- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02240355
A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)
December 20, 2016 updated by: Hoffmann-La Roche
A Multicenter, Randomized, Double Blind, Placebo Controlled, Multiple Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO6885247 Following 12 Weeks of Treatment in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH).
This multicenter, randomized, double-blind, 12-week, placebo-controlled multiple dose study will investigate the safety and tolerability of RO6885247 in adult and pediatric patients with spinal muscular atrophy (SMA).
Study Overview
Status
Terminated
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
9
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Ontario
-
Toronto, Ontario, Canada, M5G 1X8
-
-
Quebec
-
Montreal, Quebec, Canada
-
-
-
-
-
Paris, France, 75013
-
Paris, France, 75013
- Ch Pitie Salpetriere; Institut de Myologie
-
-
-
-
Lazio
-
Roma, Lazio, Italy, 00168
-
Roma, Lazio, Italy, 00168
- Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile
-
-
Lombardia
-
Milano, Lombardia, Italy, 20133
-
Milano, Lombardia, Italy, 20133
- Fondazione IRCCS Istituto Neurologico "Carlo Besta"; UO di Neurologia dello Sviluppo
-
-
-
-
-
Utrecht, Netherlands, 3584 CX
-
Utrecht, Netherlands, 3584 CX
- UMC Utrecht; Polkliniek Neuromusculaire ziekten
-
-
-
-
-
Goeteborg, Sweden, 41685
- Drottning Silvias Barn- och ungdomssjukhus; Kliniken för barnmedicin
-
Goeteborg, Sweden, 41685
-
-
-
-
-
Basel, Switzerland, 4005
- Universitäts-Kinderspitalbeider Basel_Abteilung für Neuro- und Entwicklungspädiatrie
-
Basel, Switzerland, 4005
-
-
-
-
-
Ankara, Turkey, 06100
-
Ankara, Turkey, 06100
- Hacettepe University, School of Medicine; Pediatrics Department; Pediatrics Child Neurology Unit
-
-
-
-
-
London, United Kingdom, WC1N 1EH
- UCL; GAP Unit, Institute of Child Health, UCL
-
London, United Kingdom, WC1N 1EH
-
Newcastle upon Tyne, United Kingdom, NE1 3BZ
- MRC Neuromuscular Centre - Institute of Genetic Medicine
-
Newcastle upon Tyne, United Kingdom, NE1 3BZ
-
-
-
-
California
-
Stanford, California, United States, 94305
-
-
Florida
-
Orlando, Florida, United States, 32827
-
-
Illinois
-
Chicago, Illinois, United States, 60611-2605
-
-
Massachusetts
-
Boston, Massachusetts, United States, 02115
-
-
Missouri
-
St. Louis, Missouri, United States, 63110
-
-
New York
-
New York, New York, United States, 10032
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 55 years (ADULT, CHILD)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Males and females, aged 2 to 55 years inclusive or below 7 months inclusive
- Confirmed diagnosis of 5q-autosomal recessive SMA (Types 1 to 3), for patients aged 7 months or below clinical symptoms attributable to type 1 SMA and 2 SMN2 copies
- Able and willing to provide informed consent and to comply with the study protocol. Alternatively, a legally authorized representative must be able to consent for the patient and assent must be given by the subject wherever possible.
- Female patients of childbearing potential and male patients with a female partner of childbearing potential must agree with the required contraceptive methods as defined per protocol.
- For patients aged 7 months or below, Gestational age of 37 to 42 weeks and not considered small for gestational age at birth
Exclusion Criteria:
- Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening
- Concomitant or previous participation in a SMN2-targeting antisense oligonucleotide study within 12 months prior to screening
- Concomitant or previous participation at any time in a gene therapy study
- For patients aged 2-55 years, hospitalization for pulmonary event within the last 2 months or planned at the time of screening
- Surgery for scoliosis in the last 6 months from screening or planned within 6 months from screening
- Unstable gastrointestinal, renal, hepatic, endocrine or cardiovascular system disease
- Clinically relevant ECG abnormalities at screening or baseline; personal or family history (first degree relatives) of congenital long QT syndrome
- Clinically significant abnormalities in laboratory test results at screening
- Any concomitant disease or condition that could interfere with the conduct of the study, or pose an unacceptable risk to the subject in this study
- Use of prohibited medications as per protocol within 90 days prior to randomization. Patients who are on inhaled corticosteroids, administered either through a nebulizer or an inhaler, are allowed.
