Trial record 1 of 1 for:    NCT01494701
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An Open-label Safety, Tolerability, and Dose-range Finding Study of ISIS SMNRx in Patients With Spinal Muscular Atrophy

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Isis Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01494701
First received: December 13, 2011
Last updated: April 23, 2013
Last verified: April 2013

December 13, 2011
April 23, 2013
November 2011
January 2013   (final data collection date for primary outcome measure)
The number of participants with adverse events [ Time Frame: Participants will be followed for the duration of the study; an expected 4 weeks for cohorts 1 and 2, and 12 weeks for cohorts 3 and 4 ] [ Designated as safety issue: Yes ]
The number of participants with adverse events [ Time Frame: Participants will be followed for the duration of the study; an expected 4 weeks ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT01494701 on ClinicalTrials.gov Archive Site
Plasma Pharmacokinetics (See clarification.) [ Time Frame: Plasma at 1, 2, 4 and 20 hours after dosing ] [ Designated as safety issue: No ]
  • the maximal observed plasma drug concentration (Cmax)
  • the time to reach Cmax in plasma (Tmax)
  • the area under the plasma concentrations time curve from the time of the intrathecal dose to the last collected sample (20 hours after dosing)
Same as current
Not Provided
Not Provided
 
An Open-label Safety, Tolerability, and Dose-range Finding Study of ISIS SMNRx in Patients With Spinal Muscular Atrophy
An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy

This study will test the safety, tolerability, and pharmacokinetics of escalating doses of ISIS-SMNRx administered into the spinal fluid as a single injection in patients with Spinal Muscular Atrophy.

This study will test the safety, tolerability, and pharmacokinetics of escalating doses of ISIS-SMNRx administered as a single intrathecal injection. Four dose levels will be evaluated sequentially. Each dose level will be studied in a cohort of 6 or 10 patients, where all patients will receive active drug.

Interventional
Phase 1
Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Spinal Muscular Atrophy
Drug: ISIS-SMNRx
Single intrathecal injection
  • Experimental: ISIS-SMNRx: Dose level 1
    Intervention: Drug: ISIS-SMNRx
  • Experimental: ISIS-SMNRx: Dose level 2
    Intervention: Drug: ISIS-SMNRx
  • Experimental: ISIS-SMNRx: Dose level 3
    Intervention: Drug: ISIS-SMNRx
  • Experimental: ISIS-SMNRx: Dose level 4
    Intervention: Drug: ISIS-SMNRx
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
28
January 2013
January 2013   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Documented SMN1 homozygous gene deletion
  • Clinical signs attributable to Spinal Muscular Atrophy
  • Males and females 2 to 14 years of age at time of screening
  • Able to complete all study procedures, measurements and visits and parent/patient has adequately supportive psychosocial circumstances, in the opinion of the investigator
  • Estimated life expectancy > 2 years from Screening
  • Meets age-appropriate institutional criteria for use of anesthesia/sedation, if use is planned for study procedure

Exclusion Criteria:

  • Respiratory insufficiency defined by the need for invasive or non-invasive ventilation during a 24 hour period
  • Presence of a gastric feeding tube
  • Previous scoliosis surgery or scoliosis surgery planned during the duration of the study that would interfere with the LP injection procedure
  • Hospitalization for surgery or pulmonary event within the last 2 months or planned during the study
  • Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy
  • History of brain or spinal cord disease that would interfere with lumbar puncture procedures or CSF circulation
  • Presence of an implanted shunt for the draining of CSF or an implanted CNS catheter
  • History of bacterial meningitis
  • Clinically significant abnormalities in hematology or clinical chemistry parameters
  • Treatment with another investigational drug, biological agent, or device within 1-month of Screening or 5 half-lives of study agent whichever is longer. Any history of gene therapy or cell transplantation
  • Ongoing medical condition that would interfere with the conduct and assessments of the study. Examples are medical disability (e.g. wasting or cachexia, severe anemia, etc.) that would interfere with the assessment of safety or would compromised the ability of the patient to undergo study procedures
Both
2 Years to 14 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01494701
ISIS 396443 - CS1
Yes
Isis Pharmaceuticals
Isis Pharmaceuticals
Not Provided
Not Provided
Isis Pharmaceuticals
April 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP