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

Roche

Status and phase: Terminated; Phase 1

Conditions: Muscular Atrophy, Spinal

Treatments: Drug: RO6885247; Drug: placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02240355

2014-002246-41 (EudraCT Number)

BP29420

Details and patient eligibility

About

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).

Enrollment: 9 patients

Sex: All

Ages: Under 55 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: Double Blind

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9 participants in 3 patient groups

Part 1

Experimental group

Description:

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

Treatment:

Drug: placebo

Drug: RO6885247

Part 2

Experimental group

Description:

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

Treatment:

Drug: placebo

Drug: RO6885247

Part 3

Experimental group

Description:

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

Treatment:

Drug: RO6885247