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Completed

NCT Number: NCT01780246

An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)

The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS 396443-CS1 (NCT02865109). The secondary objective was to examine the plasma pharmacokinetics of a single dose of ISIS 396443 administered intrathecally to participants with SMA who previously participated in ISIS 396443-CS1.

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Key information

Conditions

Spinal Muscular Atrophy Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Age range

2 year–15 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Boston Children's Hospital, Boston, Massachusetts, United States

Loading trial locations.

About this study

This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc.

In August 2016, sponsorship of the trial was transferred to Biogen.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Clinical signs attributable to Spinal Muscular Atrophy
  • Satisfactory completion of dosing and all study visits in ISIS 396443-CS1 (NCT01494701) with an acceptable safety profile, per Investigator judgement.
  • Able to complete all study procedures, measurements and visits and parent/participant 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

Key Exclusion Criteria:

  • Have any new or worsening of existing condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study.
  • Dosing in ISIS 396443-CS1 (NCT01494701) within 270 days (9 months) of screening, or longer ago than 450 days (15 months)
  • Dosing in ISIS 396443-CS2 (NCT01703988)
  • Hospitalization for surgery (i.e. scoliosis surgery) or pulmonary event within 2 months of screening or planned during the duration of the study
  • Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy any time during the screening period
  • Clinically significant abnormalities in hematology or clinical chemistry parameters
  • Treatment with investigational drug, biological agent, or device within 1-month of Screening or 5 half-lives of study agent, whichever is longer. Treatment with valproate or hydroxyurea within 1 months of screening. Any history of gene therapy or cell transplantation

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

nusinersen

Drug

Administered by intrathecal (IT) injection

Other names: Sprinraza, ISIS 396443, IONIS-SMN Rx, BIIB058

Primary outcomes

  1. Number of participants that experience Adverse Events (AEs) and Serious Adverse Events

    Time frame: Up to 24 Weeks

  2. Number of participants with clinically significant neurological examination abnormalities

    Time frame: Up tp 24 Weeks

  3. Number of participants with clinically significant vital sign abnormalities

    Time frame: Up to 24 Weeks

  4. Number of participants with clinically significant physical examination abnormalities

    Time frame: Up to 24 Weeks

  5. Number of participants with clinically significant weight abnormalities

    Time frame: Up to 24 Weeks

  6. Number of participants with clinically significant laboratory parameters

    Time frame: Up to 24 Weeks

  7. Number of participants with clinically significant electrocardiograms (ECGs) abnormalities

    Time frame: Up to 24 Weeks

  8. Number of participants who use concomitant medications

    Time frame: Up to 24 Weeks

Secondary outcomes

  1. PK parameters of nusinersen (ISIS 396443): Maximum observed plasma drug concentration (Cmax)

    Time frame: Plasma at 1, 2, 4 and 6 hours after dosing

  2. PK parameters of nusinersen: Time to reach maximum observed concentration (Tmax)

    Time frame: Plasma at 1, 2, 4 and 6 hours after dosing

  3. PK parameters of nusinersen: Area under the plasma concentrations time curve from the time of the intrathecal (IT) dose to the last collected sample (AUCinf)

    Time frame: Plasma at 1, 2, 4 and 6 hours after dosing

  4. PK parameters of nusinersen (ISIS 396443): Apparent terminal elimination half-life (t1/2), if possible

    Time frame: Plasma at 1, 2, 4 and 6 hours after dosing

Sponsors and collaborators

Lead sponsor

Biogen

Industry

Registry information

Official study title

An Open-label Study to Assess the Safety and Tolerability of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1

Important dates

Study start
2013
Primary completion
2014
Study completion
2014
First posted
Jan 31, 2013
Registry last updated
Feb 16, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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