A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 in adult and pediatric patients with spinal muscular atrophy.
- Conditions
- Spinal Muscular Atrophy (SMA)Therapeutic area: Diseases [C] - Nervous System Diseases [C10]MedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
- Registration Number
- EUCTR2016-004184-39-IT
- Lead Sponsor
- F. HOFFMANN - LA ROCHE LTD.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 24
- Males and females 12 to 60 years of age inclusive (at screening).
- Confirmed diagnosis of 5q-autosomal recessive SMA, including:
•Genetic confirmation of homozygous deletion or heterozygosity predictive of loss of function of the SMN1 gene,
•Clinical symptoms attributable to Type 2 or Type 3 SMA.
- Previous participation in a study with an SMN2-targeting antisense oligonucleotide or SMN2 splicing modifier other than RO7034067.
- Negative blood pregnancy test at screening (all women of childbearing potential, including those who have had a tubal ligation), and agreement to comply with measures to prevent pregnancy and restrictions on sperm donation.
For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures and agreement to refrain from donating sperm.
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Inability to meet study requirements
- Concomitant participation in any investigational drug or device study
- Previous participation in an SMN2-targeting antisense oligonucleotide or SMN2 splicing modifier study other than RO7034067 within 90 days prior to screening
- Previous participation in any investigational drug or device study, other than SMN2 targeting antisense oligonucleotide or SMN2 splicing modifier study, within 90 days prior to screening, or 5 half-lives of the drug, whichever is longer
- Any history of gene or cell therapy
- Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases as considered to be clinically significant by the Investigator
- Lactating women
- Suspicion of regular consumption of drugs of abuse
- Positive urine test for drugs of abuse or alcohol at the screening or Day - 1 visit
- Presence of clinically significant ECG abnormalities before study drug administration
- Presence of clinically significant cardiovascular, blood pressure, and heart rate measures
- History of malignancy if not considered cured
- Significant risk for suicidal behavior, in the opinion of the Investigator as assessed by the Columbia-Suicide Severity Rating Scale (C-SSRS)
- Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration
- Use of any OCT-2 and MATE substrates within 2-weeks before dosing
- Use of the following medications within 90 days prior to enrollment: riluzole, valproic acid, hydroxyurea, sodium phenylbutyrate, butyrate derivatives, creatine, carnitine, growth hormone, anabolic steroids, probenecid, agents anticipated to increase or decrease muscle strength, agents with known or presumed histone deacetylase (HDAC) inhibitory effect, and medications with known phototoxicity liabilities (e.g., oral or topical retinoids including over-the-counter formulations, amiodarone. Use of inhaled corticosteroids is allowed.
- Recently initiated treatment (within < 6 months prior to enrollment) with oral salbutamol or another ß2-adrenergic agonist taken orally is not allowed. Patients who have been on oral salbutamol (or another ß2-adrenergic agonist) for >= 6 months before enrollment and have shown good tolerance are allowed. The dose of ß2-adrenergic agonist should remain stable as much as possible for the duration of the study. Use of inhaled ß2-adrenergic agonists (e.g., for the treatment of asthma) is allowed.
- Any prior use of chloroquine, hydroxychloroquine, retigabin, vigabatrin or thioridazine is not allowed. Use of other medications known to or suspected of causing retinal toxicity within one year prior to enrollment is not allowed
- Clinically significant abnormalities in laboratory test results
- Donation or loss of blood >= 10% of blood volume within three months prior to screening
- Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to RO7034067 or to the constituents of its formulation
- Concomitant disease or condition that could interfere with, or treatment of which might interfere with, the conduct of the study, or that would, in the opinion of the Investigator, pose an unacceptable risk to the patient in this study
- Recent history (less than one year) of ophthalmological diseases
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method