An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)

An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)

Quick Info:

Status:
Currently Recruiting
Estimated Enrollment:
84
Phase:
1
Treatment Type:
Single and Multiple Ascending Dose
Trial Type:
Double Blind, Placebo Controlled
Sponsor:
Biogen
Primary Investigator:
Contact Information:

Enrollment Criteria:

Forced Ventilation

Breathing Ability

Percent lung function (FVC) or (SVC)
≥50%
Months/Onset

Months Since Onset

Number of months since first
symptoms of ALS
n/a
BiPap Allowed

Non-Invasive Ventilation (NIV)

Can PALS use a BiPAP in the trial?
Maybe
DPS Allowed

Diaphragm Pacer (DPS)

Can PALS use a DPS in the trial?
No
Edaravone Usage

Edaravone Usage

Can a PALS use edaravone (Radicut/Radicava)
while enrolled in the trial?
Unknown

Update Notes:

6/10/2019Recruitment status at locations updated
5/31/2019No significant updates
5/24/2019Location recruitment status updated
5/6/2019Location recruitment status updated
4/29/2019Location recruitment status updated
4/22/2019Location recruitment status updated
4/16/2019Location recruitment status updated
4/8/2019Location recruitment status updated
3/20/2019Location recruitment status updated
3/11/2019Recruitment status updated
9/3/2018Recruitment status updated
5/31/2018No significant updates
4/20/2018No significant updates
3/16/2018No significant updates
2/15/2018Recruitment status updated
1/19/2018No significant updates
12/5/2017No significant updates
11/13/2017No significant updates
10/5/2017No significant updates
9/15/2017Recruitment status at location updated
8/18/2017No significant updates
7/12/2017No significant updates
6/16/2017No significant updates
5/1/2017No significant updates
3/29/2017No significant updates
3/6/2017Recruitment status updated
3/6/2017No significant updates
10/12/2016No significant changes
9/9/2016Recruitment status updated
8/18/2016Recruitment status updated
7/12/2016Recruitment status updated
6/8/2016No Significant Updates
4/11/2016Sponsor/Collaborator updated.
2/3/2016Recruitment status updated.
12/11/2015Clinical trial added.

Locations:

Barrow Neurological Institute, Phoenix, 85013
University of California San Diego Medical Center, La Jolla, 92093
Forbes Norris MDA/ALS Research Center, San Francisco, 94115
Bioclinica Research, Orlando, 32806
The Emory Clinic, Atlanta, 30322
Johns Hopkins Hospital, Baltimore, 21287
Massachusetts General Hospital, MA, Boston, 02114
Washington University School of Medicine, St. Louis, 63110
Alliance for Multispecialty Research, Knoxville, 37920
UZ Leuven, Leuven, 3000
Sunnybrook Health Sciences Centre, Toronto, M4N 3M5
Montreal Neurological Institute, Montreal, H3A 2B4
Hospitalier Pitie-Salpetriere, Paris, 75013
Universitaetsklinikum Ulm, Ulm, Baden Wuerttemberg, 89081
Norrlands universitetssjukhus, Umeå, 90185
Sheffield Institute for Translational Neuroscience, Sheffield, South Yorkshire, S10 2HQ

Other Information:

Purpose: The primary objective of Parts A and B of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 in adult with ALS. The primary objective of Part C of this study is to evaluate the clinical efficacy of BIIB067 administered to adult participants with ALS and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objective of Parts A and B of this study is to evaluate the effects of BIIB067 on levels of SOD1 protein in the cerebrospinal fluid (CSF). The secondary objectives of Part C are to evaluate the safety, tolerability, and pharmacodynamic (PD) effects of BIIB067 administered to adult participants with ALS and confirmed SOD1 mutation.
Eligibility: 18 years or older, all genders, no healthy volunteers. Possible, probable, or definite ALS. It is not a requirement to have an SOD1 mutation to enroll in this study.
Details:
Collaborator(s):
News Articles and Summaries:
ALS Forum:
First Published on Clinicaltrials.gov: 11/24/2015
ClinicalTrials.gov ID: NCT02623699
Trial Protocol as Published on Clinicaltrials.gov:
ClinicalTrials.gov