Trial · NCT07070999
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
NCT07070999, Phase 1, Phase 2, Recruiting, Gemma Biotherapeutics
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1. Trials, record NCT07070999. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07070999
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
- NCT number
- NCT07070999
- Status
- Recruiting
- Phases
- Phase 1, Phase 2
- Enrollment
- 22 participants, estimated
- Lead sponsor
- Gemma Biotherapeutics
- Conditions
- Spinal Muscular Atrophy Type I
- Interventions
- GB221
- First posted
- 2025-07-17
- Last updated
- 2026-06-29
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of participants with treatment-related adverse events (AEs) and serious adverse events (SAEs) at Grade 3 or higher as characterized by CTCAEv5.0 | Up to 18 months across multiple visits | Assess the number of treatment-related AEs and SAEs as characterized by CTCAEv5.0 |
| Primary | Number of Participants with Clinically Significant Changes in Physical Functions | Up to 18 months across multiple visits | Assess the number of participants with clinically significant changes in physical functions. |
| Primary | Number of Participants with Clinically Significant Changes in Neurological Functions | Up to 18 months across multiple visits | Assess the number of participants with clinically significant changes in neurological functions. |
| Primary | Number of Participants with Clinically Significant Changes in Vital signs | Up to 18 months across multiple visits | Assess the number of participants with clinically significant changes in vital signs. |
| Primary | Change in electrocardiogram results | Up to 18 months across multiple visits | ECG will measure RR interval, P Wave, PR interval, PR segment, QRS Complex, ST segment, T wave and QT Interval. |
| Primary | Change in serum cardiac troponin I levels | Up to 18 months across multiple visits | |
| Primary | Number of Participants with Clinically Significant Laboratory Abnormalities as Measured Using Hematology, Chemistry and Coagulation Tests | Up to 18 months across multiple visits | Assess the number of participants with clinically significant laboratory changes including hematology, serum chemistry, and coagulation tests. |
| Primary | Number of Participants with Clinically Significant Laboratory Abnormalities as Measured Using Urine and CSF Tests | Up to 18 months across multiple visits | Assess the number of participants with clinically significant laboratory changes including urine and CSF tests. |
| Primary | Change in markers of immunogenicity | Up to 18 months across multiple visits | Assessment of humoral (NAb and TAb titers) and T-cell (IFNγ ELISpot) immune responses to the AAVhu68 capsid and SMN transgene product in serum and CSF. |
| Secondary | Assess the number of participants who experience permanent ventilation or death | Up to 18 months across multiple visits |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT07070999
GET https://conductscience.org/api/trials/v1/NCT07070999/registered