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This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to <18 years after the discontinuation of treatment with nusinersen or risdiplam. The study aimed to enroll approximately 28 participants across each of 2 age brackets (2 to <6 years, and 6 to <18 years).
Eligible participants received a single OAV101B administration of 1.2x1014 vector genomes on Day 1 (Treatment period) and were followed for a period of 52 weeks.
Participants were admitted to the hospital on Day -1 for pre-treatment baseline procedures. After receiving OAV101B on Day 1, participants underwent in-patient safety monitoring over the next 48 hours, after which the participant could be discharged, based on Investigator judgment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| OAV-101 | Experimental | Intrathecal administration of OAV101 at a dose of 1.2 x 10^14 vector genomes, one time dose |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| OAV101 | Genetic | Intrathecal administration of OAV101 at a dose of 1.2 x 10^14 vector genomes, one time dose |
|
| Measure | Description | Time Frame |
|---|---|---|
| Overview of Treatment-emergent Adverse Events by Age Subgroup | 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. 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. | Adverse events were reported from single dose of study treatment plus 52 weeks, up to a maximum time period of 52 weeks. |
| Treatment-emergent Adverse Events Related to Treatment by System Organ Class, Preferred Term, Age Subgroup (>= 10%) | 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. 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. | Adverse events were reported from single dose of study treatment plus 52 weeks, up to a maximum time period of 52 weeks. |
| Adverse Events of Special Interest by System Organ Class, Preferred Term, Age Subgroup | 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. 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. An adverse event of special interest (AESI) is primarily defined by using standard Medical Dictionary for Regulatory Activities (MedDRA) queries, and identified as follows: Hepatotoxicity, Transient thrombocytopenia, Thrombotic microangiopathy, Cardiac adverse events, signs and symptoms that may be suggestive dorsal root ganglia toxicity, and new malignancies. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline at Week 52 Visit in the HFMSE Total Score - Mean (SD) | The Hammersmith Functional Motor Scale Expanded (HFMSE) is a SMA-specific 33-item assessment that is administered by qualified clinical evaluators. Each motor skill item is scored on a 3-point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher ability level. |
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Inclusion Criteria
Exclusion Criteria:
Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer using an immunoassay is reported as elevated
Clinically significant abnormalities in test results during screening
Contraindications for lumbar puncture procedure
At Baseline, participants are excluded if they received:
Vaccinations 2 weeks prior to administration of OAV101
Hospitalization for a pulmonary event, or for nutritional support within 2 months prior to Screening or inpatient major surgery planned.
Presence of an infection or febrile illness up to 30 days prior to administration of OAV101
Requiring invasive ventilation
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Boston Childrens Hospital | Boston | Massachusetts | 02215 | United States | ||
| Child Hosp Of The Kings Daughters |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41360995 | Derived | Kwon JM, Munell F, Le Goff L, Yuge K, Kato T, Cances C, De Waele L, Woodcock IR, Mercuri EM, Proud CM, Darras BT, Hayes LH, Oskoui M, Visootsak J, Williams G, Ilic A, Yang L, van der Pol WL. Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial. Nat Med. 2026 Feb;32(2):488-493. doi: 10.1038/s41591-025-04119-2. Epub 2025 Dec 8. |
| Label | URL |
|---|---|
| A Plain Language Trial Summary is available on www.novctrd.com | 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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| ID | Title | Description |
|---|---|---|
| FG000 | OAV101 1.2x1014 vg - All Participants | Intrathecal administration of OAV101 at a dose of 1.2 x 10^14 vector genomes, one time dose |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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, 2023 | May 15, 2025 |
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| Adverse events were reported from single dose of study treatment plus 52 weeks, up to a maximum time period of 52 weeks. |
| Baseline, Week 52 |
| Change From Baseline at Week 52 Visit in the HFMSE Total Score - LS Means | The Hammersmith Functional Motor Scale Expanded (HFMSE) is a SMA-specific 33-item assessment that is administered by qualified clinical evaluators. Each motor skill item is scored on a 3-point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher ability level. | Baseline, Week 52 |
| Change From Baseline at Week 52 Visit in the RULM Total Score - Mean (SD) | The Revised Upper Limb Model (RULM) is a validated, SMA-specific assessment that measures motor performance in the upper limbs from childhood through adulthood in ambulatory and never ambulatory individuals with SMA. The revised version of the test 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). These item scores are summed to give a total score ranging from 0 to 37 points with lower scores reflecting poorer ability. | Baseline, Week 52 |
| Change From Baseline at Week 52 Visit in the RULM Total Score - LS Means | The Revised Upper Limb Model (RULM) is a validated, SMA-specific assessment that measures motor performance in the upper limbs from childhood through adulthood in ambulatory and never ambulatory individuals with SMA. The revised version of the test 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). These item scores are summed to give a total score ranging from 0 to 37 points with lower scores reflecting poorer ability. | Baseline, Week 52 |
| Change From Baseline at Week 52 Visit in Assessment of Caregiver Experience in ACEND Instrument Score - Mean (SD) | The Assessment of Caregiver Experience in Neuromuscular Disease (ACEND) instrument quantifies the caregiver impact experienced by parents/caregivers of children affected with severe neuromuscular diseases, including children with SMA. The total score is on a scale of 0 to 100 with a higher score indicating that caregivers experienced less intense caregiving impact. | Baseline, Week 52 |
| Change From Baseline at Week 52 Visit in Assessment of Caregiver Experience in ACEND Instrument Score - LS Means | The Assessment of Caregiver Experience in Neuromuscular Disease (ACEND) instrument quantifies the caregiver impact experienced by parents/caregivers of children affected with severe neuromuscular diseases, including children with SMA. The total score is on a scale of 0 to 100 with a higher score indicating that caregivers experienced less intense caregiving impact. | Baseline, Week 52 |
| Norfolk |
| Virginia |
| 23507 |
| United States |
| University of Wisconsin Madison Medical School | Madison | Wisconsin | 53792-7375 | United States |
| Novartis Investigative Site | Parkville | Victoria | 3052 | Australia |
| Novartis Investigative Site | Leuven | 3000 | Belgium |
| Novartis Investigative Site | Montreal | Quebec | H4A 3J1 | Canada |
| Novartis Investigative Site | Bron | 69677 | France |
| Novartis Investigative Site | Toulouse | 31059 | France |
| Novartis Investigative Site | Roma | RM | 00168 | Italy |
| Novartis Investigative Site | Kurume | Fukuoka | 830-0011 | Japan |
| Novartis Investigative Site | Shinjuku Ku | Tokyo | 162 8666 | Japan |
| Novartis Investigative Site | Utrecht | 3584 | Netherlands |
| Novartis Investigative Site | Barcelona | Catalonia | 08035 | Spain |
| A Pediatric Plain Language Trial Summary is available on www.novctrd.com | View source |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | OAV101 1.2x1014 vg - All Participants | Intrathecal administration of OAV101 at a dose of 1.2 x 10^14 vector genomes, one time dose |
| 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 | Overview of Treatment-emergent Adverse Events by Age Subgroup | 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. 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. | Full analysis set - all treated participants | Posted | Count of Participants | Participants | Adverse events were reported from single dose of study treatment plus 52 weeks, up to a maximum time period of 52 weeks. |
|
|
| |||||||||||||||||||||||||||||||||||
| Primary | Treatment-emergent Adverse Events Related to Treatment by System Organ Class, Preferred Term, Age Subgroup (>= 10%) | 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. 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. | Full analysis set - all treated participants | Posted | Count of Participants | Participants | Adverse events were reported from single dose of study treatment plus 52 weeks, up to a maximum time period of 52 weeks. |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Adverse Events of Special Interest by System Organ Class, Preferred Term, Age Subgroup | 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. 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. An adverse event of special interest (AESI) is primarily defined by using standard Medical Dictionary for Regulatory Activities (MedDRA) queries, and identified as follows: Hepatotoxicity, Transient thrombocytopenia, Thrombotic microangiopathy, Cardiac adverse events, signs and symptoms that may be suggestive dorsal root ganglia toxicity, and new malignancies. | Full analysis set - all treated participants | Posted | Count of Participants | Participants | Adverse events were reported from single dose of study treatment plus 52 weeks, up to a maximum time period of 52 weeks. |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Week 52 Visit in the HFMSE Total Score - Mean (SD) | The Hammersmith Functional Motor Scale Expanded (HFMSE) is a SMA-specific 33-item assessment that is administered by qualified clinical evaluators. Each motor skill item is scored on a 3-point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher ability level. | Full analysis set - for all treated participants with a valid measurement without a protocol deviation with impact | Posted | Mean | Standard Deviation | scores on a scale | Baseline, Week 52 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Week 52 Visit in the HFMSE Total Score - LS Means | The Hammersmith Functional Motor Scale Expanded (HFMSE) is a SMA-specific 33-item assessment that is administered by qualified clinical evaluators. Each motor skill item is scored on a 3-point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher ability level. | Full analysis set - for all treated participants with a valid measurement without