We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02628743
First Posted: December 11, 2015
Last Update Posted: November 8, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Hoffmann-La Roche
  Purpose
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 SMA who previously participated in one of the following two clinical studies: TRO19622 CL E Q 1115-1 (NCT ID XXX, open-label Phase Ib, dose-ranged, single and multiple dose study) or TRO19622 CL E Q 1275-1 (NCT01302600, Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study).

Condition Intervention Phase
Muscular Atrophy, Spinal Drug: Olesoxime Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Multicenter, Open-Label, Single Arm Study to Evaluate Long-Term Safety, Tolerability, and Effectiveness of 10 mg/kg Olesoxime in Patients With SMA

Resource links provided by NLM:


Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) [ Time Frame: Baseline up to approximately 5 years ]

Secondary Outcome Measures:
  • Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Apparent Clearance (CL/f) of Olesoxime [ Time Frame: Pre-dose (Hour 0) at Weeks 1, 13, 26, 39, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Apparent Volume of Distribution at Steady State (Vss/F) of Olesoxime [ Time Frame: Pre-dose (Hour 0) at Weeks 1, 13, 26, 39, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Pediatric Quality of Life Questionnaire (PedsQL) Generic Core Scale Version 4.0 Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • PedsQL Neuromuscular Module Version 3.0 Scale Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Caregiver PedsQL Generic Core Scales Version 4.0 Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Caregiver PedsQL Neuromuscular Module Version 3.0 Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • EuroQol 5-Dimension 5-Level (EQ-5D-5L) Questionnaire Index Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • EQ-5D-5L Visual Analogue Scale (EQ-5D-5L VAS) Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Caregiver Proxy EQ-5D-5L Questionnaire Index Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Caregiver Proxy EQ-5D-5L VAS Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Work Productivity and Activity Impairment: Caregiver (WPAI:CG) Questionnaire Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Caregiver Generic Health Related Quality of Life: Short Form-36 Questionnaire Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]

Enrollment: 131
Actual Study Start Date: January 20, 2016
Estimated Study Completion Date: May 8, 2021
Estimated Primary Completion Date: May 8, 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Olesoxime
Participants will receive 10 milligrams per kilogram (mg/kg) suspension once a day either orally or via a naso-gastric or gastrostomy tube with the main meal (preferably at the same time of the day) until olesoxime is commercially available in the participant's country, or as per local regulation, or until study termination by sponsor.
Drug: Olesoxime
Participants will receive homogeneous suspension of olesoxime.

  Eligibility

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02628743


Locations
Belgium
UZ Gent
Gent, Belgium, 9000
UZ Leuven Gasthuisberg
Leuven, Belgium, 3000
France
Hopital Femme Mere Enfant; Medecine Physique et Readaptation Pediatrique - L'ESCALE
Bron, France, 69677
Hôpital Raymond Poincare; Serv. Neurologie et Réanimation pédiatriques - Centre réf. neuromusculaire
Garches, France, 92380
Hopital Jeanne De Flandre; CIC pediatrique
Lille, France, 59037
Hopital la Timone Enfants; Service de Pediatrie et Neurologie Pediatrique
Marseille, France, 13005
CHRU de Montpellier, Hopital Gui de Chauliac; Service de Neuropediatrie
Montpellier, France, 34295
Hôpital Armand Trousseau; centre référence Maladies Neuro-musculaires Est parisien Neuropediatrie
Paris Cedex 12, France, 75571
Hopital des Enfants; Unite de Neurologie Pediatrique
Toulouse, France, 31059
Germany
Universitätsklinikum Essen; Neuropädiatrie
Essen, Germany, 45147
Uniklinikum Freiburg Zentrum für Kinder- und Jugendmedizin; Neuropädiatrie und Muskelerkrankungen
Freiburg, Germany, 79106
Dr. Von Haunersches Kinderspital
München, Germany, 80337
Italy
Ospedale Pediatrico Bambino Gesù; Dip. Neuroscienze e Salute Mentale
Roma, Lazio, Italy, 00165
Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile
Roma, Lazio, Italy, 00168
IRCCS Istituto G. Gaslini; UOC Neurologia Pediatrica e Malattie Muscolari
Genova, Liguria, Italy, 16147
I.R.C.C.S. Cà Granda - Ospedale Maggiore Policlinico; Dip. di Salute Mentale
Milano, Lombardia, Italy, 20100
ASST GRANDE OSPEDALE METROPOLITANO NIGUARDA;NEMO (NEuroMuscular Omnicentre);Centro clinico - Fonda
Milano, Lombardia, Italy, 20162
Azienda Ospedaliera Universitaria Policlinico G.Martino; Dip. Neurologia e Malattie neuromuscolari
Messina, Sicilia, Italy, 98125
Netherlands
UMC Utrecht; Polkliniek Neuromusculaire ziekten
Utrecht, Netherlands, 3584 CX
Poland
Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie; Klinika Neurologii
Warszawa, Poland, 02-097
United Kingdom
Heart of England NHS Trust
Birmingham, United Kingdom, B9 5SS
National Hospital for Neurology and Neurosurgery,; MRC Centre for Neuromuscular Diseases
London, United Kingdom, WC1 3BG
Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Newcastle University & The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT02628743     History of Changes
Other Study ID Numbers: BN29854
2015-001589-25 ( EudraCT Number )
First Submitted: December 1, 2015
First Posted: December 11, 2015
Last Update Posted: November 8, 2017
Last Verified: November 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

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