A Phase 1 interventional study of Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy, sponsored by Novartis Gene Therapies. Terminated at 11 sites in United States. Open to participants aged 6 Months to 60 Months. Per ClinicalTrials.gov, last updated 2026-01-26.
Sponsored by Novartis Gene Therapies (part of Novartis) · Phase 1, Interventional, and Treatment
Among 121 studies in Muscular Atrophy, Spinal with a site in United States — 21 open to participants now, 39 under way, 7 of them phase 3. Muscular Atrophy, Spinal · United States.
Among the 121 studies in Muscular Atrophy, Spinal with a site in United States, this one is the 39th largest by enrollment of the 76 interventional ones that give a count, is one of 4 that started in 2017, is one of 11 in phase 1, and was the 49th registered.
282 studies on the registry are indexed under Muscular Atrophy, Spinal; 73 are open to participants now.
Browse Muscular Atrophy, Spinal studies →21 of the 121 studies in Muscular Atrophy, Spinal with a site in United States are open now. Recruiting first, the newest first:
Johns Hopkins University · Sites in United States
St. Jude Children's Research Hospital · Sites in United States
Biogen · Sites in United States
Biogen · Sites in United States, China
Novartis Pharmaceuticals · Sites in United States
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 58 Muscular Atrophy, Spinal studies recruiting now, 28 (48%) set no upper age limit and 27 (47%) stop at 75 or younger; 46 take children under 18; 11 take healthy volunteers.
This study: 6 Months to 60 Months · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
121 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 18 · Phase 2 27 · Phase 3 20 · Phase 4 6
First in 1986; this study’s year is marked. 4 started in 2017.
This study's enrollment of 32 is above the median of 29 across 156 interventional studies indexed under Muscular Atrophy, Spinal.
Median enrollment 33 across the 76 interventional studies, 75 across the 43 observational. Of the 76 that completed or were terminated, 33 (43%) have results posted.
This tab as a report to print or save as PDF → With an account.
61 of 121 have an industry lead sponsor. The 10 most frequent:
Novartis Gene Therapies is the lead sponsor of 8 studies on the registry; none are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 5 (100%) have results posted.
Novartis Gene Therapies has 4 studies filed under Muscular Atrophy, Spinal — fewer than ten, so there is no comparison to draw.
7 of the studies in Muscular Atrophy, Spinal with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05067790Phase 3BiogenA Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) | 2027-09-24 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
| NCT07223320Teachers College, Columbia UniversityHABIT-ILE + FST in Children With SMA | 2026-12-31 |
| NCT07400198Jacqueline MontesGait and Bone Health in SMA | 2027-06-30 |
| NCT06532474St. Jude Children's Research HospitalExploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies | 2027-09 |
Among the 121 studies in Muscular Atrophy, Spinal with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.
This is a Phase 1, single-dose administration study of infants and children with a genetic diagnosis consistent with spinal muscular atrophy (SMA), bi-allelic deletion of survival motor neuron 1 gene (SMN1) and 3 copies of survival motor neuron 2 gene (SMN2) without the genetic modifier who are able to sit but cannot stand or walk at the time of study entry. Patients will receive onasemnogene abeparvovec-xioi in a dose comparison safety study of two (or three) potential therapeutic doses. Patients will be stratified in two groups, those ≥6 months and \< 24 months of age at time of dosing and those ≥ 24 months and \< 60 months of age at time of dosing. At least 15 patients ≥ 6 months and \< 24 months, and at least 12 patients ≥ 24 \< 60 months will be enrolled.
The first cohort will enroll three patients (Cohort 1) ≥ 6 months and \< 24 months of age who will receive administration of 6.0 × 1013 vg of onasemnogene abeparvovec-xioi (Dose A). There will be at least a four week interval between the dosing of each patient within the cohort. Novartis Gene Therapies, Inc. will confer with the Data Safety Monitoring Board (DSMB) on all Grade III or higher AEs within approximately 48 hours of awareness that are possibly, probably or definitely related to the study agent before continuing enrollment. Safety data will be reviewed by the DSMB during quarterly meetings; following enrollment of the three patients and based upon the available safety data a decision will be made whether to: a) stop due to toxicity, or b) proceed to Cohort 2 using Dose B.
