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Clinical Trials/NCT02240355
NCT02240355
Terminated
Phase 1

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).

Hoffmann-La Roche9 sites in 7 countries9 target enrollmentStarted: November 2014Last updated:
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ConditionsMuscular Atrophy, Spinal
InterventionsRO6885247placebo
DrugsRO6885247placebo

Overview

Phase
Phase 1
Status
Terminated
Enrollment
9
Locations
9
Primary Endpoint
Safety: Incidence of adverse events (AEs)
OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Overview

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).

Study Design

Study Type
Interventional
Allocation
Randomized
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Eligibility Criteria

Ages
— to 55 Years (Child, Adult)
Sex
All
Accepts Healthy Volunteers
No

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.

Arms & Interventions

Part 1

Experimental

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

Intervention: RO6885247 (Drug)

Part 1

Experimental

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

Intervention: placebo (Drug)

Part 2

Experimental

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

Intervention: RO6885247 (Drug)

Part 2

Experimental

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

Intervention: placebo (Drug)

Part 3

Experimental

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

Intervention: RO6885247 (Drug)

Outcomes

Primary Outcomes

Safety: Incidence of adverse events (AEs)

Time Frame: Up to 20 weeks

Secondary Outcomes

  • Pharmacodynamics: SMN protein levels in blood(Up to 20 weeks)
  • Pharmacokinetics: RO6885247 plasma concentrations(Up to 16 weeks)
  • Pharmacodynamics: In vivo splicing modification of SMN2 mRNA 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)
  • Pharmacokinetics: RO6885247 exposure, area under the concentration-time curve (AUC-tau, over the 24-hour dosing interval)(Up to 12 weeks)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (9)

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