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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-002611-26 | EudraCT Number | ||
| 205305 | Other Identifier | JapicCTI | |
| COAV101A12103 | Other Identifier | Novartis Pharmaceuticals |
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This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi | Other | Participants received treatment with IV onasemnogene abeparvovec-xioi in an onasemnogene abeparvovec-xioi or received treatment with IT onasemnogene abeparvovec-xioi in an onasemnogene. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Onasemnogene Abeparvovec-xioi | Biological | Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec-xioi administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Who Reach Developmental Milestones | Assessed via the developmental milestone checklist, formed of 10 yes/no questions. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone. | Up to 5 years |
| Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score | The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments rated from 0 (unable to perform functional task) to 2 (able to perform functional task unassisted). Higher scores on the total scale of 0-66 indicates higher levels of motor ability. | Up to 5 years |
| Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support | Participants will receive pulmonary assessments by a pulmonologist or appropriate clinician. Respiratory device data will be reviewed for participants receiving non-invasive ventilatory support. | Up to 15 years |
| Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support | Assessed via the swallowing function questionnaire, formed of 4 yes/ no questions and 1 body weight question. | Up to 5 years |
| Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings | The physical examination includes review of the following systems: head, ears, eyes, nose and throat, lungs/thorax, cardiovascular, abdomen, musculoskeletal, neurologic, dermatologic, lymphatic, and genitourinary. In addition, visual inspection of the spine, back, shoulders, and hips looking for spinal curvature and asymmetry will be carried out. Joints will be assessed for loss of mobility and contractures. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Sitra Tauscher-Wisniewski, MD | Novartis Gene Therapies, Inc. | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University Medical Center | Palo Alto | California | 94304 | United States | ||
| Children's Hospital Colorado |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34383289 | Derived | Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12. |
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| Up to 5 years |
| Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements | Vital sign measurements will include blood pressure, respiratory rate, pulse, axillary temperature, and pulse oximetry. | Up to 5 years |
| Change From Baseline in Height Measurements | Up to 5 years |
| Change From Baseline in Weight Measurements | Up to 5 years |
| Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments | Blood samples will be collected for hematology (including complete blood cell count) and chemistry. | Up to 5 years |
| Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments | Cardiac assessments will include a 12-lead electrocardiogram, transthoracic echocardiogram and Troponin-I. | Up to 5 years |
| Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results | The observational phase questionnaire includes 7 yes/no questions. Observation categories include: adverse events, hospitalizations, concomitant medications, ventilatory support and feeding support. | Year 6 to Year 15 |
| Number of Participants Who Experience at Least One Serious Adverse Event (SAE) | An SAE is defined as any adverse event (appearance of [or worsening of any pre existing]) undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria:
| Up to 15 years |
| Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI) | An AESI is defined as an AE occurring during any study phase that fulfills one of the following criteria:
| Up to 15 years |
| Change From Baseline in Bayley Scales of Infant and Toddler Development | Third Edition (Bayley-III) to be performed in all patients up to 42 months, 15 days of age. | Up to 42 months, 15 days of age |
| Change From Baseline in Revised Upper Limb Module (RULM) Score | RULM score is based on a scale from 0 to 37 where lower scores reflect poorer upper limb functional ability. | Up to 5 years |
| Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older | The Cogstate Computerized Cognitive Battery consists of the Identification Test (scored 0 (best) to 1.5708 (worst)), the International Shopping List Test (scored 0 (worst) to 999 (best)), the International Shopping List Test-Delayed Recall (scored 0 (worst) to 999 (best)), the One Card Learning Test (scored 0 (worst) to 1.5708 (best)), and the One Back Test (scored 0 (worst) to 1.5708 (best)). | Up to 5 years |
| Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age | The CELF-5 Following Directions and Sentence Repetition subtests use scoring that varies based on age, but will be administered to participants 5-21 years of age. The Following Directions subtest will be scored from 0-33 with higher score being more advanced and the Recalling Sentences subtest will be scored from 0-78 with higher score being more advanced. | Up to 5 years |
| Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) | ACEND score is based on a scale from 1 to 41 where higher scores represent a better caregiver experience | Up to 5 years |
| Number of Participants With Concomitant Medications Overall and by Type of Medications | Up to 5 years |
| Number of Participants With Other SMA Therapies Overall and by Type of Medications | Year 6 to Year 15 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Ann Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| John Hopkins Hospital - David M. Rubenstein Child Health Building | Baltimore | Maryland | 21287 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Spectrum Health Hospitals Helen DeVos Children's Hospital | Grand Rapids | Michigan | 49503 | United States |
| Washington Unviersity School of Medicine in Saint Louis | St Louis | Missouri | 63110 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| Duke University | Durham | North Carolina | 27713 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Clinic for Special Children | Strasburg | Pennsylvania | 17579 | United States |
| Children's Health Specialty Center Dallas Campus | Dallas | Texas | 75235 | United States |
| University of Utah Health | Salt Lake City | Utah | 84112 | United States |
| Children's Hospital of The King's Daughters | Norfolk | Virginia | 23507 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| University of Wisconsin, Madison | Madison | Wisconsin | 53792 | United States |
| Sydney Children's Hospital | Randwick | New South Wales | 2145 | Australia |
| Universitair Ziekenhuis Gent | Ghent | 9000 | Belgium |
| Centre de Référence des Maladies Neuromusculaires | Liège | B-4000 | Belgium |
| Children's Hospital of Eastern Ontario Research Institute | Ottawa | Ontario | K1H8L1 | Canada |
| Hôpital Armand Trousseau | Paris | 75012 | France |
| Instituto Gianninia Gaslini | Genova | 16147 | Italy |
| Universita Degli Studi Di Milano | Milan | 20122 | Italy |
| Istituto Neurologico di Ricerca | Milan | 20133 | Italy |
| Fondazione Policlinico Universitario Agostino Gemelli | Roma | 00168 | Italy |
| Tokyo Women's Medical University Hospital | Tokyo | 162-8666 | Japan |
| National Taiwan University Hospital | Taipei | 10048 | Taiwan |
| Great Ormond Street Hospital for Children NHS Foundation Trust | London | WC1N 3JH | United Kingdom |
| The Newcastle Upon Tyne Hospitals NHS Foundation Trust | Newcastle upon Tyne | NE1 4LP | United Kingdom |
| ID | Term |
|---|---|
| D014897 | Spinal Muscular Atrophies of Childhood |
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| 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 |
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| ID | Term |
|---|---|
| C000710948 | Zolgensma |
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