Should the determination be made to advance to Dose B, three patients \< 60 months of age will be enrolled (Cohort 2) and will receive administration of 1.2 × 1014 vg of onasemnogene abeparvovec-xioi (Dose B). Again, there will be at least a four-week interval between dosing of the three patients within the cohort. Based on the available safety data from the three Cohort 2 patients and all of the Cohort 1 patients, the DSMB will decide and document during quarterly meetings whether further four-week intervals between patients dosing is necessary. Novartis Gene Therapies, Inc. will take this recommendation into consideration and will make the final determination whether to persist with four-week intervals between patients dosing going forward; the decision will be communicated to sites and Institutional Review Boards (IRBs) in a formal sponsor letter. Novartis Gene Therapies, Inc. will confer with the DSMB on all Grade III or higher AEs within approximately 48 hours of awareness that are possibly, probably or definitely related to the study agent before continuing enrollment. Safety data will be reviewed by the DSMB during quarterly meetings; following enrollment of the first six patients and based upon available safety data, a decision will be made whether to: a) stop due to toxicity, or b) continue to enroll an additional 21 patients until there are a total of 12 patients > 6 months and \< 24 months and 12 patients ≥ 24 and \< 60 months that have all received Dose B.
Based upon an ongoing assessment of safety and efficacy data from patients treated with the 1.2 × 1014 vg dose, an option for testing of a third dose (Dose C), will be considered. If, based on all available data, this is judged to be safe and necessary, three patients \< 60 months of age will receive Dose C, 2.4 × 1014 vg administered IT. A meeting of the DSMB will be called to obtain a recommendation on the safety of escalating to a higher dose prior to proceeding. If a decision is made to proceed to testing a higher dose, there will again be a four-week interval between dosing of the first three patients receiving Dose C, as in Cohorts 1 and 2. Safety data will be reviewed by the DSMB during quarterly meetings. Following enrollment of the first three Dose C patients and based upon available safety data, the DSMB will be consulted and a decision will be made whether to: a) stop dosing Dose C due to safety concerns, or b) continue to enroll an additional 21 patients until there are a total of 12 patients > 6 months and \< 24 months and 12 patients ≥ 24 and \< 60 months that have received Dose C.
Patients from Cohort 3 will be followed for a total of 15 months post-dose. The primary analyses for efficacy will be assessed when all patients reach 12 months post-dose and the primary analyses for safety will be assessed when the last patient of Cohort 3 reaches 15 months post-dose (and database lock will be performed after the last patient reaches 15 months post-dose).
Key Inclusion Criteria
Key Exclusion Criteria
Severe non-pulmonary/respiratory tract infection within four (4) weeks before study dosing or concomitant illness that in the opinion of the Principal Investigator (PI) creates unnecessary risks for gene transfer such as:
Anti-AAV9 antibody titers >1:50 as determined by Enzyme-linked Immunosorbent Assay (ELISA) binding immunoassay
Participation in recent SMA treatment clinical trial or receipt of an or approved compound product or therapy received with the intent to treat SMA at any time prior to screening for this study
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Intrathecal administration 6.0 X 10\^13 vg of onasemnogene abeparvovec-xioi
Biological: Onasemnogene Abeparvovec-xioi
Intrathecal administration 1.2 X 10\^14 vg of onasemnogene abeparvovec-xioi
Biological: Onasemnogene Abeparvovec-xioi
Intrathecal administration 2.4 X 10\^14 vg of onasemnogene abeparvovec-xioi
Biological: Onasemnogene Abeparvovec-xioi
Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
Also known as: Zolgensma
Age 6 to <24 Months Only: Number of Participants Who Achieved the Ability to Stand Alone
Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported.
Time frame: From Day 1 up to Month 12
Age 24 to <60 Months Only: Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score at Month 12
The HFMSE contained 33 items which were scored on a scale of 0-2 with a total achievable score ranging from 0, if all activities are failed, to 66, if all the activities are achieved. A positive change from baseline indicates a better outcome.
Time frame: Baseline and Month 12
Number of Participants Who Experienced a Treatment-emergent Adverse Event (TEAE)
A TEAE was defined as any event that began or worsened in severity on or after the administration of AVXS-101 through the last study visit. Evaluation of TEAEs included the number of participants with at least one: * TEAE * Serious TEAE * TEAE related to AVXS-101 * TEAE with Common Terminology Criteria for Adverse Events (CTCAE) grade ≥ 3 (grade 3 = severe or medically significant to grade 5 = death related to TEAE)
Time frame: Adverse events were collected from the single dose of study treatment until the end of study visit (12 months for Cohort 1 and 2 and 15 months for Cohort 3)
Number of Participants Who Achieved the Ability to Walk Alone
Defined by the BSID GM subtest performance criteria number 43, confirmed by video recording, as a participant who takes 5 coordinated independent steps.
Time frame: From Day 1 up to Month 12
Average Number of Hours Per Day of Non-invasive Ventilatory Support
Participants were assessed by a pulmonologist and may have been fitted with a non-invasive positive pressure ventilatory (e.g., Bilevel Positive Airway Pressure BiPAP) at the discretion of the pulmonologist and/or investigator. The number of hours per day of non-invasive ventilatory support was captured continuously by the device.
Time frame: Months 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, and 12
A total of 32 participants took part in the trial at 11 sites in the United States between December 2017 and May 2021.
| Milestone | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | Cohort 3: 2.4E14 vg - Age 24 to <60 Months |
|---|---|---|---|---|---|
| Started | 3 | 13 | 12 | 4 | 0 |
| Received avxs-101 | 3 | 13 | 12 | 4 | 0 |
| Completed | 3 | 13 | 12 | 4 | 0 |
| Not completed | 0 | 0 | 0 | 0 | 0 |
Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported.
| Participants | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months |
|---|---|---|---|
| Able to Stand Alone | 1 | 1 | 0 |
| Unable to Stand Alone | 2 | 12 | 4 |
The HFMSE contained 33 items which were scored on a scale of 0-2 with a total achievable score ranging from 0, if all activities are failed, to 66, if all the activities are achieved. A positive change from baseline indicates a better outcome.
| score on a scale | Cohort 2: 1.2E14 vg - Age 24 to <60 Months |
|---|---|
| Age 24 to <60 Months Only: Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score at Month 12 | 6.0 (3.7 to 8.3) |
A TEAE was defined as any event that began or worsened in severity on or after the administration of AVXS-101 through the last study visit. Evaluation of TEAEs included the number of participants with at least one: * TEAE * Serious TEAE * TEAE related to AVXS-101 * TEAE with Common Terminology Criteria for Adverse Events (CTCAE) grade ≥ 3 (grade 3 = severe or medically significant to grade 5 = death related to TEAE)
| Participants | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months |
|---|---|---|---|---|
| TEAE | 3 | 13 | 12 | 4 |
| Serious TEAE | 1 | 2 | 4 | 0 |
| TEAE Related to AVXS-101 | 0 | 7 | 4 | 1 |
| TEAE with CTCAE Grade ≥ 3 | 1 | 4 | 4 | 0 |
Defined by the BSID GM subtest performance criteria number 43, confirmed by video recording, as a participant who takes 5 coordinated independent steps.
| Participants | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months |
|---|---|---|---|---|
| Able to Walk Alone | 0 | 1 | 0 | 0 |
| Unable to Walk Alone | 3 | 12 | 12 | 4 |
Participants were assessed by a pulmonologist and may have been fitted with a non-invasive positive pressure ventilatory (e.g., Bilevel Positive Airway Pressure BiPAP) at the discretion of the pulmonologist and/or investigator. The number of hours per day of non-invasive ventilatory support was captured continuously by the device.
| hours/day | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months |
|---|---|---|---|---|
| Month 2 | — | — | 10.530 ± NA | — |
| Month 3 | — | — | 2.930 ± NA | — |
| Month 4 | — | 0.000 ± NA | 5.860 ± NA | — |
| Month 5 | — | 0.000 ± NA | 4.610 ± NA | — |
| Month 6 | — | 2.590 ± NA | 4.490 ± NA | — |
| Month 7 | — | 4.550 ± NA | 7.490 ± NA | — |
| Month 8 | — | 7.170 ± NA | 8.290 ± NA | — |
| Month 9 | — | 8.690 ± NA | 7.690 ± NA | — |
| Month 10 | — | 10.320 ± NA | 9.900 ± NA | — |
| Month 11 | — | 14.050 ± NA | 3.460 ± NA | — |
| Month 12 | — | 10.120 ± NA | 0.040 ± NA | — |
Collected over Adverse events were collected from the single dose of study treatment until the end of study visit (12 months for Cohort 1 and 2 and 15 months for Cohort 3). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | 0/3 (0%) | 1/3 (33.3%) | 3/3 (100%) |
| Cohort 2: 1.2E14 vg - Age 6 to <24 Months | 0/13 (0%) | 2/13 (15.4%) | 13/13 (100%) |
| Cohort 2: 1.2E14 vg - Age 24 to <60 Months | 0/12 (0%) | 4/12 (33.3%) | 12/12 (100%) |
| Cohort 3: 2.4E14 vg - Age 6 to <24 Months | 0/4 (0%) | 0/4 (0%) | 4/4 (100%) |
| Event | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months |
|---|---|---|---|---|
| Blood alkaline phosphatase increasedInvestigations | 1/3 | 1/13 | 0/12 | 0/4 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 1/3 | 0/13 | 0/12 | 0/4 |
| BronchitisInfections and infestations | 0/3 | 0/13 | 1/12 | 0/4 |
| InfluenzaInfections and infestations | 0/3 | 0/13 | 1/12 | 0/4 |
| PneumoniaInfections and infestations | 0/3 | 0/13 | 1/12 | 0/4 |
| Pneumonia respiratory syncytial viralInfections and infestations | 0/3 | 0/13 | 1/12 | 0/4 |
| Respiratory tract infection viralInfections and infestations | 0/3 | 0/13 | 1/12 | 0/4 |
| Rhinovirus infectionInfections and infestations | 0/3 | 0/13 | 1/12 | 0/4 |
| Acute respiratory failureRespiratory, thoracic and mediastinal disorders | 0/3 | 0/13 | 1/12 | 0/4 |
| AsthmaRespiratory, thoracic and mediastinal disorders | 0/3 | 0/13 | 1/12 | 0/4 |
| Event | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months |
|---|---|---|---|---|
| PyrexiaGeneral disorders | 3/3 | 6/13 | 7/12 | 2/4 |
| Upper respiratory tract infectionInfections and infestations | 2/3 | 10/13 | 5/12 | 3/4 |
| CoughRespiratory, thoracic and mediastinal disorders | 0/3 | 3/13 | 7/12 | 1/4 |
| VomitingGastrointestinal disorders | 0/3 | 5/13 | 3/12 | 2/4 |
| ConstipationGastrointestinal disorders | 0/3 | 2/13 | 3/12 | 2/4 |
| TeethingGastrointestinal disorders | 1/3 | 2/13 | 0/12 | 1/4 |
| Infusion site bruisingGeneral disorders | 1/3 | 0/13 | 0/12 | 0/4 |
| HypersensitivityImmune system disorders | 1/3 | 0/13 | 0/12 | 0/4 |
| Otitis mediaInfections and infestations | 1/3 | 0/13 | 3/12 | 0/4 |
| PneumoniaInfections and infestations | 1/3 | 0/13 | 1/12 | 0/4 |
Safety Analysis Set: All participants given an AVXS-101 intrathecal injection. Participants were analyzed according to actual dose received.
| Age, Categorical(Participants) | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | Total |
|---|---|---|---|---|---|
| <=18 years | 3 | 13 | 12 | 4 | 32 |
| Between 18 and 65 years | 0 | 0 | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 | 0 | 0 |
| Age, Continuous(months) | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | Total |
|---|---|---|---|---|---|
| Mean | 17.2 ± 4.1 | 16.7 ± 4.5 | 37.5 ± 10.6 | 16.9 ± 5.6 | 24.6 ± 12.5 |
| Sex: Female, Male(Participants) | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | Total |
|---|---|---|---|---|---|
| Female | 2 | 6 | 6 | 0 | 14 |
| Male | 1 | 7 | 6 | 4 | 18 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 2 | 3 | 0 | 0 | 5 |
| Not Hispanic or Latino | 1 | 10 | 12 | 4 | 27 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 |
| Race/Ethnicity, Customized(Participants) | Cohort 1: 6.0E13 Vector Genomes (vg) - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 6 to <24 Months | Cohort 2: 1.2E14 vg - Age 24 to <60 Months | Cohort 3: 2.4E14 vg - Age 6 to <24 Months | Total |
|---|---|---|---|---|---|
| White | 2 | 10 | 8 | 3 | 23 |
| Asian | 0 | 1 | 4 | 1 | 6 |
| Other | 0 | 1 | 0 | 0 | 1 |
| Multiple | 1 | 1 | 0 | 0 | 2 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Dec 2017 to primary completion in Nov 2021, 47 months.
Results were first posted in Feb 2023, 1 year 2 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Jan 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Onasemnogene abeparvovec