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

NCT ID: NCT02628743

Last Updated: 2019-08-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 131 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-01-20
• Study Completion Date: 2018-12-18

Brief Summary

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).

Conditions

Muscular Atrophy, Spinal

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Olesoxime

Participants who have consented to the dose increase will receive 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube with breakfast and dinner, preferably at the same time of the day throughout the study. If the drug administration does not coincide with one of the scheduled meals, a snack should be taken prior to drug administration. Preferably there should be at least 10 hours between the morning and evening dose. The total dose in this study will not exceed 2000 mg.

Participants who do not consent to the dose increase will continue with the previous dosage and receive a dose of 10 mg/kg suspension once a day orally or via a naso-gastric or gastronomy tube with the main meal, preferably at the same time of the day.

Group Type: EXPERIMENTAL

Olesoxime

Intervention Type: DRUG

Participants will receive homogeneous suspension of olesoxime.

Interventions

Olesoxime

Participants will receive homogeneous suspension of olesoxime.

Intervention Type: DRUG

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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hoffmann-La Roche

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

UZ Gent

Ghent, , Belgium

UZ Leuven Gasthuisberg

Leuven, , Belgium

Hopital Femme Mere Enfant; Medecine Physique et Readaptation Pediatrique - L'ESCALE

Bron, , France

Hôpital Raymond Poincare; Serv. Neurologie et Réanimation pédiatriques - Centre réf. neuromusculaire

Garches, , France

Hopital Jeanne De Flandre; CIC pediatrique

Lille, , France

Hopital la Timone Enfants; Service de Pediatrie et Neurologie Pediatrique

Marseille, , France

CHRU de Montpellier, Hopital Gui de Chauliac; Service de Neuropediatrie

Montpellier, , France

Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie

Paris, , France

Hopital des Enfants; Unite de Neurologie Pediatrique

Toulouse, , France

Universitätsklinikum Essen; Neuropädiatrie

Essen, , Germany

Uniklinikum Freiburg Zentrum für Kinder- und Jugendmedizin; Neuropädiatrie und Muskelerkrankungen

Freiburg im Breisgau, , Germany

Dr. Von Haunersches Kinderspital

München, , Germany

Ospedale Pediatrico Bambino Gesù; Dip. Neuroscienze e Salute Mentale

Rome, Lazio, Italy

Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile

Rome, Lazio, Italy

IRCCS Istituto G. Gaslini; UOC Neurologia Pediatrica e Malattie Muscolari

Genoa, Liguria, Italy

I.R.C.C.S. Cà Granda - Ospedale Maggiore Policlinico; Dip. di Salute Mentale

Milan, Lombardy, Italy

ASST GRANDE OSPEDALE METROPOLITANO NIGUARDA;NEMO (NEuroMuscular Omnicentre);Centro clinico - Fonda

Milan, Lombardy, Italy

Azienda Ospedaliera Universitaria Policlinico G.Martino; Dip. Neurologia e Malattie neuromuscolari

Messina, Sicily, Italy

UMC Utrecht; Polkliniek Neuromusculaire ziekten

Utrecht, , Netherlands

Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie; Klinika Neurologii

Warsaw, , Poland

Heart of England NHS Trust

Birmingham, , United Kingdom

National Hospital for Neurology and Neurosurgery,; MRC Centre for Neuromuscular Diseases

London, , United Kingdom

Great Ormond Street Hospital

London, , United Kingdom

Newcastle University & The Newcastle upon Tyne Hospitals NHS Foundation Trust

Newcastle upon Tyne, , United Kingdom

Countries

Belgium; France; Germany; Italy; Netherlands; Poland; United Kingdom

References

Vincent-Genod D, Rippert P, Coton J, Le Goff L, Barriere A, Berruyer A, Bernard M, Garde C, Gutierrez-Garcia M, Gilabert S, Gomes-Lisboade-Souza A, Daron A, Servais L, Thomann G, Vuillerot C. Scoring People With Spinal Muscular Atrophy on the Motor Function Measure Using the Microsoft Kinect. Pediatr Phys Ther. 2023 Jan 1;35(1):36-41. doi: 10.1097/PEP.0000000000000968. Epub 2022 Oct 25.

Reference Type: DERIVED

PMID: 36288197 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2015-001589-25

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

BN29854

Identifier Type: -

Identifier Source: org_study_id