Recruiting PHASE1/PHASE2 INTERVENTIONAL 4-arm NCT07070999

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

A Phase 1-2, Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of GB221 Delivered Into the Cisterna Magna of Pediatric Participants From 2 Weeks to Younger Than 12 Months of Age With Spinal Muscular Atrophy Type 1

Sponsor: Gemma Biotherapeutics

Conditions Spinal Muscular Atrophy Type I

Interventions GB221

Updated 3 times since 2025 Last updated: Feb 26, 2026 Started: Jan 6, 2026 Primary completion: Dec 31, 2028 Completion: Apr 30, 2029

This information is for research purposes only and is not medical advice. Consult a healthcare provider before making any medical decision.

Listed as NCT07070999, this PHASE1/PHASE2 trial focuses on Spinal Muscular Atrophy Type I and remains actively recruiting participants. Sponsored by Gemma Biotherapeutics, it has been updated 3 times since 2026, reflecting limited change activity. This study adds to the evidence base for this therapeutic area through structured, versioned documentation.

Status Flow

Change History

3 versions recorded

Mar 2026 — Present [monthly]

Recruiting PHASE1/PHASE2

Status: Not Yet Recruiting → Recruiting
Jan 2026 — Mar 2026 [monthly]

Not Yet Recruiting PHASE1/PHASE2
Aug 2025 — Jan 2026 [monthly]

Not Yet Recruiting PHASE1/PHASE2

First recorded

Eligibility Summary

No eligibility information available.

Contact Information

Sponsor contact:

Gemma Biotherapeutics

Data source: Gemma Biotherapeutics

For direct contact, visit the study record on ClinicalTrials.gov .

Study Locations

• Porto Alegre, Brazil