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

NCT02628743 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: BN298542015-001589-25
• Study Type: interventional
• Target: 131
• Locations: 7 countries
• Sites: 24

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

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs)

  Baseline up to approximately 3 years

Secondary Outcomes (20)

• Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score

  Baseline (Week 1), Weeks 26, 52, 78, 104 and 130

• Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score

  Baseline (Week 1), Weeks 26, 52, 78, 104 and 130

• Plasma Concentrations of Olesoxime

  Pre-dose (Hour 0) at Weeks 1, 13, 26, 39, 52, 78, 104 and 130

• Change From Baseline in Pediatric Quality of Life Questionnaire (PedsQL) Generic Core Scale Version 4.0 Score

  Baseline (Week 1), Weeks 26, 52, 78, 104 and 130

• Change From Baseline in Caregiver PedsQL Generic Core Scales Version 4.0 Score

  Baseline (Week 1), Weeks 26, 52, 78, 104 and 130

Study Arms (1)

Olesoxime

EXPERIMENTAL

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.

Drug: Olesoxime

Interventions

Olesoxime DRUG

Participants will receive homogeneous suspension of olesoxime.

Olesoxime

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (24)

UZ Gent

Ghent, 9000, Belgium

Location

UZ Leuven Gasthuisberg

Leuven, 3000, Belgium

Location

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

Bron, 69677, France

Location

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

Garches, 92380, France

Location

Hopital Jeanne De Flandre; CIC pediatrique

Lille, 59037, France

Location

Hopital la Timone Enfants; Service de Pediatrie et Neurologie Pediatrique

Marseille, 13005, France

Location

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

Montpellier, 34295, France

Location

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

Paris, 75571, France

Location

Hopital des Enfants; Unite de Neurologie Pediatrique

Toulouse, 31059, France

Location

Universitätsklinikum Essen; Neuropädiatrie

Essen, 45147, Germany

Location

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

Freiburg im Breisgau, 79106, Germany

Location

Dr. Von Haunersches Kinderspital

München, 80337, Germany

Location

Ospedale Pediatrico Bambino Gesù; Dip. Neuroscienze e Salute Mentale

Rome, Lazio, 00165, Italy

Location

Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile

Rome, Lazio, 00168, Italy

Location

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

Genoa, Liguria, 16147, Italy

Location

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

Milan, Lombardy, 20100, Italy

Location

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

Milan, Lombardy, 20162, Italy

Location

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

Messina, Sicily, 98125, Italy

Location

UMC Utrecht; Polkliniek Neuromusculaire ziekten

Utrecht, 3584 CX, Netherlands

Location

Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie; Klinika Neurologii

Warsaw, 02-097, Poland

Location

Heart of England NHS Trust

Birmingham, B9 5SS, United Kingdom

Location

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

London, WC1 3BG, United Kingdom

Location

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

Location

Newcastle University & The Newcastle upon Tyne Hospitals NHS Foundation Trust

Newcastle upon Tyne, NE1 4LP, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 36288197 DERIVED

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

olesoxime

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Results Point of Contact

• Title: Medical Communications
• Organization: Hoffmann-La Roche

genentech@druginfo.com

800 821-8590

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 1, 2015
• First Posted: December 11, 2015
• Study Start: January 20, 2016
• Primary Completion: December 18, 2018
• Study Completion: December 18, 2018
• Last Updated: August 9, 2019
• Results First Posted: August 9, 2019

Record last verified: 2019-07

Locations

DE (3); FR (7); PL (1); BE (2); IT (6); GB (4); NL (1)