---
title: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
nct_id: NCT04851873
overall_status: COMPLETED
phase: PHASE3
sponsor: Novartis Pharmaceuticals
study_type: INTERVENTIONAL
primary_condition: Spinal Muscular Atrophy
countries: United States, Australia, Belgium, Canada, France, Italy, Portugal, Taiwan, United Kingdom
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT04851873.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT04851873"
ct_last_update_post_date: 2024-10-09
last_seen_at: "2026-05-12T07:05:07.085Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

**Official Title:** A Phase IIIb, Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Gene Replacement Therapy With Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

**NCT ID:** [NCT04851873](https://clinicaltrials.gov/study/NCT04851873)

## Key Facts

- **Status:** COMPLETED
- **Phase:** PHASE3
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 24
- **Lead Sponsor:** Novartis Pharmaceuticals
- **Conditions:** Spinal Muscular Atrophy
- **Start Date:** 2021-09-08
- **Completion Date:** 2023-06-13
- **CT.gov Last Update:** 2024-10-09

## Brief Summary

To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.

## Detailed Description

This was an open-label, single arm, multi-center study designed to evaluate the safety, tolerability and efficacy of OAV101 in participants with SMA who weigh ≥ 8.5 kg and ≤ 21 kg. The study aimed to enroll approximately 24 to 30 participants, with approximately 6 to 10 participants across each of 3 weight brackets (8.5 to 13 kg, \>13 to 17 kg, \>17 to 21 kg).

Eligible participants received a single administration of OAV101 at the approved dose of 1.1e14 vg/kg on Day 1 (Treatment period), and were followed for a period of 12 months.

Participants were admitted to the hospital on Day -1 for pre-treatment baseline procedures. After receiving OAV101 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.

After study completion, eligible participants could enroll into a Long Term follow-up study to collect additional safety and efficacy data. (COAV101A12308 (NCT05335876) https://classic.clinicaltrials.gov/ct2/show/NCT05335876?term=COAV101A12308\&draw=2\&rank=1))

## Eligibility

- **Maximum age:** 17 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
Inclusion

* Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor neuron 2 (SMN2) gene.
* Weight ≥ 8.5 kg and ≤ 21 kg at the time of Screening Visit 2
* Naive to treatment or have discontinued an approved drug/therapy

Exclusion:

* Previous OAV101 use or previous use of any adeno-associated virus serotype 9 (AAV9) gene therapy
* BMI \< 3rd percentile
* Participant with history of aspiration pneumonia or signs of aspiration
* Elevated anti-AAV9 antibody
* History of gene therapy, hematopoietic transplantation, or solid organ transplantation
* Inability to take corticosteroids
* Concomitant use of immunosuppressive therapy
* Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation 9. Administration of vaccines 2 weeks prior to infusion of OAV101
* Awake hypoxemia or awake oxygen saturation level decrease
* Hepatic dysfunction
* Presence of a confirmed or suspected infection
* If previously treated with disease modifying therapy, specified washout times apply
* Documented any parental consanguinity.
```

## Arms

- **OAV101** (EXPERIMENTAL) — Participants received a single IV dose administration of OAV101

## Interventions

- **OAV101** (GENETIC) — Gene Therapy - 1.1e14 vector genome (vg)/kg as a one-time IV infusion was administered over approximately 60 minutes.

## Primary Outcomes

- **Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket** _(time frame: Up to Month 12)_ — An 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.
- **Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket** _(time frame: Up to Month 12)_ — Important identified and important potential risks included the following AESIs: Hepatotoxicity, Thrombocytopenia, Cardiac adverse events, Dorsal root ganglia toxicity and Thrombotic microangiopathy.

These were assessed by the investigator.
- **Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure** _(time frame: 12 months)_ — Change from baseline in vital signs measurements - systolic and diastolic blood pressure (mmHg).

Systolic Blood Pressure-Low:\<=5th percentile of the age(Any Age), High:\>=90th percentile of the age, gender, and height group (\<18 yrs).

Diastolic Blood Pressure-High:\>=90th percentile of the age, gender, and height group(\<18 yrs).
- **Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg)** _(time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52)_
- **Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg)** _(time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52)_
- **Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min)** _(time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52)_ — Change from baseline in vital signs measurements - Respiratory Rate (breaths/min)
- **Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min)** _(time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52)_ — Change from baseline in vital signs measurements - Pulse Rate (beats/min
- **Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature** _(time frame: 12 months)_ — Change from baseline in vital signs measurements - temperature (degrees Celsius)

Temperature-Low:\<=35ºC(Any Age),High:\>=38.4ºC(\<18 yrs).
- **Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius)** _(time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52)_
- **Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level** _(time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52)_ — Change from baseline in vital signs measurements - oxygen saturation level (%).

Oxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin (unsaturated+saturated) in the blood and then multiplied by 100.

## Secondary Outcomes

- **Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development.** _(time frame: Baseline, Week 26 and Week 52)_
- **Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age** _(time frame: Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52)_
- **Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age.** _(time frame: Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52)_

## Locations (13)

- Novartis Investigative Site, Boston, Massachusetts, United States
- Novartis Investigative Site, St Louis, Missouri, United States
- Novartis Investigative Site, Randwick, New South Wales, Australia
- Novartis Investigative Site, Leuven, Belgium
- Novartis Investigative Site, Montreal, Quebec, Canada
- Novartis Investigative Site, Garches, France
- Novartis Investigative Site, Strasbourg, France
- Novartis Investigative Site, Roma, RM, Italy
- Novartis Investigative Site, Lisbon, Portugal
- Novartis Investigative Site, Kaohsiung City, Taiwan
- Novartis Investigative Site, Taipei, Taiwan
- Novartis Investigative Site, London, United Kingdom
- Novartis Investigative Site, Newcastle upon Tyne, United Kingdom

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.novartis investigative site|boston|massachusetts|united states` — added _(2026-05-12)_
- `locations.novartis investigative site|st louis|missouri|united states` — added _(2026-05-12)_
- `locations.novartis investigative site|randwick|new south wales|australia` — added _(2026-05-12)_
- `locations.novartis investigative site|leuven||belgium` — added _(2026-05-12)_
- `locations.novartis investigative site|montreal|quebec|canada` — added _(2026-05-12)_
- `locations.novartis investigative site|garches||france` — added _(2026-05-12)_
- `locations.novartis investigative site|strasbourg||france` — added _(2026-05-12)_
- `locations.novartis investigative site|roma|rm|italy` — added _(2026-05-12)_
- `locations.novartis investigative site|lisbon||portugal` — added _(2026-05-12)_
- `locations.novartis investigative site|kaohsiung city||taiwan` — added _(2026-05-12)_
- `locations.novartis investigative site|taipei||taiwan` — added _(2026-05-12)_
- `locations.novartis investigative site|london||united kingdom` — added _(2026-05-12)_
- `locations.novartis investigative site|newcastle upon tyne||united kingdom` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT04851873.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT04851873*  
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