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A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 in Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis

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ClinicalTrials.gov Identifier: NCT03626012
Recruitment Status : Recruiting
First Posted : August 10, 2018
Last Update Posted : November 19, 2018
Sponsor:
Information provided by (Responsible Party):
Biogen

Brief Summary:
The primary objective of this study is to evaluate the safety and tolerability of BIIB078 in adults with C9ORF72-ALS. The secondary objective of this study is to evaluate the pharmacokinetic profile of BIIB078.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: BIIB078 Drug: Placebo Phase 1

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis
Actual Study Start Date : September 10, 2018
Estimated Primary Completion Date : July 11, 2022
Estimated Study Completion Date : July 11, 2022


Arm Intervention/treatment
Experimental: Cohort 1: BIIB078 First Dosage
BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days.
Drug: BIIB078
Administered as specified in the treatment arm.

Experimental: Cohort 2: BIIB078 Second Dosage
BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days.
Drug: BIIB078
Administered as specified in the treatment arm.

Experimental: Cohort 3: BIIB078 Third Dosage
BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days.
Drug: BIIB078
Administered as specified in the treatment arm.

Experimental: Cohort 4: BIIB078 Fourth Dosage
BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days.
Drug: BIIB078
Administered as specified in the treatment arm.

Experimental: Cohort 5: BIIB078 Fifth Dosage
BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days.
Drug: BIIB078
Administered as specified in the treatment arm.

Experimental: Cohort 6: BIIB078 Sixth Dosage
BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days.
Drug: BIIB078
Administered as specified in the treatment arm.

Placebo Comparator: Cohorts 1-6: Placebo
Matching placebo will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days.
Drug: Placebo
Administered as specified in the treatment arm.




Primary Outcome Measures :
  1. Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline through End of Study (Approximately Day 239) ]
    An AE is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death, places participant at immediate risk of death, requires initial or prolonged inpatient hospitalization, results in persistent or significant disability/incapacity, results in congenital anomaly, is a medically important event.


Secondary Outcome Measures :
  1. Serum BIIB078 Concentration [ Time Frame: Baseline and at multiple time points up to Day 176 ]
  2. Area Under the Concentration-Time Curve (AUC) from Time 0 to Infinity (AUCinf) [ Time Frame: Baseline and at multiple time points up to Day 176 ]
  3. AUC from Time 0 to Time of the Last Measurable Concentration (AUClast) [ Time Frame: Baseline and at multiple time points up to Day 176 ]
  4. Maximum Observed Concentration (Cmax) [ Time Frame: Baseline and at multiple time points up to Day 176 ]
  5. Time to Reach Cmax (Tmax) [ Time Frame: Baseline and at multiple time points up to Day 176 ]
  6. Terminal Elimination Half-Life (t 1/2) [ Time Frame: Baseline and at multiple time points up to Day 176 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Ability of the subject to understand the purpose and risks of the study, to provide signed and dated informed consent, and to authorize the use of confidential health information in accordance with national and local subject privacy regulations; or, in the event of the subject's physical incapacity to sign, to confirm that understanding and consent orally to a legally authorized representative (LAR) for the express purpose of having said informed consent and authorization signed on his/her behalf.
  • All subjects of childbearing potential must agree to practice highly effective contraception during the study and be willing and able to continue contraception for 5 months after their last dose of study treatment.
  • Must meet the possible, laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria and have documentation of a clinical genetic test demonstrating the presence of a pathogenic mutation in C9ORF72.
  • Slow vital capacity (SVC) ≥ 50% of predicted value as adjusted for sex, age, and height (from the sitting position).
  • Subjects 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).
  • Subjects 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).
  • ALS Cognitive Behavioral Screen (ALS-CBS) score ≥ 11 for the cognitive portion; ≥ 33 for the behavioral portion.
  • 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.
  • Screening values of coagulation parameters including platelet count, international normalized ratio (INR), prothrombin time (PT), and activated partial thromboplastin time (APTT) should be within normal ranges.
  • Has an informant/caregiver who, in the Investigator's judgment, has frequent and sufficient contact with the subject as to be able to provide accurate information about the subject's cognitive and functional abilities at Screening.

Key Exclusion Criteria:

  • History of drug abuse or alcoholism ≤ 6 months of Screening that would limit participation in the study, as determined by the Investigator.
  • Tracheostomy.
  • History of or positive test result at Screening for human immunodeficiency virus. .
  • History of, or positive test result at Screening for, hepatitis C virus antibody.
  • Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of study agent, whichever is longer.
  • Treatment with anti-platelet or anticoagulant therapy ≤ 14 days before Screening (with the exception of aspirin ≤ 325 mg/day) or anticipated use during the study.
  • Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system during the study period.
  • Female subjects who are pregnant or currently breastfeeding.

NOTE: Other protocol defined 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): NCT03626012


Contacts
Contact: US Biogen Clinical Trial Center 866-633-4636 clinicaltrials@biogen.com
Contact: Global Biogen Clinical Trial Center clinicaltrials@biogen.com

Locations
United States, California
Research Site Active, not recruiting
La Jolla, California, United States, 92037
United States, Maryland
Research Site Active, not recruiting
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Research Site Active, not recruiting
Boston, Massachusetts, United States, 02114
Research Site Recruiting
Cambridge, Massachusetts, United States, 02142
United States, Tennessee
Research Site Active, not recruiting
Knoxville, Tennessee, United States, 37920
Sponsors and Collaborators
Biogen
Investigators
Study Director: Medical Director Biogen

Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT03626012     History of Changes
Other Study ID Numbers: 245AS101
2017-000294-36 ( EudraCT Number )
First Posted: August 10, 2018    Key Record Dates
Last Update Posted: November 19, 2018
Last Verified: November 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases