A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)

Stopped early · Phase 1 · Has a placebo group

Conditions studied: Muscular Atrophy, Spinal

In brief

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).

Key facts

Study ID

NCT02240355

Run by

Hoffmann-La Roche

People needed

9

Starts

2014-11-01

Expected to finish

2015-07-01

Last updated by the study team

2016-12-22

Who can join

Age: any, up to 55. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Study site — Stanford, California, United States
• Study site — Orlando, Florida, United States
• Study site — Chicago, Illinois, United States
• Study site — Boston, Massachusetts, United States
• Study site — St Louis, Missouri, United States
• Study site — New York, New York, United States
• Study site — Toronto, Ontario, Canada
• Study site — Montreal, Quebec, Canada
• Ch Pitie Salpetriere; Institut de Myologie — Paris, France
• Study site — Paris, France
• Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile — Rome, Lazio, Italy
• Study site — Rome, Lazio, Italy
• Fondazione IRCCS Istituto Neurologico "Carlo Besta"; UO di Neurologia dello Sviluppo — Milan, Lombardy, Italy
• Study site — Milan, Lombardy, Italy
• UMC Utrecht; Polkliniek Neuromusculaire ziekten — Utrecht, Netherlands
• Study site — Utrecht, Netherlands
• Drottning Silvias Barn- och ungdomssjukhus; Kliniken för barnmedicin — Gothenburg, Sweden
• Study site — Gothenburg, Sweden
• Universitäts-Kinderspitalbeider Basel_Abteilung für Neuro- und Entwicklungspädiatrie — Basel, Switzerland
• Study site — Basel, Switzerland
• Hacettepe University, School of Medicine; Pediatrics Department; Pediatrics Child Neurology Unit — Ankara, Turkey (Türkiye)
• Study site — Ankara, Turkey (Türkiye)
• UCL; GAP Unit, Institute of Child Health, UCL — London, United Kingdom
• Study site — London, United Kingdom
• MRC Neuromuscular Centre - Institute of Genetic Medicine — Newcastle upon Tyne, United Kingdom

Full record on ClinicalTrials.gov

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