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

NCT02240355 | Terminated Phase 1

• 2 other identifiers: BP294202014-002246-41
• Study Type: interventional
• Target: 9
• Locations: 9 countries
• Sites: 26

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

Outcome Measures

Primary Outcomes (1)

• Safety: Incidence of adverse events (AEs)

  Up to 20 weeks

Secondary Outcomes (6)

• Pharmacokinetics: RO6885247 plasma concentrations

  Up to 16 weeks

• Pharmacokinetics: RO6885247 exposure, area under the concentration-time curve (AUC-tau, over the 24-hour dosing interval)

  Up to 12 weeks

• Pharmacodynamics: SMN protein levels in blood

  Up to 20 weeks

• Effect of RO6885247 on muscle electrophysiology, as assessed by Compound Muscle Action Potential (CMAP)

  Up to 20 weeks

• Effect of RO6885247 on Electrical Impedance Myography

  Up to 20 weeks

Study Arms (3)

Part 1

EXPERIMENTAL

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

Drug: RO6885247; Drug: placebo

Part 2

EXPERIMENTAL

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

Drug: RO6885247; Drug: placebo

Part 3

EXPERIMENTAL

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

Drug: RO6885247

Interventions

RO6885247 DRUG

oral solution

Part 1; Part 2; Part 3

placebo DRUG

oral solution

Part 1; Part 2

Eligibility Criteria

• Age: Up to 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (26)

Unknown Facility

Stanford, California, 94305, United States

Location

Unknown Facility

Orlando, Florida, 32827, United States

Location

Unknown Facility

Chicago, Illinois, 60611-2605, United States

Location

Unknown Facility

Boston, Massachusetts, 02115, United States

Location

Unknown Facility

St Louis, Missouri, 63110, United States

Location

Unknown Facility

New York, New York, 10032, United States

Location

Unknown Facility

Toronto, Ontario, M5G 1X8, Canada

Location

Unknown Facility

Montreal, Quebec, Canada

Location

Ch Pitie Salpetriere; Institut de Myologie

Paris, 75013, France

Location

Unknown Facility

Paris, 75013, France

Location

Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile

Rome, Lazio, 00168, Italy

Location

Unknown Facility

Rome, Lazio, 00168, Italy

Location

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

Milan, Lombardy, 20133, Italy

Location

Unknown Facility

Milan, Lombardy, 20133, Italy

Location

UMC Utrecht; Polkliniek Neuromusculaire ziekten

Utrecht, 3584 CX, Netherlands

Location

Unknown Facility

Utrecht, 3584 CX, Netherlands

Location

Drottning Silvias Barn- och ungdomssjukhus; Kliniken för barnmedicin

Gothenburg, 41685, Sweden

Location

Unknown Facility

Gothenburg, 41685, Sweden

Location

Universitäts-Kinderspitalbeider Basel_Abteilung für Neuro- und Entwicklungspädiatrie

Basel, 4005, Switzerland

Location

Unknown Facility

Basel, 4005, Switzerland

Location

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

Ankara, 06100, Turkey (Türkiye)

Location

Unknown Facility

Ankara, 06100, Turkey (Türkiye)

Location

UCL; GAP Unit, Institute of Child Health, UCL

London, WC1N 1EH, United Kingdom

Location

Unknown Facility

London, WC1N 1EH, United Kingdom

Location

MRC Neuromuscular Centre - Institute of Genetic Medicine

Newcastle upon Tyne, NE1 3BZ, United Kingdom

Location

Unknown Facility

Newcastle upon Tyne, NE1 3BZ, United Kingdom

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

RG7800

Condition Hierarchy (Ancestors)

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

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 11, 2014
• First Posted: September 15, 2014
• Study Start: November 1, 2014
• Primary Completion: July 1, 2015
• Study Completion: July 1, 2015
• Last Updated: December 22, 2016

Record last verified: 2016-12

Locations

GB (4); SE (2); NL (2); CA (2); US (6); FR (2); CH (2); TU (2); IT (4)