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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-003474-31 | EudraCT Number |
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This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never ambulatory SMA patients 2 to <18 years of age.
Eligible participants received a single administration of OAV101B at the dose of 1.2 x 10^14 vector genomes intrathecally or the sham procedure on Day 1 (Treatment Period 1), and were followed for a period of 52 weeks for Period 1. In Period 2, participants who received the sham treatment in Period 1 were administered OAV101B, and participants who received OAV101B in Period 1 underwent the sham procedure. Participants were followed up for 12 weeks in Period 2.
The study consisted of a Screening and Baseline Period followed by two Treatment and Follow-up Periods. Participants were admitted to the hospital on Day 1 (or Day -1 as per local standards of care). After receiving OAV101B or the sham procedure on Day 1, participants underwent in-patient safety monitoring through Day 2 and optionally for Day 3.
After Period 1, eligible participants could continue to Period 2 subsequently entering Period 2 in a rolling seamless fashion as participants completed Follow-up Period 1. In Treatment Period 2, eligible participants who received a sham procedure on Study Day 1 of Treatment Period 1 were hospitalized to receive OAV101B on Week 52 + 1 day and participants who received OAV101B on Study Day 1 of Treatment Period 1 were hospitalized to receive a sham procedure on the Week 52 + 1 Day. The total duration of the study including both Period 1 and Period 2 was 64 weeks. At the end of the study, all participants who received OAV101B were eligible to enroll in a long-term follow-up study to monitor long-term safety and efficacy.
Approximately 125 participants were planned to be randomized in a 3:2 ratio to receive OAV101B (N= ~75) or a sham procedure (N= ~50). The unequal randomization ratio allowed more participants to receive active treatment in Period 1. It was anticipated that approximately 65 randomized participants would be aged 2 to <5 years and approximately 60 randomized participants would be aged 5 to <18 years.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| OAV101 in Treatment Period 1; Sham Control in Treatment Period 2 | Experimental | OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10^14 vector genomes (vg) in Treatment Period 1; Sham Control in Treatment Period 2 (Week 52 +1 day). |
|
| Sham control in Treatment Period 1; OAV101 in Treatment Period 2 | Sham Comparator | A skin prick in the lumbar region in Treat Period 1; OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10^14 vector genomes (vg) in Treatment Period 2 (Week 52 +1 day) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| OAV101 | Genetic | Gene therapy |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline at the End of Period 1 in the Hammersmith Functional Motor Scale Expanded - Total Score - in the ≥ 2 to < 18 Years Age Group | The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability. | Baseline, Week 52 (or Week 48) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in HFMSE Total Score at the End of Follow-up Period 1 in Treated Patients Compared to Sham Controls in the ≥ 2 to < 5 Years Age Group | The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability. |
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Key Inclusion criteria:
Key Exclusion criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Connecticut Children's Medical Center | Farmington | Connecticut | 06032 | United States | ||
| Ann & Robert H. Lurie Children's Hospital of Chicago |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41360993 | Derived | Proud CM, Vu DC, Wilmshurst JM, Sanmaneechai O, Gulati S, Xiong H, Moreno HC, Tay SKH, Thong MK, Born AP, Banzzatto Ortega A, Jong YJ, Al-Muhaizea MA, Lee AW, Visootsak J, Tauscher-Wisniewski S, Alecu I, Parlikar R, Finkel RS; STEER Study Group. Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial. Nat Med. 2026 Feb;32(2):481-487. doi: 10.1038/s41591-025-04103-w. Epub 2025 Dec 8. |
| Label | URL |
|---|---|
| Results for COAV101B12301 from the Novartis Clinical Trials Website | View source |
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Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.
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121 total participants were treated with a single dose of OAV101B (either in Period 1 (even if they did not complete Period 1) or Period 2).
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| ID | Title | Description |
|---|---|---|
| FG000 | OAV101 First, Then Sham Control | OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10^14 vector genomes (vg) in Period 1, then a skin prick in the lumbar region without any medication in Period 2 |
| FG001 | Sham Control First, Then OAV101 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Period 1 - First Intervention (52 weeks) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 17, 2024 | Oct 27, 2025 |
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A participant will receive a single, one-time dose of OAV101.
