Phase
Condition
Spinal Muscular Atrophy
Myasthenia Gravis (Chronic Weakness)
Muscular Dystrophy
Treatment
BIIB115
Salanersen
Salanersen-Matching Placebo
Clinical Study ID
Ages 6-55 All Genders Accepts Healthy Volunteers
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
Part A:
Male healthy participants aged 18 to 55 years, inclusive
Have a body mass index of 18 to 30 kilograms per meter square (kg/m^2), inclusive
Must be in good health as determined by the investigator, based on medical historyand screening evaluations
Part B:
Age 0.5 to 12 years old, inclusive, at the time of informed consent
Weight ≥7 kg at the time of informed consent
Genetic diagnosis of SMA (5q SMA homozygous survival motor neuron 1 (SMN1) genedeletion or mutation or compound heterozygous mutation)
Survival motor neuron 2 (SMN2) copy number ≥1
Must have received intravenous (IV) onasemnogene abeparvovec per the approved labelor per guidelines including the steroid regimen and monitoring specified therein
Treatment with onasemnogene abeparvovec ≥180 days prior to first BIIB115 dose
Potential for improvement due to suboptimal clinical status secondary to SMA, asdetermined by the Investigator
Part B LTE
Completion of the assessments in Part B
Meets age-appropriate institutional criteria for use of anesthesia/sedation, if useis planned for study procedures (as assessed by the Investigator and eitheranesthesiologist or pulmonologist).
Exclusion
Key Exclusion Criteria:
Part A:
Any reason, anatomical or otherwise (including abnormal hematology/coagulation),that presents increase of risk of complication from multiple lumbar puncture (LP)procedures required for dosing and CSF collection, per the investigator discretion
History of any clinically significant cardiac, endocrine, gastrointestinal,hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic,dermatologic, psychiatric, or renal disease, or other major disease, as determinedby the Investigator
Chronic, recurrent, or serious infection, as determined by the investigator, within 90 days prior to screening or between screening and Day -1
Current enrollment or a plan to enroll in any interventional clinical study of adrug, biologic, or device, in which an investigational treatment or approved therapyfor investigational use is administered within 3 months (or 5 half-lives of theagent, whichever is longer) prior to randomization
Part B:
Severe or serious AEs related to onasemnogene abeparvovec therapy that are ongoingduring Screening
Interval of <180 days between onasemnogene abeparvovec therapy and first BIIB115dose
Ongoing steroid treatment following onasemnogene abeparvovec at time of screening
History of drug induced liver injury or liver failure per Hy's law definition
History of thrombotic micrangiopathy
Treatment with any SMN2-splicing modifier (nusinersen or risdiplam) after receivingonasemnogene abeparvovec. Treatment with nusinersen <12 months from the first doseof BIIB115.
Any reason, anatomical or otherwise (including abnormal hematology/coagulation),that presents increase of risk of complication from the LP procedures, CSFcirculation, or safety assessments, including a history of hydrocephalus orimplanted shunt for CSF drainage.
Permanent ventilation, defined as tracheostomy or ≥16 hours ventilation /daycontinuously for >21 days in the absence of an acute reversible event
Part B LTE:
Any new condition or worsening of an existing condition that, according to theInvestigator, would make the participant unsuitable for inclusion, could interferewith the assessment of safety, or would compromise the ability of the participant toundergo study procedures.
Clinically significant abnormalities in hematology, blood chemistry parameters, orelectrocardiograms (ECGs) prior to first LTE visit that would make the participantunsuitable for inclusion as assessed by the Investigator.
Treatment with an approved SMN2-splicing modifier (nusinersen or risdiplam).
NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply.
Study Design
Study Description
Connect with a study center
Universitair Ziekenhuis Gent
Gent, 9000
BelgiumSite Not Available
Children's Hospital of Eastern Ontario
Ontario, K1H 8L1
CanadaSite Not Available
Hôpital Armand Trousseau
Paris, 75012
FranceSite Not Available
Universitatsklinikum Essen
Essen, 45147
GermanySite Not Available
Universitaetsklinikum Freiburg
Freiburg, 79106
GermanySite Not Available
Universitaetsklinikum Heidelberg
Heidelberg, 69120
GermanySite Not Available
Schneider Children's Medical Center
Petach-Tikvah, 4920235
IsraelSite Not Available
Fondazione Serena Onlus - Centro Clinico Nemo
Milano, 20162
ItalySite Not Available
Pediatric Neurology Unit, Catholic University
Rome, 00168
ItalySite Not Available
Kyungpook National University Hospital
Daegu, 700-721
Korea, Republic ofSite Not Available
Seoul National University Hospital
Seoul, 03080
Korea, Republic ofSite Not Available
Centre For Human Drug Reasearch
Leiden, 2333
NetherlandsSite Not Available
Centre For Human Drug Research
Leiden, 2333
NetherlandsSite Not Available
UMC Utrecht
Utrecht, 3584 CX
NetherlandsSite Not Available
Instytut Centrum Zdrowia Matki Polki Dept of Neurology
Lodz, 93-338
PolandSite Not Available
Instytut "Pomnik - Centrum Zdrowia Dziecka
Warsaw, 04-730
PolandSite Not Available
PRATIA S.A. MTZ Clinical Research Powered by Pratia
Warszawa, 02-172
PolandSite Not Available
Great Ormond Street Hospital for Children
Bloomsbury, WC1N 3JH
United KingdomSite Not Available
Sheffield Childrens Hospital
Sheffield, S10 2TH
United KingdomSite Not Available
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