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

Phase 1
Terminated
Conditions
Muscular Atrophy, Spinal
Interventions
Drug: RO6885247
Drug: placebo
Registration Number
NCT02240355
Lead Sponsor
Hoffmann-La Roche
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).

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
9
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
Read More
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
Read More

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Part 1RO6885247Up to 2 cohorts of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
Part 1placeboUp to 2 cohorts of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
Part 2RO68852471 cohort of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
Part 2placebo1 cohort of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
Part 3RO68852471 cohort of patients, within each cohort patients will receive RO6885247 once daily for 12 weeks or 20 weeks
Primary Outcome Measures
NameTimeMethod
Safety: Incidence of adverse events (AEs)Up to 20 weeks
Secondary Outcome Measures
NameTimeMethod
Pharmacodynamics: SMN protein levels in bloodUp to 20 weeks
Pharmacokinetics: RO6885247 plasma concentrationsUp to 16 weeks
Pharmacodynamics: In vivo splicing modification of SMN2 mRNA in bloodUp 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 MyographyUp to 20 weeks
Pharmacokinetics: RO6885247 exposure, area under the concentration-time curve (AUC-tau, over the 24-hour dosing interval)Up to 12 weeks

Trial Locations

Locations (9)

Ch Pitie Salpetriere; Institut de Myologie

🇫🇷

Paris, France

Drottning Silvias Barn- och ungdomssjukhus; Kliniken för barnmedicin

🇸🇪

Goeteborg, Sweden

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

🇹🇷

Ankara, Turkey

Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile

🇮🇹

Roma, Lazio, Italy

UCL; GAP Unit, Institute of Child Health, UCL

🇬🇧

London, United Kingdom

UMC Utrecht; Polkliniek Neuromusculaire ziekten

🇳🇱

Utrecht, Netherlands

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

🇮🇹

Milano, Lombardia, Italy

MRC Neuromuscular Centre - Institute of Genetic Medicine

🇬🇧

Newcastle upon Tyne, United Kingdom

Universitäts-Kinderspitalbeider Basel_Abteilung für Neuro- und Entwicklungspädiatrie

🇨🇭

Basel, Switzerland

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