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NCT06178393
Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States
trial in Spinal Muscular Atrophy in 162 participants. Completed in 30 December 2022.
30 December 2022
Quick facts
| Lead sponsor | Novartis Pharmaceuticals |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 162 |
| Start date | 5 May 2022 |
| Primary completion | 30 December 2022 |
| Estimated completion | 30 December 2022 |
| Sites | 1 location across United States |
Conditions studied
- Spinal Muscular Atrophy — all drugs for Spinal Muscular Atrophy →
Sponsor
Novartis Pharmaceuticals — full company profile →
Who can join
Under 5, any sex, with Spinal Muscular Atrophy. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This global, retrospective, non-interventional, medical chart review (MCR), descriptive study collected patient-level data in regions outside the US. The study required a repeated data collection at follow-up dates from start of treatment with nusinersen, onasemnogene abeparvovec-xioi (OA), and/or risdiplam. At the start of data collection, the study team reached out to the health care providers (HCPs) involved in treating pediatric SMA patients for participating in this study. The physicians across the participating countries conducted a retrospective MCR of pediatric patients diagnosed with SMA who were treated with at least 1of the 3 novel disease-modifying treatments (DMTs): nusinersen, OA, and/or risdiplam. All health care encounters data i.e., emergency and inpatient admissions, surgery, and outpatient consultations of recruited patients, including their treatment with nusinersen, OA, and/or risdiplam, were abstracted to understand the treatment patterns as per routine clinical practice for SMA management globally. The first date of initial administration of 1 of the 3 target drugs was used as the "index date." Based on this, the record abstraction was performed through a retrospective MCR during the pre-index period, at index date and in the post-index period.
Publications & conference data
No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.
Verify or expand the search:
- PubMed search for NCT06178393
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
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Related trials
Other recruiting trials for Spinal Muscular Atrophy
Currently open trials in the same condition.
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- NCT06955897 — Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA · recruiting
- NCT06396325 — A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With · NA · recruiting
- NCT06288230 — An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy · Phase 1, PHASE2 · recruiting
- NCT06321965 — Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy · NA · recruiting
Other Novartis Pharmaceuticals trials
Trials by the same sponsor.
- NCT07498335 — Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Atrasentan in Pediatric Patients With Primary · Phase 3 · not yet recruiting
- NCT07489573 — Study of Efficacy and Safety of Secukinumab in Chinese Adult Patients With Moderate to Severe Hidradenitis Suppurativa · Phase 4 · not yet recruiting
- NCT07484269 — PULSE Registry: for Patients Receiving Lutetium (177Lu) Vipivotide Tetraxetan · not yet recruiting
- NCT07416162 — A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy · not yet recruiting
- NCT07387926 — Safety and Efficacy of Asciminib in Pediatrics and Young Adults With Relapse/Refractory (r/r) Philadelphia Positive (Ph+ · Phase 1, PHASE2 · not yet recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT06178393 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
- Last refreshed: 21 December 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06178393.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing