ong-Term Evaluation of BIIB067

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 182

Inclusion Criteria

- Must have diagnosis of SOD1-ALS, and must have completed the End of
Study Visit for either Parts A, Band/or C of Study 233AS101 (i.e., were
not withdrawn).
- If taking riluzole, must be receiving a stable dose for =30 days prior to
Day 1 and expected to remain at that dose until the final study visit.
- For female participants of childbearing potential must agree to practice
effective contraception during the study and for 5 months after their last
dose of study treatment.
- Medically able to undergo the study procedures, and to adhere to the
visit schedule at the time of study entry, as determined by the
Investigator.
- Participants from Study 233AS101 Parts A and B must have a washout
=16 weeks between the last dose of study treatment received in Study
233AS101 and the first dose of BIIB067 received in the current Study
233AS102.
- If taking edaravone, participant must have initiated edaravone = 60
days (2 treatment cycles) prior to Day 1. Edaravone may not be administered on dosing
days.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 146
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 37

Exclusion Criteria

- History of allergies to a broad range of anaesthetics.
- Presence of risk for increased or uncontrolled bleeding and/or risk of
bleeding that is not managed optimally and could place a participant at
an increased risk for bleeding during or after an LP procedure. These
risks could include, but are not limited to, anatomical factors at or near
the LP site (e.g., vascular abnormalities, neoplasms, or other
abnormalities) and underlying disorders of the coagulation cascade,
platelet function, or platelet count (e.g., hemophilia, Von Willebrand's
disease, liver disease).
- Presence of an implanted shunt for the drainage of CSF or an implanted
CNS catheter.
- Prior or current treatment with small interfering ribonucleic acid
(RNA), stem cell therapy, or gene therapy.
- Treatment with another investigational drug, biological agent
(excluding BIIB067), or device within 1 month or 5 half-lives of study
agent, whichever is longer.
- Current or anticipated need, in the opinion of the Investigator, of a
diaphragm pacing system (DPS) during the study period.
- Female subjects who are pregnant or currently breastfeeding.
- Current enrollment in any other interventional study.
- Current or recent (within 1 month) use, or anticipated need, in the
opinion of the Investigator, of copper (II) (diacetyl-bis (N4-
methylthiosemicarbazone)) or pyrimethamine.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of the study is to evaluate the long-term safety<br>and tolerability of BIIB067 in participants with ALS and confirmed<br>superoxide dismutase 1 (SOD-1) mutation.;Secondary Objective: The secondary objectives are to evaluate the pharmacokinetic (PK) and<br>pharmacodynamic (PD) profiles and effects on disease progression of<br>BIIB067 administered to subjects with ALS and confirmed SOD1<br>mutation.;Primary end point(s): Numero di partecipanti con eventi avversi ed eventi avversi gravi;Timepoint(s) of evaluation of this end point: Up to Week 248

Secondary Outcome Measures

Name	Time	Method

Updated 7/3/2017

ong-Term Evaluation of BIIB067

Recruitment & Eligibility

Study & Design

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