Status:
TERMINATED
A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)
Lead Sponsor:
Hoffmann-La Roche
Conditions:
Muscular Atrophy, Spinal
Eligibility:
All Genders
Up to 55 years
Phase:
PHASE1
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 muscul...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
November 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2015
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT02240355
Start Date
November 1 2014
End Date
July 1 2015
Last Update
December 22 2016
Active Locations (26)
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1
Stanford, California, United States, 94305
2
Orlando, Florida, United States, 32827
3
Chicago, Illinois, United States, 60611-2605
4
Boston, Massachusetts, United States, 02115