Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain

NCT06632730 | Completed

• 1 other identifier: COAV101A1ES05
• Study Type: observational
• Target: 69
• Locations: 1 country
• Sites: 1

Brief Summary

An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).

Conditions

Spinal Muscular Atrophy Type I; Spinal Muscular Atrophy Type II

Outcome Measures

Primary Outcomes (3)

• Cost per Year of Use of Resources for Medical Visits

  Medical visits included: * Primary care * Pediatric neurology * Rehabilitation physician * Traumatologist * Nutritionist * Pulmonologist * Cardiologist * Physical therapist * Nursing staff * Phoniatrist * Speech therapist * Rehabilitation sessions * Other specialists * Total medical visits * Hospital emergency room visits * Outpatient emergency room visit * Hospitalizations/Admissions to ward * Hospitalizations due to admissions to the intensive care unit (ICU) * Total hospitalizations (ward and ICU) * Therapy session separately from medical visits

  Up to 12 months

• Costs per Year of Use of Resources for Medical Tests

  Medical tests included: * Blood count * Vitamin D test * Blood creatine kinase test * Electromyogram * Brain magnetic resonance imaging * Muscle magnetic resonance imaging * Bone densitometry * Nocturnal polysomnography * Muscle biopsy * Nerve conduction velocity study * Blood amino acid test * DNA test to confirm diagnosis * Total additional tests

  Up to 12 months

• Cost per Year of Use of Resources for Treatment

  Treatments included: * Nusinersen (loading and maintenance dose) * Total Nusinersen * Risdiplam * Onasemnogene abeparvovec * Total treatments for SMA * Palivizumab * Influenza vaccine * Meningococcal vaccine * High protein shakes * Total adjuvant treatments * Total treatments

  Up to 12 months

Secondary Outcomes (46)

• Number of Patients per Sociodemographic Category

  Baseline

• Sociodemographic: Age

  Baseline

• Sociodemographic: Weight

  Baseline

• Sociodemographic: Height

  Baseline

• Sociodemographic: Body mass index (BMI)

  Baseline

Study Arms (1)

Spinal Muscular Atrophy (SMA) Cohort

Pediatric patients with SMA type I, SMA type II, or presymptomatic SMA.

Eligibility Criteria

• Age: Up to 14 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

This was a retrospective, noninterventional cohort study.

You may qualify if:

• Patients diagnosed with SMA type I or SMA type II.
• Patients with medical records available in the hospital.
• Patients diagnosed with SMA type I or SMA type II after 2017 for whom a minimum of 6 months of follow-up was available.

You may not qualify if:

• Patients who participated in an experimental design study except for those in the long-term follow-up period, during the observation period.

Sponsors & Collaborators

• Novartis: lead

Study Sites (1)

Novartis Pharmaceuticals

East Hanover, New Jersey, 07936, United States

Location

MeSH Terms

Conditions

Spinal Muscular Atrophies of Childhood

Condition Hierarchy (Ancestors)

Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Motor Neuron Disease; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 7, 2024
• First Posted: October 9, 2024
• Study Start: July 5, 2023
• Primary Completion: November 20, 2023
• Study Completion: November 20, 2023
• Last Updated: October 9, 2024

Record last verified: 2024-10

Locations

US (1)