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

Phase 1

• Conditions: Spinal Muscular Atrophy; MedDRA version: 18.0Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2014-002246-41-ES
• Lead Sponsor: Roche Farma S.A., en nombre de F. Hoffmann-La Roche Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-03-03
• Last Update Posted: 9 January 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 64

Inclusion Criteria

- Males and females aged below 7 months (inclusive) or 2 to 55 years (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 two 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 patient 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 52
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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
- History of cell therapy
- Hospitalization for pulmonary event within the last 2 months or planned at the time of screening (for patients aged 2-55 years)
- 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 patient 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.
- Ascertained or presumptive hypersensitivity (e.g. anaphylactic reaction) to RO6885247 or to the constituents of its formulation
- Patients aged 7 months or below requiring invasive ventilation, tracheotomy, day-time non-invasive ventilation or patients with nocturnal hypoxemia; patients having had severe pneumonia and have not fully recovered their pulmonary function at the time of screening; patients with contractures or hip subluxation/dislocation at birth; patients with non-SMA related morbidities.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified