Muscular Atrophy, SpinalPhase 1NCT02240355
A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)
A Phase 1 study testing RO6885247 in people with Muscular Atrophy, Spinal, run by Hoffmann-La Roche at 26 sites in 9 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages Up to 55 years
- Conditions the study is for
- Muscular Atrophy, Spinal
- What is being tested?
- RO6885247
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 9 participants
- Where
- 26 sitesin 9 countries
- Study dates
- Nov 2014to Jul 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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 locations and contacts
26 sites in 9 countries.
26 sites in 9 countries
United States6 sites
Study site
Boston, Massachusetts, 02115
Study site
Chicago, Illinois, 60611-2605
Study site
New York, New York, 10032
Study site
Orlando, Florida, 32827
Study site
St Louis, Missouri, 63110
Study site
Stanford, California, 94305
Italy4 sites
Fondazione IRCCS Istituto Neurologico "Carlo Besta"; UO di Neurologia dello Sviluppo
Milan, Lombardy, 20133
Study site
Milan, Lombardy, 20133
Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile
Rome, Lazio, 00168
Study site
Rome, Lazio, 00168
United Kingdom4 sites
UCL; GAP Unit, Institute of Child Health, UCL
London, WC1N 1EH
Study site
London, WC1N 1EH
MRC Neuromuscular Centre - Institute of Genetic Medicine
Newcastle upon Tyne, NE1 3BZ
Study site
Newcastle upon Tyne, NE1 3BZ
France2 sites
Ch Pitie Salpetriere; Institut de Myologie
Paris, 75013
Study site
Paris, 75013
Netherlands2 sites
UMC Utrecht; Polkliniek Neuromusculaire ziekten
Utrecht, 3584 CX
Study site
Utrecht, 3584 CX
Sweden2 sites
Drottning Silvias Barn- och ungdomssjukhus; Kliniken för barnmedicin
Gothenburg, 41685
Study site
Gothenburg, 41685
Switzerland2 sites
Universitäts-Kinderspitalbeider Basel_Abteilung für Neuro- und Entwicklungspädiatrie
Basel, 4005
Study site
Basel, 4005
Turkey (Türkiye)2 sites
Hacettepe University, School of Medicine; Pediatrics Department; Pediatrics Child Neurology Unit
Ankara, 06100
Study site
Ankara, 06100
About this study
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).
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).
- Conditions
- Muscular Atrophy, Spinal
- Being tested
- RO6885247, placebo
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RO6885247Drug
oral solution
placeboDrug
oral solution
| Study group | Role | What they receive |
|---|---|---|
| Part 1 | Experimental | Up to 2 cohorts of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks Receives: Drug: RO6885247; Drug: placebo |
| Part 2 | Experimental | 1 cohort of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks Receives: Drug: RO6885247; Drug: placebo |
| Part 3 | Experimental | 1 cohort of patients, within each cohort patients will receive RO6885247 once daily for 12 weeks or 20 weeks Receives: Drug: RO6885247 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Incidence of adverse events (AEs)
Measured: Up to 20 weeks
Secondary outcomes (6)
Pharmacokinetics: RO6885247 plasma concentrations
Measured: Up to 16 weeks
Pharmacokinetics: RO6885247 exposure, area under the concentration-time curve (AUC-tau, over the 24-hour dosing interval)
Measured: Up to 12 weeks
Pharmacodynamics: SMN protein levels in blood
Measured: Up to 20 weeks
Effect of RO6885247 on muscle electrophysiology, as assessed by Compound Muscle Action Potential (CMAP)
Measured: Up to 20 weeks
Effect of RO6885247 on Electrical Impedance Myography
Measured: Up to 20 weeks
Pharmacodynamics: In vivo splicing modification of SMN2 mRNA in blood
Measured: Up to 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2014 (estimated)
- Study startNov 2014
- Primary completionJul 2015
- Study completionJul 2015
Why it stopped: 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.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02240355
- Sponsor's protocol ID
- BP29420
- Other IDs
- 2014-002246-41, 2014
- Status last verified
- Dec 2016
- First submitted
- 11 Sep 2014
- Last updated
- 22 Dec 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Dec 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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).
- ClinicalTrials.gov ID
- NCT02240355
- Acronym / sponsor ID
- BP29420
- Conditions
- Muscular Atrophy, Spinal
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: 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.
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- Nov 2014
- Primary completion
- Jul 2015 (actual)
- Study completion
- Jul 2015 (actual)
- First posted
- 15 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Part 1 | Experimental | Drug: RO6885247, Drug: placeboUp to 2 cohorts of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks |
| Part 2 | Experimental | Drug: RO6885247, Drug: placebo1 cohort of patients, within each cohort patients will receive either RO6885247 or placebo once daily for 12 weeks |
| Part 3 | Experimental | Drug: RO68852471 cohort of patients, within each cohort patients will receive RO6885247 once daily for 12 weeks or 20 weeks |
Who may qualify
- Ages
- Up to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RO6885247oral solution | Drug | Part 1, Part 2, Part 3 |
| placebooral solution | Drug | Part 1, Part 2 |
Primary outcome
Safety: Incidence of adverse events (AEs)
Time frame: Up to 20 weeks
6 secondary outcomes
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
Results and safety
The study was stopped early (reason given: 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.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
26 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RO6885247 in people with Muscular Atrophy, Spinal, run by Hoffmann-La Roche at 26 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Dec 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, Italy, United Kingdom, Canada, France and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: 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.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety: Incidence of adverse events (AEs) (time frame: Up to 20 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Dec 2016; AzTrials last refreshed its copy on 8 Nov 2026.