RecruitingPHASE1, PHASE2NCT07070999
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
Studying Spinal Muscular Atrophy
Last synced from ClinicalTrials.gov
ℹ
Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing dataKey facts
- Sponsor
- Gemma Biotherapeutics
- Principal Investigator
- May Orfali, MDGemma Biotherapeutics
- Intervention
- GB221(biological)
- Enrollment
- 22 enrolled
- Eligibility
- All sexes
- Timeline
- 2026 – 2029
Study locations (1)
- Hospital de Clínicas de Porto Alegre, Porto Alegre, Rio Grande do Sul, Brazil
Primary source
Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.
Open NCT07070999 on ClinicalTrials.govOther trials for Spinal Muscular Atrophy
Additional recruiting or active studies for the same condition.
- RECRUITINGNANCT07488338HABIT-ILE + FST in Children With SMA: Preliminary EffectivenessTeachers College, Columbia University
- RECRUITINGNCT07400198Gait and Bone Health in SMAJacqueline Montes
- RECRUITINGNANCT07321977Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic VolunteersInstitut de Myologie, France
- RECRUITINGPHASE2NCT07287982A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophyargenx
- RECRUITINGNANCT07286565Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn ScreeningCentre Hospitalier Universitaire de Liege
- RECRUITINGNCT06532474Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed TherapiesSt. Jude Children's Research Hospital
- RECRUITINGPHASE3NCT07265232Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).Lantu Biopharma
- RECRUITINGNANCT07223489Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health SystemMedstar Health Research Institute