Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are ≥ 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory

ClinicalTrials.gov Identifier: NCT05089656

Novartis Reference Number: COAV101B12301

Last Update: Feb 02, 2023

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

To evaluate the efficacy, safety and tolerability of intrathecal (IT) OAV101 in treatment naive patients with Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 18 years of age over a 15 month trial duration.

Condition 
Type 2 Spinal Muscular Atrophy
Phase 
Phase 3
Overall status 
Recruiting
Start date 
Jan 12, 2022
Completion date 
Oct 21, 2024
Gender 
All
Age(s)
2 Years - 17 Years (Child)

Interventions

Genetic
OAV101
Gene therapy
Procedure
Sham control
The sham procedure will consist of a small needle prick on the lower back at the location where the LP injection is normally made. The needle will break the skin, but no needle insertion for lumbar puncture will occur.

Eligibility Criteria

Key Inclusion criteria:

Diagnostic confirmation during screening period of 5q SMA
The patient must be treatment naive (historical or current use) for all SMN-targeting therapies (e.g., risdiplam (Evrysdi) and nusinersen (Spinraza)).
Onset of clinical signs and symptoms at ≥ 6 months of age
A complete Hammersmith Functional Motor Scale - Expanded (HFMSE) assessment during the screening period for trial eligibility
Able to sit independently at screening, but has never had the ability to walk independently.

Key Exclusion criteria:

Anti-adeno-associated virus serotype 9 (AAV9) antibody titer reported as elevated (reference to > 1:50 or validated result consistent with being elevated) at screening as determined by sponsor designated lab.
Infectious process (e.g. viral, bacterial) or febrile illness prior to start of screening, and up to OAV101 treatment or sham procedure
Hepatic dysfunction (i.e. alanine aminotransferase (ALT), total bilirubin, gamma-glutamyl transferase (GGT) or glutamate dehydrogenase (GLDH), > upper limit of normal (ULN).
Requiring invasive ventilation, awake noninvasive ventilation for > 6 hours during a 24-hour period, noninvasive ventilation for > 12 hours during a 24-hour period or requiring tracheostomy
Complications at screening that would interfere with motor efficacy assessments including but not limited to, severe contractures or Cobb angle > 40 in a sitting position
Surgery for scoliosis or hip fixation in the 12 months prior to Screening or planned within the next 64 weeks
Clinically significant sensory abnormalities in the neurological examination at Screening

Study Locations

United States
Connecticut Children's Medical Center
Recruiting
Farmington, 06032 - Connecticut
Contact: Gyula Acsadi, MD (860-837-7528) - [email protected] - Gyula Acsadi, MD
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Recruiting
Chicago, 60611 - Illinois
Contact: Molly Gallivan (312-227-3813) - [email protected] -
United States
Clinic for Special Children
Recruiting
Strasburg, 17579 - Pennsylvania
Contact: Karlla Brigatti (717-687-9407) - [email protected] -
United States
St Jude Children's Research Hospital
Recruiting
Memphis, 38105 - Tennessee
Contact: Jean Laboe (901-595-1693) - [email protected] -
United States
Children's Specialty Group/CHKD
Recruiting
Norfolk, 23507 - Virginia
Contact: Crystal Proud, M.D. (757-668-5755) - [email protected] - Crystal Proud, M.D.
United States
China
Novartis Investigative Site
Recruiting
Chongqing,
Chongqing
China
Novartis Investigative Site
Recruiting
Guangzhou, 510623
Guangdong
China
Novartis Investigative Site
Recruiting
Shenzhen, 518034
Guangdong
China
Novartis Investigative Site
Recruiting
Chengdu, 610041
Sichuan
China
Novartis Investigative Site
Recruiting
Hangzhou, 310052
Zhejiang
China
Peking University First Hospital
Recruiting
Beijing, 100034
Contact: Hui Xiong (010-83573002) - [email protected] - Hui Xiong
China
Novartis Investigative Site
Recruiting
Beijing, 100069
-
China
Novartis Investigative Site
Recruiting
Beijing, 100730
-
China
Denmark
Paediatric Neurology
Recruiting
Copenhagen, 2100 O
Contact: Alfred Peter Born, MD, Ph.D. (+4535455090) - [email protected] - Alfred Peter Born, MD, Ph.D.
Denmark
India
Sir Ganga Ram Hospital
Recruiting
New Delhi, 110 060 - Delhi
Contact: Ratna Dua Puri, MD (+91 9811869192) - [email protected] - Ratna Dua Puri, MD
India
P.D. Hinduja National Hospital & MRC
Recruiting
Mumbai, 400016
Contact: Neelu Desai, MD (9920614333) - [email protected] - Neelu Desai, MD
India
AIIMS, Ansari Nagar
Recruiting
New Delhi, 110029
Contact: Sheffali Gulati, MD (9810386847) - [email protected] - Professor Sheffali Gulati, MD
India
Malaysia
Novartis Investigative Site
Recruiting
Kuala Lumpur, 50300
-
Malaysia
Novartis Investigative Site
Recruiting
Kuala Lumpur, 59100
-
Malaysia
Mexico
Novartis Investigative Site
Recruiting
Mexico, 06720
Distrito Federal
Mexico
Hospital Civil De Guadalajara Fray Antonio Alcalde
Recruiting
Guadalajara, 44280 - Jalisco
Contact: Javier Macias (+523334411361) - [email protected] -
Mexico
Saudi Arabia
Novartis Investigative Site
Recruiting
Riyadh, 11211
-
Saudi Arabia
Singapore
Novartis Investigative Site
Recruiting
Singapore, 119074
-
Singapore
South Africa
Red Cross War Memorial Childrens Hospital
Recruiting
Cape Town, 7925
Contact: Jaqueline Martin (0815037912) - [email protected] -
South Africa
Taiwan
Kaohsiung Medical University Hospital
Recruiting
Kaohsiung, 80756
Contact: Yun-Hui Chou (+886972977320) - [email protected] -
Taiwan
Thailand
Siriraj Hospital
Recruiting
Bangkok, 10700
Contact: Cheewasan Apirukpanakhet (+66 87 7929516) - [email protected] -
Thailand
Vietnam
National Children's Hospital
Recruiting
Hanoi, 100000
-
Vietnam

Contacts

Name: 
Novartis Gene Therapies Med Info (US, Latin America, Canada)
Name: 
Novartis Gene Therapies Med Info (Europe, Middle East, Africa, Asia-Pacific)

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