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| Sham control | Procedure | The sham procedure will consist of a small needle prick on the lower back at the location where the LP injection is normally made. The needle will break the skin, but no needle insertion for lumbar puncture will occur. |
|
| Baseline, Week 52 (or Week 48) |
| Change From Baseline in Revised Upper Limb Module (RULM) Total Score at the End of Follow-up Period 1 in Treated Patients Compared to Sham Controls in the ≥ 2 to < 18 Years Age Group | The RULM is a validated SMA specific assessment of motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. The scale consists of 19 scorable items: 18 items scored on 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). Total scores range from 0-37 points. Higher scores reflect higher level of motor ability. | Baseline, Week 52 (or Week 48) |
| Change From Baseline in the RULM Total Score at the End of Follow-up Period 1 in Treated Patients Compared to Sham Controls in the ≥ 2 to < 5 Years Age Group | The RULM is a validated SMA specific assessment of motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. The scale consists of 19 scorable items: 18 items scored on a 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). Total scores range from 0-37 points. Higher scores reflect higher level of motor ability. | Baseline, Week 52 (or Week 48) |
| % of Participants Who Achieved at Least a 3-point Improvement From Baseline in HFMSE Total Score at the End of Follow-up Period 1 in the ≥ 2 to < 18 Years Age Group | The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability. | Baseline, Week 52 (or Week 48) (end of Period 1) |
| % of Participants Who Achieved at Least a 3-point Improvement From Baseline in HFMSE Total Score at the End of Follow-up Period 1 for Participants Aged ≥ 2 to < 5 Years | The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability. | Baseline, Week 52 (or Week 48)(end of Period 1) |
| Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. A Treatment Emergent Adverse Event (TEAE) is defined as an event that emerges during treatment, having been absent pretreatment, or worsens relative to the pretreatment state. The occurrence of AEs must be sought by non-directive questioning of the participant at each visit during the study. Adverse events also may be detected when they are volunteered by the participant during or between visits or through physical examination findings, laboratory test findings, or other assessments. pt = participant pts = participants | Adverse events are reported from the start of treatment period 1 plus 64 weeks, up to a maximum time period of 64 weeks. |
| Number of Participants With Adverse Events of Special Interest (AESI) | An AESI is primarily defined by using standard Medical Dictionary for Regulatory Activities (MedDRA) queries, and identified as follows:
Adverse events for Periods 1 and 2 are combined in the overall OAV101 arm because the same active treatment (and same single dose) was administered in either Period1 or Period 2. Because this is a gene therapy, which permanently impacts the genetics of the study participant, participants randomized to OAV101 in Period 1 are still considered on treatment with OAV101 in Period 2 (after receiving sham control in Period 2). Therefore, adverse events for the Sham arm can only be considered from Period 1. | Adverse events are reported from the start of treatment period 1 plus 64 weeks, up to a maximum time period of 64 weeks. |
| Number (and Percentage) of Patients With Intracardiac Thrombi | Intracardiac thrombi is defined as the presence of thrombus on post-baseline echocardiograms. Events for Periods 1 and 2 are combined in the overall OAV101 arm because the same active treatment (and same single dose) was administered in either Period1 or Period 2. Because this is a gene therapy, which permanently impacts the genetics of the study participant, participants randomized to OAV101 in Period 1 are still considered on treatment with OAV101 in Period 2 (after receiving sham control in Period 2). Therefore, events for the Sham arm can only be considered from Period 1. | Baseline up to 64 weeks |
| Number(and Percentage) of Patients With Low Cardiac Function | Low cardiac function is defined as left ventricular ejection fraction <56% or left ventricular fractional shortening <28% on post-baseline echocardigrams. Events for Periods 1 and 2 are combined in the overall OAV101 arm because the same active treatment (and same single dose) was administered in either Period1 or Period 2. Because this is a gene therapy, which permanently impacts the genetics of the study participant, participants randomized to OAV101 in Period 1 are still considered on treatment with OAV101 in Period 2 (after receiving sham control in Period 2). Therefore, events for the Sham arm can only be considered from Period 1. | Baseline up to 64 weeks |
| Chicago |
| Illinois |
| 60611 |
| United States |
| Clinic for Special Children | Strasburg | Pennsylvania | 17579 | United States |
| St Jude Children's Research Hospital | Memphis | Tennessee | 38105 | United States |
| Child Hosp Of The Kings Daughters | Norfolk | Virginia | 23507 | United States |
| Children's Specialty Group/CHKD | Norfolk | Virginia | 23507 | United States |
| Novartis Investigative Site | Curitiba | Paraná | 81520-060 | Brazil |
| Novartis Investigative Site | São Paulo | São Paulo | 05403 000 | Brazil |
| Novartis Investigative Site | Beijing | Beijing Municipality | 100034 | China |
| Novartis Investigative Site | Chongqing | Chongqing Municipality | 400010 | China |
| Novartis Investigative Site | Guangzhou | Guangdong | 510623 | China |
| Novartis Investigative Site | Shenzhen | Guangdong | 518034 | China |
| Novartis Investigative Site | Chengdu | Sichuan | 610041 | China |
| Novartis Investigative Site | Hangzhou | Zhejiang | 310052 | China |
| Peking University First Hospital | Beijing | 100034 | China |
| Novartis Investigative Site | Beijing | 100069 | China |
| Novartis Investigative Site | Beijing | 100730 | China |
| Novartis Investigative Site | Copenhagen | 2100 O | Denmark |
| Paediatric Neurology | Copenhagen | 2100 O | Denmark |
| Sir Ganga Ram Hospital | New Delhi | National Capital Territory of Delhi | 110 060 | India |
| Novartis Investigative Site | New Delhi | National Capital Territory of Delhi | 110029 | India |
| Novartis Investigative Site | New Delhi | National Capital Territory of Delhi | 110060 | India |
| AIIMS, Ansari Nagar | New Delhi | New Delhi | 110029 | India |
| Novartis Investigative Site | Kolkata | West Bengal | 700094 | India |
| Novartis Investigative Site | Hyderabad | 500034 | India |
| Novartis Investigative Site | Mumbai | 400016 | India |
| P.D. Hinduja National Hospital & MRC | Mumbai | 400016 | India |
| Novartis Investigative Site | Kuala Lumpur | 50300 | Malaysia |
| Novartis Investigative Site | Kuala Lumpur | 59100 | Malaysia |
| Hospital Civil De Guadalajara Fray Antonio Alcalde | Guadalajara | Jalisco | 44280 | Mexico |
| Novartis Investigative Site | Guadalajara | Jalisco | 44280 | Mexico |
| Novartis Investigative Site | Mexico City | Mexico City | 06720 | Mexico |
| Novartis Investigative Site | Riyadh | 11211 | Saudi Arabia |
| Novartis Investigative Site | Singapore | 119074 | Singapore |
| Novartis Investigative Site | Cape Town | 7925 | South Africa |
| Red Cross War Memorial Childrens Hospital | Cape Town | 7925 | South Africa |
| Kaohsiung Medical University Hospital | Kaohsiung City | 80756 | Taiwan |
| Novartis Investigative Site | Kaohsiung City | 80756 | Taiwan |
| Novartis Investigative Site | Bangkok | 10700 | Thailand |
| Siriraj Hospital | Bangkok | 10700 | Thailand |
| National Children's Hospital | Hanoi | 100000 | Vietnam |
| Novartis Investigative Site | Hanoi | 100000 | Vietnam |
| A Plain Language Trial Summary is available on www.novctrd.com | View source |
| A Pediatric Plain Language Trial Summary is available on www.novctrd.com | View source |
A skin prick in the lumbar region without any medication in Period 1, then OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10^14 vector genomes (vg) in Period 2 |
| Patients completed follow-up period 1 |
|
| Patients completed follow-up period 1 but did not enter treatment period 2 |
|
| Patient who completed Week 48 but not Week 52 |
|
| Primary reason for not entering treatment period 2 | Protocol-specified withdrawal criterion met |
|
| COMPLETED | Patients completed follow-up period 1 and entered treatment period 2 |
|
| NOT COMPLETED |
|
|
| Period 2 - 2nd Intervention (12 weeks) |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | OAV101 First, Then Sham Control | OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10^14 vector genomes (vg) in Period 1, then a skin prick in the lumbar region without any medication in Period 2 |
| BG001 | Sham Control First, Then OAV101 | A skin prick in the lumbar region without any medication in Period 1, then OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10^14 vector genomes (vg) in Period 2 |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline at the End of Period 1 in the Hammersmith Functional Motor Scale Expanded - Total Score - in the ≥ 2 to < 18 Years Age Group | The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability. | Full Analysis Set - all participants who were treated in treatment period 1 with a valid measurement (including 1 pt who dropped out between Week 48 and week 52; The data collected at Week 48 are included in this table.) (Participants who early terminated before Week 48 are not included). | Posted | Least Squares Mean | Standard Error | Scores on a scale | Baseline, Week 52 (or Week 48) |
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| Secondary | Change From Baseline in HFMSE Total Score at the End of Follow-up Period 1 in Treated Patients Compared to Sham Controls in the ≥ 2 to < 5 Years Age Group | The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability. | Full Analysis Set - all participants who were treated in treatment period 1 with a valid measurement in the 2 to <5 years age group. (Participants who early terminated before Week 48 are not included.) | Posted | Least Squares Mean | Standard Error | Scores on a scale | Baseline, Week 52 (or Week 48) |
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| Secondary | Change From Baseline in Revised Upper Limb Module (RULM) Total Score at the End of Follow-up Period 1 in Treated Patients Compared to Sham Controls in the ≥ 2 to < 18 Years Age Group | The RULM is a validated SMA specific assessment of motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. The scale consists of 19 scorable items: 18 items scored on 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). Total scores range from 0-37 points. Higher scores reflect higher level of motor ability. | Full Analysis Set - all participants who were treated in treatment period 1 with a valid measurement (including 1 pt who dropped out between Week 48 and week 52; The data collected at Week 48 are included in this table.) (Participants who early terminated before Week 48 are not included). | Posted | Least Squares Mean | Standard Error | Scores on a scale | Baseline, Week 52 (or Week 48) |
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| Secondary | Change From Baseline in the RULM Total Score at the End of Follow-up Period 1 in Treated Patients Compared to Sham Controls in the ≥ 2 to < 5 Years Age Group | The RULM is a validated SMA specific assessment of motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. The scale consists of 19 scorable items: 18 items scored on a 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). Total scores range from 0-37 points. Higher scores reflect higher level of motor ability. | Full Analysis Set - all participants who were treated in treatment period 1 with a valid measurement in the 2 to <5 years age group. (Participants who early terminated before Week 48 are not included.) | Posted | Least Squares Mean | Standard Error | Scores on a scale | Baseline, Week 52 (or Week 48) |
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| Secondary | % of Participants Who Achieved at Least a 3-point Improvement From Baseline in HFMSE Total Score at the End of Follow-up Period 1 in the ≥ 2 to < 18 Years Age Group | The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability. | Full Analysis Set - all participants who were treated in treatment period 1 with a valid measurement (including 1 pt who dropped out between Week 48 and week 52; The data collected at Week 48 are included in this table.) (Participants who early terminated before Week 48 are not included). | Posted | Number | 95% Confidence Interval | % of participants | Baseline, Week 52 (or Week 48) (end of Period 1) |
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| Secondary | % of Participants Who Achieved at Least a 3-point Improvement From Baseline in HFMSE Total Score at the End of Follow-up Period 1 for Participants Aged ≥ 2 to < 5 Years | The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability. | Full Analysis Set - all participants who were treated in treatment period 1 with a valid measurement in the 2 to <5 years age group. (Participants who early terminated before Week 48 are not included.) | Posted | Number | 95% Confidence Interval | % of participants | Baseline, Week 52 (or Week 48)(end of Period 1) |
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| Secondary | Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. A Treatment Emergent Adverse Event (TEAE) is defined as an event that emerges during treatment, having been absent pretreatment, or worsens relative to the pretreatment state. The occurrence of AEs must be sought by non-directive questioning of the participant at each visit during the study. Adverse events also may be detected when they are volunteered by the participant during or between visits or through physical examination findings, laboratory test findings, or other assessments. pt = participant pts = participants | Safety analysis set - all treated pts. AEs for Periods 1 and 2 are combined in the overall OAV101 arm because the same active treatment (and same single dose) was administered in either Period1 or 2. Because this is a gene therapy, which permanently impacts the genetics of the study pt, pts randomized to OAV101 in Period 1 are still considered on treatment with OAV101 in Period 2 (after receiving sham control in Period 2). Therefore, AEs for the Sham arm can only be considered from Period 1. | Posted | Count of Participants | Participants | Adverse events are reported from the start of treatment period 1 plus 64 weeks, up to a maximum time period of 64 weeks. |
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| Secondary | Number of Participants With Adverse Events of Special Interest (AESI) | An AESI is primarily defined by using standard Medical Dictionary for Regulatory Activities (MedDRA) queries, and identified as follows:
Adverse events for Periods 1 and 2 are combined in the overall OAV101 arm because the same active treatment (and same single dose) was administered in either Period1 or Period 2. Because this is a gene therapy, which permanently impacts the genetics of the study participant, participants randomized to OAV101 in Period 1 are still considered on treatment with OAV101 in Period 2 (after receiving sham control in Period 2). Therefore, adverse events for the Sham arm can only be considered from Period 1. | Safety analysis set - all treated participants. | Posted | Count of Participants | Participants | Adverse events are reported from the start of treatment period 1 plus 64 weeks, up to a maximum time period of 64 weeks. |
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| Secondary | Number (and Percentage) of Patients With Intracardiac Thrombi | Intracardiac thrombi is defined as the presence of thrombus on post-baseline echocardiograms. Events for Periods 1 and 2 are combined in the overall OAV101 arm because the same active treatment (and same single dose) was administered in either Period1 or Period 2. Because this is a gene therapy, which permanently impacts the genetics of the study participant, participants randomized to OAV101 in Period 1 are still considered on treatment with OAV101 in Period 2 (after receiving sham control in Period 2). Therefore, events for the Sham arm can only be considered from Period 1. | Safety analysis set - all treated participants. | Posted | Count of Participants | Participants | Baseline up to 64 weeks |
