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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02240355
Recruitment Status : Terminated (There was an unexpected toxicology finding observed in the 39 week monkey study. Dosing was suspended, the study was put on hold and eventually terminated.)
First Posted : September 15, 2014
Last Update Posted : December 22, 2016
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Description
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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).

Condition or disease Intervention/treatment Phase
Muscular Atrophy, Spinal Drug: RO6885247 Drug: placebo Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: 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).
Study Start Date : November 2014
Actual Primary Completion Date : July 2015
Actual Study Completion Date : July 2015

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Part 1
Up to 2 cohorts of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
 Drug: RO6885247
oral solution

Drug: placebo
oral solution
Experimental: Part 2
1 cohort of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks
 Drug: RO6885247
oral solution

Drug: placebo
oral solution
Experimental: Part 3
1 cohort of patients, within each cohort patients will receive RO6885247 once daily for 12 weeks or 20 weeks
 Drug: RO6885247
oral solution


Outcome Measures
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Primary Outcome Measures :
  1. Safety: Incidence of adverse events (AEs) [ Time Frame: Up to 20 weeks ]

Secondary Outcome Measures :
  1. Pharmacokinetics: RO6885247 plasma concentrations [ Time Frame: Up to 16 weeks ]
  2. Pharmacokinetics: RO6885247 exposure, area under the concentration-time curve (AUC-tau, over the 24-hour dosing interval) [ Time Frame: Up to 12 weeks ]
  3. Pharmacodynamics: SMN protein levels in blood [ Time Frame: Up to 20 weeks ]
  4. Effect of RO6885247 on muscle electrophysiology, as assessed by Compound Muscle Action Potential (CMAP) [ Time Frame: Up to 20 weeks ]
  5. Effect of RO6885247 on Electrical Impedance Myography [ Time Frame: Up to 20 weeks ]
  6. Pharmacodynamics: In vivo splicing modification of SMN2 mRNA in blood [ Time Frame: Up to 20 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   up to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02240355


Locations
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Sponsors and Collaborators
Hoffmann-La Roche
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche
More Information
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Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT02240355    
Other Study ID Numbers: BP29420
2014-002246-41 ( EudraCT Number )
First Posted: September 15, 2014    Key Record Dates
Last Update Posted: December 22, 2016
Last Verified: December 2016
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
 Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases