Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults C9ORF72-Associated Amyotrophic Lateral Sclerosis (ALS)
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT04288856 |
|
Recruitment Status :
Terminated
(There was no evidence of benefit across efficacy endpoints in the randomized trial, 245AS101. Accordingly, Biogen has made the difficult decision to discontinue the BIIB078 program including this open label extension trial, 245AS102.)
First Posted : February 28, 2020
Last Update Posted : February 1, 2023
|
Sponsor:
Biogen
Information provided by (Responsible Party):
Biogen
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Brief Summary:
The primary objective is to evaluate the long-term safety and tolerability of BIIB078 in participants with chromosome 9 open reading frame 72-amyotrophic lateral sclerosis (C9ORF72-ALS).
The secondary objective is to evaluate the pharmacokinectic (PK) of BIIB078 in participants with C9ORF72-ALS.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Amyotrophic Lateral Sclerosis | Drug: BIIB078 | Phase 1 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 75 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | None (Open Label) |
| Masking Description: | During the blinded loading period, the following individuals will be masked:
After the loading period has been completed, subsequent doses will be open-label. |
| Primary Purpose: | Treatment |
| Official Title: | An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis |
| Actual Study Start Date : | April 28, 2020 |
| Actual Primary Completion Date : | May 3, 2022 |
| Actual Study Completion Date : | May 3, 2022 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Amyotrophic lateral sclerosis
Juvenile primary lateral sclerosis
MedlinePlus related topics:
Amyotrophic Lateral Sclerosis
| Arm | Intervention/treatment |
|---|---|
|
Experimental: Cohort A: BIIB078 First Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
|
Drug: BIIB078
Administered as specified in the treatment arm. |
|
Experimental: Cohort B: BIIB078 Second Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
|
Drug: BIIB078
Administered as specified in the treatment arm. |
|
Experimental: Cohort C: BIIB078 Third Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
|
Drug: BIIB078
Administered as specified in the treatment arm. |
|
Experimental: Possible Cohort D: BIIB078 Fourth Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
|
Drug: BIIB078
Administered as specified in the treatment arm. |
Primary Outcome Measures :
- Number of Participants with Adverse Events (AEs) [ Time Frame: Baseline up to Day 785 ]AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention.
- Number of Participants with Serious Adverse Events (SAEs) [ Time Frame: Screening up to Day 785 ]An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect or any other situation according to medical or scientific judgment.
Secondary Outcome Measures :
- Serum concentration of BIIB078 [ Time Frame: Baseline and at multiple time points up to Day 729 ]
- Cerebrospinal Fluid (CSF) concentration of BIIB078 [ Time Frame: Baseline and at multiple time points up to Day 729 ]
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Key Inclusion Criteria:
- Participants must have completed study NCT03626012 through the first follow-up clinic visit that follows the final dosing visit without missing more than 1 dose of study treatment.
- Participants taking concomitant riluzole at study entry must be on a stable dose for ≥30 days prior to the first dose of study treatment (Day 1). Participants taking concomitant riluzole must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that riluzole should be discontinued for medical reasons, in which case it may not be restarted during the study.
- Participants taking concomitant edaravone at study entry must be on a stable dose for ≥60 days prior to the first dose of study treatment (Day 1). Participants taking concomitant edaravone must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. Edaravone may not be administered on dosing days of this study.
Key Exclusion Criteria:
- History of drug abuse or alcoholism ≤6 months before study enrollment that would limit participation in the study, as determined by the Investigator.
- Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter.
- History of or positive test result at Screening for human immunodeficiency virus. The requirement for testing at Screening may be omitted if it is not permitted by local regulations.
- Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs) or biological agent within 1 month of Screening or 5 half-lives of study agent, whichever is longer.
Note: Other protocol-specific inclusion/exclusion criteria may apply.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04288856
Locations
| United States, California | |
| Research Site | |
| La Jolla, California, United States, 92037 | |
| Research Site | |
| Los Angeles, California, United States, 90048 | |
| Research Site | |
| Palo Alto, California, United States, 94304 | |
| United States, Florida | |
| Research Site | |
| Jacksonville, Florida, United States, 32224 | |
| Research Site | |
| Miami, Florida, United States, 33136 | |
| United States, Georgia | |
| Research Site | |
| Atlanta, Georgia, United States, 30322 | |
| United States, Maryland | |
| Research Site | |
| Baltimore, Maryland, United States, 21287 | |
| United States, Massachusetts | |
| Research Site | |
| Boston, Massachusetts, United States, 02114 | |
| United States, Missouri | |
| Research Site | |
| Saint Louis, Missouri, United States, 63110 | |
| United States, Nebraska | |
| Research Site | |
| Lincoln, Nebraska, United States, 68506 | |
| United States, New York | |
| Research Site | |
| New York, New York, United States, 10032 | |
| United States, Tennessee | |
| Research Site | |
| Knoxville, Tennessee, United States, 37920 | |
| Canada, Alberta | |
| Research Site | |
| Calgary, Alberta, Canada, T2N 1N4 | |
| Research Site | |
| Edmonton, Alberta, Canada, T6G 2B7 | |
| Canada, Ontario | |
| Research Site | |
| Toronto, Ontario, Canada, M4N 3M5 | |
| Canada, Quebec | |
| Research Site | |
| Montreal, Quebec, Canada, H3A 2B4 | |
| Netherlands | |
| Research Site | |
| Utrecht, Netherlands, 3508 GA | |
| Switzerland | |
| Research Site | |
| St. Gallen, Switzerland, 9007 | |
| United Kingdom | |
| Research Site | |
| London, Greater London, United Kingdom, NW1 2PG | |
| Research Site | |
| London, Greater London, United Kingdom, SE5 9RS | |
| Research Site | |
| Sheffield, South Yorkshire, United Kingdom, S10 2HQ | |
Sponsors and Collaborators
Biogen
Investigators
| Study Director: | Medical Director | Biogen |
| Responsible Party: | Biogen |
| ClinicalTrials.gov Identifier: | NCT04288856 |
| Other Study ID Numbers: |
245AS102 2019-004798-14 ( EudraCT Number ) |
| First Posted: | February 28, 2020 Key Record Dates |
| Last Update Posted: | February 1, 2023 |
| Last Verified: | January 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/ |
| URL: | https://vivli.org/ |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Additional relevant MeSH terms:
|
Motor Neuron Disease Amyotrophic Lateral Sclerosis Sclerosis Disease Progression Pathologic Processes Neurodegenerative Diseases Nervous System Diseases |
Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases Disease Attributes |