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Virus Therapy

Long-term Follow-up with Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

Phase 3

Waitlist Available

Research Sponsored by Novartis Gene Therapies

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in a Novartis Gene Therapies-sponsored clinical study

Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in an Novartis Gene Therapies, Inc. sponsored clinical study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up year 6 to year 15

Awards & highlights

Pivotal Trial

All Individual Drugs Already Approved

No Placebo-Only Group

Approved for 5 Other Conditions

Summary

This trial is for people with Spinal Muscular Atrophy who have been treated with onasemnogene abeparvovec-xioi in the past. Researchers will monitor safety and efficacy of the treatment long-term.

Who is the study for?

This trial is for patients with Spinal Muscular Atrophy who previously received the gene therapy onasemnogene abeparvovec-xioi in a Novartis-sponsored study. Participants or their guardians must consent to and follow the study's procedures.

What is being tested?

The long-term safety and effectiveness of onasemnogene abeparvovec-xioi, a gene replacement therapy for SMA, are being monitored as participants transition from earlier trials for ongoing evaluation.

What are the potential side effects?

While specific side effects aren't listed here, they may include typical gene therapy-related reactions such as immune response to the vector, potential liver issues, and other unforeseen long-term effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have SMA and was treated with onasemnogene abeparvovec-xioi in a Novartis study.

Select...

I have SMA and was treated with onasemnogene abeparvovec-xioi in a Novartis study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ year 6 to year 15

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~year 6 to year 15

This trial's timeline: 3 weeks for screening, Varies for treatment, and year 6 to year 15 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND)

Change From Baseline in Bayley Scales of Infant and Toddler Development

Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age

+13 more

Side effects data

From 2019 Phase 3 trial • 22 Patients • NCT03306277

55%

Pyrexia

50%

Upper respiratory tract infection

41%

Constipation

41%

Scoliosis

32%

Cough

27%

Respiratory distress

27%

Aspartate aminotransferase increased

23%

Teething

23%

Alanine aminotransferase increased

23%

Respiration abnormal

23%

Rash

23%

Use of accessory respiratory muscles

18%

Gastroesophageal reflux disease

18%

Vomiting

18%

Contusion

18%

Diarrhoea

14%

Arthropod bite

14%

Tachypnoea

14%

Otitis media

14%

Nasal congestion

14%

Dermatitis atopic

14%

Sleep apnoea syndrome

14%

Feeding disorder

14%

Pectus excavatum

14%

Eczema

14%

Conjunctivitis

Respiratory track congestion

Respiratory syncytial virus bronchiolitis

High arched palate

Bronchiolitis

Pneumonia

Gamma-glutamyltransferase increased

Weight decreased

Pericardial effusion

Rhinorrhea

Muscle contractions involuntary

Abdominal distension

Haematochezia

Dermatitis contact

Deformity thorax

Nasopharyngitis

Joint contracture

Diastolic hypertension

Respiratory failure

Upper respiratory tract congestion

Kyphosis

Asphyxiating thoracic dystrophy

Cryptorchism

Thrombocytopenia

Blood creatine phosphokinase MB increased

Lymphocyte count decreased

Urticaria

Dysphagia

Dermatitis diaper

Gastroenteritis

Torticollis

Weight gain poor

Sepsis

Device malfunction

Failure to thrive

Device related infection

Rhinovirus infection

Bacterial tracheitis

Human metapneumovirus test positive

Transaminases increased

Cyanosis

Acute respiratory failure

Hydrocephalus

Atelectasis

Pneumonia aspiration

Respiratory arrest

Abnormal weight gain

Pneumonia bacterial

100%

80%

60%

40%

20%

Study treatment Arm

Onasemnogene Abeparvovec-xioi

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioiExperimental Treatment1 Intervention

Participants received treatment with IV onasemnogene abeparvovec-xioi in an onasemnogene abeparvovec-xioi or received treatment with IT onasemnogene abeparvovec-xioi in an onasemnogene.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Onasemnogene abeparvovec

FDA approved

0 / 2Eligibility criteria met