A Study to Assess the Efficacy and Safety of IONIS-SMN Rx in Infants With Spinal Muscular Atrophy

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT02193074
First received: July 14, 2014
Last updated: May 6, 2016
Last verified: May 2016
  Purpose
This study will test the Clinical Efficacy and Safety of IONIS-SMN Rx in patients with infantile-onset Spinal Muscular Atrophy.

Condition Intervention Phase
Spinal Muscular Atrophy
Drug: IONIS-SMN Rx
Procedure: Sham procedure
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy

Resource links provided by NLM:


Further study details as provided by Ionis Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Time to death or permanent ventilation [ Time Frame: Up to 13 months ] [ Designated as safety issue: No ]
  • Proportion of motor milestone responders [ Time Frame: Up to 13 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Proportion of CHOP-INTEND responders [ Time Frame: Up to 13 months ] [ Designated as safety issue: No ]
  • Proportion of CMAP responders [ Time Frame: Up to 13 months ] [ Designated as safety issue: No ]
  • Percent of subjects not requiring permanent ventilation [ Time Frame: Up to 13 months ] [ Designated as safety issue: No ]
  • Survival rate [ Time Frame: Up to 13 months ] [ Designated as safety issue: No ]
  • Time to death or permanent ventilation in the subgroups of subjects below the study median disease duration [ Time Frame: Up to 13 months ] [ Designated as safety issue: No ]
  • Time to death or permanent ventilation in the subgroups of subjects above the study median disease duration [ Time Frame: Up to 13 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 111
Study Start Date: July 2014
Estimated Study Completion Date: July 2017
Estimated Primary Completion Date: June 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: IONIS-SMN Rx
Administered by intrathecal injection
Drug: IONIS-SMN Rx
Administered by intrathecal injection
Other Name: ISIS 396443
Sham Comparator: Sham procedure
Small needle prick on the lower back at the location where the IT injection is normally made
Procedure: Sham procedure
Small needle prick on the lower back at the location where the IT injection is normally made

Detailed Description:
This is a Phase 3 multicenter, double-blind, randomized, sham-procedure controlled study of IONIS-SMN Rx in patients with infantile-onset SMA. Subjects will be randomized 2:1 to receive IONIS-SMN Rx or a sham procedure control, respectively.
  Eligibility

Ages Eligible for Study:   up to 210 Days   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Be 7 months (210 days) of age or younger at screening
  • Be born (gestational age) between 37 and 42 weeks
  • Be medically diagnosed with spinal muscular atrophy (SMA)
  • Have SMN2 Copy # of 2
  • Body weight equal to or greater than 3rd percentile for age using appropriate country-specific guidelines
  • Be able to follow all study procedures
  • Reside within 9 hours ground travel each way, for the duration of the study
  • Meet additional study-related criteria

Exclusion Criteria:

  • Hypoxemia (O2 saturation awake less than 96% or O2 saturation asleep less than 96%, without ventilation support) during screening evaluation
  • Meet additional study-related criteria
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02193074

  Show 36 Study Locations
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
  More Information

Additional Information:
Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02193074     History of Changes
Other Study ID Numbers: ISIS 396443-CS3B 
Study First Received: July 14, 2014
Last Updated: May 6, 2016
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Japan: Pharmaceuticals and Medical Devices Agency
Germany: Federal Institute for Drugs and Medical Devices
Australia: Department of Health and Ageing Therapeutic Goods Administration
France: Agence Nationale de Sécurité du Médicament et des produits de santé
Hong Kong: Department of Health
Italy: The Italian Medicines Agency
Korea: Food and Drug Administration
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Sweden: Medical Products Agency
Taiwan : Food and Drug Administration

Keywords provided by Ionis Pharmaceuticals, Inc.:
Spinal Muscular Atrophy
SMA
SMN
SMNRx
ISIS-SMNRx
ISIS-SMN Rx
ISIS 396443
Endear
IONIS-SMNRx
IONIS-SMN Rx

Additional relevant MeSH terms:
Atrophy
Muscular Atrophy
Muscular Atrophy, Spinal
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases

ClinicalTrials.gov processed this record on July 21, 2016