Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 (SPR1NT)
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ClinicalTrials.gov Identifier: NCT03505099 |
Recruitment Status :
Completed
First Posted : April 23, 2018
Results First Posted : January 11, 2022
Last Update Posted : September 7, 2022
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Sponsor:
Novartis Gene Therapies
Collaborator:
PRA Health Sciences
Information provided by (Responsible Party):
Novartis ( Novartis Gene Therapies )
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Study Type | Interventional |
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Study Design | Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
Condition |
Spinal Muscular Atrophy |
Intervention |
Biological: onasemnogene abeparvovec-xioi |
Enrollment | 30 |
Participant Flow
Recruitment Details | A total of 30 participants took part in the trial at 16 sites in the United States, the United Kingdom, Belgium, Canada, Australia and Japan between April 2018 and June 2021. |
Pre-assignment Details | A total of 44 participants were screened, of which 14 were screen failures and 30 were enrolled and received study drug. One participant had 4 copies of SMN2. This participant was excluded from all analysis populations and data is not reported due to privacy concerns. |
Arm/Group Title | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 |
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Participants with bi-allelic deletions of survival of motor neuron 1 (SMN1) and 2 copies of SMN2 received a single dose of AVXS-101 administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 × 10^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued. | Participants with bi-allelic deletions of SMN1 and 3 copies of SMN2 received a single dose of AVXS-101 administered as an IV infusion over 60 minutes at a dose of 1.1 × 10^14 vg/kg on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued. |
Period Title: Overall Study | ||
Started | 14 | 15 |
Received AVXS-101 | 14 | 15 |
Completed | 14 | 15 |
Not Completed | 0 | 0 |
Baseline Characteristics
Arm/Group Title | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | Total | |
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Participants with bi-allelic deletions of survival of motor neuron 1 (SMN1) and 2 copies of SMN2 received a single dose of AVXS-101 administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 × 10^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued. | Participants with bi-allelic deletions of SMN1 and 3 copies of SMN2 received a single dose of AVXS-101 administered as an IV infusion over 60 minutes at a dose of 1.1 × 10^14 vg/kg on Day 1 of the overall study. Participants also received daily doses of prophylactic oral prednisolone starting at a dose of 1-2 mg/kg/day from 1 day prior to AVXS-101 infusion until at least 30 days post-infusion at which point the prednisolone dose could be tapered downwards. At week 9, prednisolone could be discontinued | Total of all reporting groups | |
Overall Number of Baseline Participants | 14 | 15 | 29 | |
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[Not Specified]
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Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 14 participants | 15 participants | 29 participants | |
<=18 years |
14 100.0%
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15 100.0%
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29 100.0%
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Between 18 and 65 years |
0 0.0%
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0 0.0%
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0 0.0%
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>=65 years |
0 0.0%
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0 0.0%
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0 0.0%
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 14 participants | 15 participants | 29 participants | |
Female |
10 71.4%
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9 60.0%
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19 65.5%
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Male |
4 28.6%
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6 40.0%
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10 34.5%
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 14 participants | 15 participants | 29 participants | |
Hispanic or Latino |
4 28.6%
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2 13.3%
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6 20.7%
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Not Hispanic or Latino |
10 71.4%
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13 86.7%
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23 79.3%
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Unknown or Not Reported |
0 0.0%
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0 0.0%
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0 0.0%
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Race/Ethnicity, Customized
Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 14 participants | 15 participants | 29 participants |
Asian |
2 14.3%
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2 13.3%
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4 13.8%
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American Indian or Alaska Native |
0 0.0%
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1 6.7%
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1 3.4%
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Black or African American |
1 7.1%
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0 0.0%
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1 3.4%
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White |
7 50.0%
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10 66.7%
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17 58.6%
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Other |
4 28.6%
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2 13.3%
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6 20.7%
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SMN2 gene modifier mutation (c.859G>C) Present
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 14 participants | 15 participants | 29 participants | |
0 0.0%
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0 0.0%
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0 0.0%
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Outcome Measures
Adverse Events
Limitations and Caveats
Link to the full study results: https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17902
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
Depending on local requirements, Sponsor's consent necessary before publication of study, or Sponsor can review results communications before public release with a right to request changes to communications regarding trial results between 40 to 60 and up to 90 or 120 days, as applicable, from the time submitted to Sponsor for review to remove references to Sponsor's Confidential Information or delay results communications to permit Sponsor to obtain appropriate Intellectual Property protection.
Results Point of Contact
Name/Title: | EMEA Medical Information |
Organization: | Novartis Gene Therapies EU Limited |
Phone: | +353 (1) 566-2364 |
EMail: | medinfoemea.gtx@novartis.com |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Novartis ( Novartis Gene Therapies ) |
ClinicalTrials.gov Identifier: | NCT03505099 |
Other Study ID Numbers: |
AVXS-101-CL-304 2017-004087-35 ( EudraCT Number ) JapicCTI-184203 ( Registry Identifier: JapicCTI ) COAV101A12303 ( Other Identifier: Novartis Pharmaceuticals ) |
First Submitted: | April 13, 2018 |
First Posted: | April 23, 2018 |
Results First Submitted: | December 14, 2021 |
Results First Posted: | January 11, 2022 |
Last Update Posted: | September 7, 2022 |