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

NCT ID: NCT02240355

Last Updated: 2016-12-22

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-11-30
• Study Completion Date: 2015-07-31

Brief Summary

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).

Conditions

Muscular Atrophy, Spinal

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Part 1

Up to 2 cohorts of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks

Group Type: EXPERIMENTAL

RO6885247

Intervention Type: DRUG

oral solution

placebo

Intervention Type: DRUG

oral solution

Part 2

1 cohort of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks

Group Type: EXPERIMENTAL

RO6885247

Intervention Type: DRUG

oral solution

placebo

Intervention Type: DRUG

oral solution

Part 3

1 cohort of patients, within each cohort patients will receive RO6885247 once daily for 12 weeks or 20 weeks

Group Type: EXPERIMENTAL

RO6885247

Intervention Type: DRUG

oral solution

Interventions

RO6885247

oral solution

Intervention Type: DRUG

placebo

oral solution

Intervention Type: DRUG

Eligibility Criteria

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

• Maximum Eligible Age: 55 Years
• 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

Stanford, California, United States

Orlando, Florida, United States

Chicago, Illinois, United States

Boston, Massachusetts, United States

St Louis, Missouri, United States

New York, New York, United States

Toronto, Ontario, Canada

Montreal, Quebec, Canada

Ch Pitie Salpetriere; Institut de Myologie

Paris, , France

Paris, , France

Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile

Rome, Lazio, Italy

Rome, Lazio, Italy

Fondazione IRCCS Istituto Neurologico "Carlo Besta"; UO di Neurologia dello Sviluppo

Milan, Lombardy, Italy

Milan, Lombardy, Italy

UMC Utrecht; Polkliniek Neuromusculaire ziekten

Utrecht, , Netherlands

Utrecht, , Netherlands

Drottning Silvias Barn- och ungdomssjukhus; Kliniken för barnmedicin

Gothenburg, , Sweden

Gothenburg, , Sweden

Universitäts-Kinderspitalbeider Basel_Abteilung für Neuro- und Entwicklungspädiatrie

Basel, , Switzerland

Basel, , Switzerland

Hacettepe University, School of Medicine; Pediatrics Department; Pediatrics Child Neurology Unit

Ankara, , Turkey (Türkiye)

Ankara, , Turkey (Türkiye)

UCL; GAP Unit, Institute of Child Health, UCL

London, , United Kingdom

London, , United Kingdom

MRC Neuromuscular Centre - Institute of Genetic Medicine

Newcastle upon Tyne, , United Kingdom

Newcastle upon Tyne, , United Kingdom

Countries

Czechia; United States; Canada; France; Italy; Netherlands; Sweden; Switzerland; Turkey (Türkiye); United Kingdom

Other Identifiers

2014-002246-41

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

BP29420

Identifier Type: -

Identifier Source: org_study_id