ transplant 6. Subjects with severe scoliosis 7. Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients.

Outcomes

Primary Outcomes

Number of participants with treatment-related adverse events (AEs) and serious adverse events (SAEs) at Grade 3 or higher as characterized by CTCAEv5.0

Assess the number of treatment-related AEs and SAEs as characterized by CTCAEv5.0