NCT01302600

Brief Summary

Assess the efficacy and the safety of olesoxime in SMA type 2 or type 3 non ambulant patients aged 3-25 years

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
165

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Nov 2010

Typical duration for phase_2

Geographic Reach
7 countries

22 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2010

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

February 18, 2011

Completed
6 days until next milestone

First Posted

Study publicly available on registry

February 24, 2011

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2013

Completed
Last Updated

November 22, 2016

Status Verified

November 1, 2016

Enrollment Period

2.9 years

First QC Date

February 18, 2011

Last Update Submit

November 21, 2016

Conditions

Spinal Muscular Atrophy Type IISpinal Muscular Atrophy Type III Non Ambulant

Outcome Measures

Primary Outcomes (1)

  • Motor Function Measure

    Motor function Measure (MFM) D1+D2 score

    every 6 months

Secondary Outcomes (1)

  • responder analyses on MFM and HFMS, time to 4 point decrease on HFMS, CMAP/MUNE, PedsQL, FVC, CGI and safety

    every 3 months

Study Arms (2)

Olesoxime

EXPERIMENTAL

100 patients in this arm. liquid suspension

Drug: Olesoxime

Placebo

PLACEBO COMPARATOR

50 patients enrolled in this arm. liquid suspension

Drug: Placebo

Interventions

OlesoximeDRUG

Liquid suspension formulation, 100 mg/ml at a dose of 10 mg/kg will be administered once a day with food at dinner

Olesoxime
PlaceboDRUG

0.1ml/kg once a day with food at dinner.

Placebo

Eligibility Criteria

Age3 Years - 25 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Weakness and hypotonia consistent with a clinical diagnosis of spinal muscular atrophy (SMA) type II or III
  • Laboratory documentation of homozygous absence of SMNI exon 7 and/or deletion and mutation on other allele
  • MFM relative score (percentage of the maximum sum of both dimensions) \>= 15% (D1 + D2 score)
  • HFMS score at baseline \>= 3
  • Non ambulant patients defined as patients with HFMS score =\< 38
  • Must be 3 years of age or older, but younger than 26 years of age, at time of enrolment
  • Age of onset of symptoms =\< 3 years of age
  • Signed informed consent of patient and/or parents/guardian
  • Laboratory results drawn within 31 days prior to start of study entry demonstrating no clinically significant abnormalities
  • Ability to take the study treatment (tested at screening after informed consent)

You may not qualify if:

  • Evidence of renal dysfunction, blood dysplasia, hepatic insufficiency, symptomatic pancreatitis, congenital heart defect, known history of metabolic acidosis, hypertension,significant central nervous system impairment, or neurodegenerative or neuromuscular disease other than SMA
  • Any clinically significant ECG abnormality
  • Any acute co-morbid condition interfering with the well-being of the subject within 7 days of enrolment including bacterial infection, viral infectious processes, food poisoning, temperature \> 37.0 °C, the need for acute treatment or observation due to any other reason, as judged by the investigator; patient can be included after resolution of the acute event
  • Spinal rod or fixation for scoliosis within the past 6 months or anticipated need of rod or fixation within 6 months of enrolment.
  • Inability to meet study visit requirements or cooperate reliably with functional testing
  • Coexisting medical conditions that contraindicate travel, testing or study medications
  • Olesoxime is contraindicated in subjects/patients who develop drug hypersensitivity to it or one of the formulation excipients including hypersensitivity to sesame oil.
  • Patients with hemostasis disorders
  • Patients with known biliary tract obstruction
  • Current or planned pregnancy or nursing period
  • For Women: Failure to use one of the following safe methods of contraception:
  • Female condoms, diaphragm or coil, each used in combination with spermicides
  • Intra-uterine device
  • Hormonal contraception in combination with a mechanical method of contraception
  • Participation in any other investigational drug or therapy study within the previous 3 months.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (22)

University Hospitals

Ghent, 9000, Belgium

Location

University Hospitals

Leuven, 3000, Belgium

Location

Hôpital Femme-Mère-Enfant

Bron, 69677, France

Location

Hôpital Raymond Poincaré

Garches, 92380, France

Location

CHRU Hôpital R. Salengro

Lille, 59037, France

Location

Hôpital La Timone

Marseille, 13385, France

Location

CHU de Montpellier, Hôpital Gui de Chauliac

Montpellier, 34295, France

Location

Groupe hospitalier Armand-Trousseau

Paris, 75012, France

Location

University of Essen

Essen, 45122, Germany

Location

Universitat Klinikum Freiburg

Freiburg im Breisgau, 79106, Germany

Location

Friedrich-Baur-Institute

München, 80336, Germany

Location

Istituto Giannina Gaslini

Genova, 16147, Italy

Location

AOU Policlinico

Messina, 98 125, Italy

Location

Centro Dino Ferrari, Milano

Milan, 20122, Italy

Location

NEuroMuscular Omnicentre (NEMO)

Milan, 20162, Italy

Location

Bambino Gesu' Research Children's Hospital

Roma, 165, Italy

Location

Policlinico Universitario "Agostino Gemelli",

Roma, 168, Italy

Location

University Medical Center

Utrecht, 3508 GA, Netherlands

Location

Medical University of Warsaw

Warsaw, 02-097, Poland

Location

Birmingham Heartlands Hospital

Birmingham, B92 0AN, United Kingdom

Location

Dubowitz Neuromuscular Centre

London, WC1N 1EH, United Kingdom

Location

Institute of Human Genetics

Newcastle, NE1 3BZ, United Kingdom

Location

Related Publications (1)

  • Bertini E, Dessaud E, Mercuri E, Muntoni F, Kirschner J, Reid C, Lusakowska A, Comi GP, Cuisset JM, Abitbol JL, Scherrer B, Ducray PS, Buchbjerg J, Vianna E, van der Pol WL, Vuillerot C, Blaettler T, Fontoura P; Olesoxime SMA Phase 2 Study Investigators. Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial. Lancet Neurol. 2017 Jul;16(7):513-522. doi: 10.1016/S1474-4422(17)30085-6. Epub 2017 Apr 28.

    PMID: 28460889DERIVED

MeSH Terms

Conditions

Spinal Muscular Atrophies of Childhood

Interventions

olesoxime

Condition Hierarchy (Ancestors)

Muscular Atrophy, SpinalSpinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesMotor Neuron DiseaseNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Enrico Bertini, MD

    Bambino Gesu Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 18, 2011

First Posted

February 24, 2011

Study Start

November 1, 2010

Primary Completion

October 1, 2013

Study Completion

October 1, 2013

Last Updated

November 22, 2016

Record last verified: 2016-11

Locations

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