NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)

NCT02628743

CompletedPhase 2

Sponsor: Hoffmann-La Roche

Conditions: Muscular Atrophy, Spinal

Interventions: Olesoxime

Countries: Belgium, France, Germany, Italy, Netherlands, Poland, United Kingdom

The purpose of this open-label, single arm study is to further evaluate long-term tolerability, safety and efficacy outcomes of olesoxime in participants with Spinal Muscular Atrophy (SMA) who previously participated in one of the following two clinical studies: TRO19622 CL E Q 1115-1 (open-label Phase Ib, multicenter, single- and multiple- dose study) or TRO19622 CL E Q 1275-1 (NCT01302600, Phase II/III, adaptive, parallel-group, double blind, randomized, placebo-controlled, multicenter, multinational study).

Eligibility overview

Sex: ALL

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
Inclusion Criteria:

* Participation in the previous studies (TRO19622 CL E Q 1115-1 or TRO19622 CL E Q 1275-1)
* For women of childbearing potential: agreement to use an acceptable birth control method during the treatment period and for at least 28 days after the last dose of olesoxime

Exclusion Criteria:

* Female participants who are pregnant or lactating, or intending to become pregnant during the study
* Participants who, in the opinion of the investigator, are not suitable to participate in this open-label study
* Participants who have developed study drug hypersensitivity to olesoxime or one of the formulation excipients, including sesame oil
* Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening
* Concomitant or previous participation in a survival motor neuron 2 (SMN2) targeting antisense oligonucleotide study within 6 months prior to screening
* History of human immunodeficiency virus infection, history of Hepatitis B infection within the past year, history of Hepatitis C infection which has not been adequately treated
* History of illicit drug or alcohol abuse within 12 months prior to screening, in the investigator's judgment
* Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the participant's safe participation in and completion of the study
* History or presence of an abnormal electrocardiogram (ECG) that is clinically significant in the investigator's opinion
Locations (24)
  • Ghent, Belgium
  • Leuven, Belgium
  • Bron, France
  • Garches, France
  • Lille, France
  • Marseille, France
  • Montpellier, France
  • Paris, France
  • Toulouse, France
  • Essen, Germany
  • Freiburg im Breisgau, Germany
  • München, Germany
  • Rome, Lazio, Italy
  • Rome, Lazio, Italy
  • Genoa, Liguria, Italy
  • Milan, Lombardy, Italy
  • Milan, Lombardy, Italy
  • Messina, Sicily, Italy
  • Utrecht, Netherlands
  • Warsaw, Poland
  • Birmingham, United Kingdom
  • London, United Kingdom
  • London, United Kingdom
  • Newcastle upon Tyne, United Kingdom