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| Secondary | Number(and Percentage) of Patients With Low Cardiac Function | Low cardiac function is defined as left ventricular ejection fraction <56% or left ventricular fractional shortening <28% on post-baseline echocardigrams. Events for Periods 1 and 2 are combined in the overall OAV101 arm because the same active treatment (and same single dose) was administered in either Period1 or Period 2. Because this is a gene therapy, which permanently impacts the genetics of the study participant, participants randomized to OAV101 in Period 1 are still considered on treatment with OAV101 in Period 2 (after receiving sham control in Period 2). Therefore, events for the Sham arm can only be considered from Period 1. | Safety analysis set - all treated participants. | Posted | Count of Participants | Participants | Baseline up to 64 weeks |
|
Adverse events (AEs) are reported from the start of treatment period 1 plus 64 weeks, up to a maximum time period of 64 weeks.
AEs for Periods 1 and 2 are combined in the overall OAV101 arm because the same active treatment (and same single dose) was administered in either Period1 or Period 2. Because this is a gene therapy, which permanently impacts the genetics of the study participant, participants randomized to OAV101 in Period 1 are still considered on treatment with OAV101 in Period 2 (after receiving sham control in Period 2). Therefore, AEs for the Sham arm can only be considered from Period 1.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | OAV101 Period 1 | OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10^14 vector genomes (vg) in Period 1 | 0 | 75 | 21 | 75 | 63 | 75 |
| EG001 | Sham Control Period 1 | A skin prick in the lumbar region without any medication in Period 1 | 0 | 51 | 17 | 51 | 43 | 51 |
| EG002 | Overall OAV101 in Periods 1 and 2 | OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10^14 vector genomes (vg). -For participants randomized to OAV101B in Period 1: All AEs from Period 1 and 2 -For participants randomized to the sham control in Period 1 and who were administered OAV101 in Period 2: All AEs from Period 2 | 0 | 121 | 34 | 121 | 85 | 121 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Arrhythmia | Cardiac disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Gastrointestinal disorder | Gastrointestinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Pyrexia | General disorders and administration site conditions | MedDRA (28.0) | Systematic Assessment |
| |
| Hepatitis | Hepatobiliary disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Bronchitis viral | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Coronavirus pneumonia | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Metapneumovirus infection | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Mycoplasma infection | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Pneumonia mycoplasmal | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Pneumonia viral | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Musculoskeletal discomfort | Musculoskeletal and connective tissue disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Peripheral sensory neuropathy | Nervous system disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Bronchiectasis | Respiratory, thoracic and mediastinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA (28.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Constipation | Gastrointestinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Pyrexia | General disorders and administration site conditions | MedDRA (28.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (28.0) | Systematic Assessment |
| |
| Osteoporosis | Musculoskeletal and connective tissue disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Respiratory tract inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (28.0) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (28.0) | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | + 1 862 778 8300 | Novartis.email@Novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 28, 2024 | Oct 27, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D014897 | Spinal Muscular Atrophies of Childhood |
| D009133 | Muscular Atrophy |
| D009134 | Muscular Atrophy, Spinal |
| D009135 | Muscular Diseases |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D016472 | Motor Neuron Disease |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020879 | Neuromuscular Manifestations |
| D009461 | Neurologic Manifestations |
| D001284 | Atrophy |
| D020763 | Pathological Conditions, Anatomical |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012816 | Signs and Symptoms |
| D009140 | Musculoskeletal Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000710948 | Zolgensma |
Not provided
Not provided
Not provided
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Units | Counts |
|---|---|
| Participants |
|
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|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| OG001 | Sham Control Period 1 | A skin prick in the lumbar region without any medication in Period 1 |
| OG002 | Overall OAV101 in Periods 1 and 2 | OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10^14 vector genomes (vg).
|
|
|
| OG002 |
| Overall OAV101 in Periods 1 and 2 |
OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10^14 vector genomes (vg).
|
|
|
|
|
|
|