a protocol deviation with impact | Posted | Least Squares Mean | 95% Confidence Interval | scores on a scale | Baseline, Week 52 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Week 52 Visit in the RULM Total Score - Mean (SD) | The Revised Upper Limb Model (RULM) is a validated, SMA-specific assessment that measures motor performance in the upper limbs from childhood through adulthood in ambulatory and never ambulatory individuals with SMA. The revised version of the test 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). These item scores are summed to give a total score ranging from 0 to 37 points with lower scores reflecting poorer ability. | Full analysis set - for all treated participants with a valid measurement without a protocol deviation with impact | Posted | Mean | Standard Deviation | scores on a scale | Baseline, Week 52 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Week 52 Visit in the RULM Total Score - LS Means | The Revised Upper Limb Model (RULM) is a validated, SMA-specific assessment that measures motor performance in the upper limbs from childhood through adulthood in ambulatory and never ambulatory individuals with SMA. The revised version of the test 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). These item scores are summed to give a total score ranging from 0 to 37 points with lower scores reflecting poorer ability. | Full analysis set - for all treated participants with a valid measurement without a protocol deviation with impact | Posted | Least Squares Mean | 95% Confidence Interval | scores on a scale | Baseline, Week 52 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Week 52 Visit in Assessment of Caregiver Experience in ACEND Instrument Score - Mean (SD) | The Assessment of Caregiver Experience in Neuromuscular Disease (ACEND) instrument quantifies the caregiver impact experienced by parents/caregivers of children affected with severe neuromuscular diseases, including children with SMA. The total score is on a scale of 0 to 100 with a higher score indicating that caregivers experienced less intense caregiving impact. | Full analysis set - for all treated participants with a valid measurement without a protocol deviation with impact | Posted | Mean | Standard Deviation | scores on a scale | Baseline, Week 52 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline at Week 52 Visit in Assessment of Caregiver Experience in ACEND Instrument Score - LS Means | The Assessment of Caregiver Experience in Neuromuscular Disease (ACEND) instrument quantifies the caregiver impact experienced by parents/caregivers of children affected with severe neuromuscular diseases, including children with SMA. The total score is on a scale of 0 to 100 with a higher score indicating that caregivers experienced less intense caregiving impact. | Full analysis set - for all treated participants with a valid measurement without a protocol deviation with impact | Posted | Least Squares Mean | 95% Confidence Interval | scores on a scale | Baseline, Week 52 |
|
|
Adverse events were reported from single dose of study treatment plus 52 weeks, up to a maximum time period of 52 weeks.
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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 1.2x1014 vg - All Participants | Intrathecal administration of OAV101 at a dose of 1.2 x 10^14 vector genomes, one time dose | 0 | 27 | 4 | 27 | 27 | 27 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cyclic vomiting syndrome | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Pyrexia | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| Bronchitis viral | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Influenza virus test positive | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Lung disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Fatigue | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| Pyrexia | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| Swelling face | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Increased appetite | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Scoliosis | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Affect lability | Psychiatric disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Initial insomnia | Psychiatric disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | 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 | Dec 5, 2024 | May 15, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| D009133 | Muscular Atrophy |
| D055534 | Bulbo-Spinal Atrophy, X-Linked |
| D009135 | Muscular Diseases |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |
| D020879 | Neuromuscular Manifestations |
| D009461 | Neurologic Manifestations |
| D001284 | Atrophy |
| D020763 | Pathological Conditions, Anatomical |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012816 | Signs and Symptoms |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D009140 | Musculoskeletal Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000710948 | Zolgensma |
Not provided
Not provided
Not provided
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
| Any serious treatment-emergent adverse event related to study treatment |
|
| Any severe treatment-emergent adverse event |
|
| Any treatment-emergent adverse event leading to study discontinuation |
|
| Any treatment-emergent adverse event leading to death |
|
| Any treatment-emergent adverse event of special interest |
|
|
| Participants |
|
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