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

This study has been 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.)
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche
ClinicalTrials.gov Identifier:
NCT02240355
First received: September 11, 2014
Last updated: December 20, 2016
Last verified: December 2016
  Purpose
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 Intervention Phase
Muscular Atrophy, Spinal
Drug: RO6885247
Drug: placebo
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, 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).

Resource links provided by NLM:


Further study details as provided by Hoffmann-La Roche:

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

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

Enrollment: 9
Study Start Date: November 2014
Study Completion Date: July 2015
Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02240355

  Show 26 Study Locations
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT02240355     History of Changes
Other Study ID Numbers: BP29420
2014-002246-41 ( EudraCT Number )
Study First Received: September 11, 2014
Last Updated: December 20, 2016

Additional relevant MeSH terms:
Atrophy
Muscular Atrophy, Spinal
Muscular Atrophy
Pathological Conditions, Anatomical
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Signs and Symptoms

ClinicalTrials.gov processed this record on March 22, 2017