- Recently initiated treatment (within <6 months prior to randomization) with oral salbutamol or another beta2-adrenergic agonist taken orally is not allowed. Patients who have been on oral salbutamol (or another beta2-adrenergic agonist) for at least 6 months before randomization are allowed. Use of inhaled beta2-adrenergic agonists is allowed.
- For patients aged 7 months or below, patients requiring invasive ventilation or tracheostomy, presence of non-SMA related morbidities
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: SINGLE_GROUP
- Masking: DOUBLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: Part 1
Up to 2 cohorts of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
|
oral solution
oral solution
|
EXPERIMENTAL: Part 2
1 cohort of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
|
oral solution
oral solution
|
EXPERIMENTAL: Part 3
1 cohort of patients, within each cohort patients will receive RO6885247 once daily for 12 weeks or 20 weeks
|
oral solution
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Safety: Incidence of adverse events (AEs)
Time Frame: Up to 20 weeks
|
Up to 20 weeks
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Pharmacokinetics: RO6885247 plasma concentrations
Time Frame: Up to 16 weeks
|
Up to 16 weeks
|
Pharmacokinetics: RO6885247 exposure, area under the concentration-time curve (AUC-tau, over the 24-hour dosing interval)
Time Frame: Up to 12 weeks
|
Up to 12 weeks
|
Pharmacodynamics: SMN protein levels in blood
Time Frame: Up to 20 weeks
|
Up to 20 weeks
|
Effect of RO6885247 on muscle electrophysiology, as assessed by Compound Muscle Action Potential (CMAP)
Time Frame: Up to 20 weeks
|
Up to 20 weeks
|
Effect of RO6885247 on Electrical Impedance Myography
Time Frame: Up to 20 weeks
|
Up to 20 weeks
|
Pharmacodynamics: In vivo splicing modification of SMN2 mRNA in blood
Time Frame: Up to 20 weeks
|
Up to 20 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
November 1, 2014
Primary Completion (ACTUAL)
July 1, 2015
Study Completion (ACTUAL)
July 1, 2015
Study Registration Dates
First Submitted
September 11, 2014
First Submitted That Met QC Criteria
September 11, 2014
First Posted (ESTIMATE)
September 15, 2014
Study Record Updates
Last Update Posted (ESTIMATE)
December 22, 2016
Last Update Submitted That Met QC Criteria
December 20, 2016
Last Verified
December 1, 2016
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- BP29420
- 2014-002246-41 (EUDRACT_NUMBER)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Muscular Atrophy, Spinal
-
Marco CapogrossoRoche-GenentechRecruitingSpinal Muscular Atrophy Type 3 | Spinal Muscular Atrophy Type 4United States
-
Institut de Myologie, FranceInstitut RocheCompletedType 2 Spinal Muscular Atrophy | Type 3 Spinal Muscular AtrophyBelgium, France, Germany
-
Marco CapogrossoRoche-GenentechNot yet recruitingSpinal Muscular Atrophy | Spinal Muscular Atrophy Type 3 | SMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy 4United States
-
Novartis Gene TherapiesActive, not recruitingSMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type I | Spinal Muscular Atrophy Type IIIUnited States, Belgium, France, Japan, United Kingdom, Italy, Taiwan, Australia, Canada
-
Hoffmann-La RocheRecruitingSpinal Muscular Atrophy (SMA)Belgium, United States, Croatia, Netherlands, Spain, Canada, Poland, United Kingdom, Italy, Portugal, Australia
-
Northwell HealthCompletedAdult Spinal Muscular AtrophyUnited States
-
Hugh McMillanFamilies of Spinal Muscular Atrophy; Gwendolyn Strong FoundationTerminatedSpinal Muscular Atrophy (SMA)Canada
-
Istanbul Medipol University HospitalIstanbul UniversityRecruitingNeuromuscular Diseases | Spinal Muscular Atrophy Type 3Turkey
-
Washington University School of MedicineActive, not recruitingSpinal Muscular Atrophy | Spinal Muscular Atrophy Type 3 | Spinal Muscular Atrophy Type IIUnited States, Canada
-
Hoffmann-La RocheAssociation Française contre les Myopathies (AFM), ParisCompletedSpinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type III Non AmbulantGermany, Italy, France, Belgium, Poland, Netherlands, United Kingdom