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出境医 / 临床实验 / A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis
Study Description
Brief Summary:
The primary objective of this study is to evaluate the safety, tolerability of single-ascending doses of BIIB100 in adults with amyotrophic lateral sclerosis (ALS). The secondary objective of the study is to characterize the pharmacokinetic profile of BIIB100.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Amyotrophic Lateral Sclerosis | Drug: BIIB100 Drug: Placebo | Phase 1 |
Study Design
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 48 participants |
| Allocation: | Randomized |
| Intervention Model: | Sequential Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 1, Double-Blind, Placebo-Controlled, Single-Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adult Participants With Amyotrophic Lateral Sclerosis |
| Actual Study Start Date : | May 30, 2019 |
| Estimated Primary Completion Date : | June 14, 2021 |
| Estimated Study Completion Date : | June 14, 2021 |
Arms and Interventions
| Arm | Intervention/treatment |
|---|---|
|
Experimental: Cohort 1: BIIB100 Dose 1
Participants will receive single oral dose of BIIB100 on Day 1.
|
Drug: BIIB100
Administered as specified in the treatment arm.
|
|
Experimental: Cohort 2: BIIB100 Dose 2
Participants will receive single oral dose of BIIB100 on Day 1.
|
Drug: BIIB100
Administered as specified in the treatment arm.
|
|
Experimental: Cohort 3: BIIB100 Dose 3
Participants will receive single oral dose of BIIB100 on Day 1.
|
Drug: BIIB100
Administered as specified in the treatment arm.
|
|
Experimental: Cohort 4: BIIB100 Dose 4
Participants will receive single oral dose of BIIB100 on Day 1.
|
Drug: BIIB100
Administered as specified in the treatment arm.
|
|
Experimental: Cohort 5: BIIB100 Dose 5
Participants will receive single oral dose of BIIB100 on Day 1.
|
Drug: BIIB100
Administered as specified in the treatment arm.
|
|
Experimental: Cohort 6: BIIB100 Dose 6
Participants will receive single oral dose of BIIB100 on Day 1.
|
Drug: BIIB100
Administered as specified in the treatment arm.
|
|
Placebo Comparator: Cohort 1-6: Matching Placebo
Participants will receive single oral dose of matching placebo on Day 1.
|
Drug: Placebo
Administered as specified in the treatment arm.
|
Outcome Measures
Primary Outcome Measures :
- Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Screening (Day -28 ) up to Day 15 ]An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, or is a medically important event.
Secondary Outcome Measures :
- Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]BIIB100 will be measured in the plasma.
- Area Under the Concentration-Time Curve From Time 0 to Infinity (AUCinf) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]BIIB100 will be measured in the plasma.
- Maximum Observed Concentration (Cmax) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]BIIB100 will be measured in the plasma.
- Time to Reach Cmax (Tmax) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]BIIB100 will be measured in the plasma.
- Terminal Elimination Half-life (t1/2) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]BIIB100 will be measured in the plasma.
- Apparent Clearance (CL/F) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]BIIB100 will be measured in the plasma.
- Apparent Volume of Distribution During the Terminal Elimination (Vz/F) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]BIIB100 will be measured in the plasma.
Eligibility Criteria
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Key Inclusion Criteria:
- Must meet the laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria.
- Participants taking concomitant riluzole at study entry must be on a stable dose for greater than or equals to (>=) 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).
- Adequate respiratory function as indicated by slow vital capacity (SVC) >= 65% of predicted value as adjusted for sex, age, and height (from the sitting position).
Key Exclusion Criteria:
- Ongoing medical condition (e.g., wasting or cachexia, severe anemia) that would, in the opinion of the Investigator, interfere with the conduct or assessments of the study.
- Significant cognitive impairment or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression less than or equals to (<=) 90 days of Screening, which in the opinion of the Investigator would interfere with the study procedures.
- Treatment with drugs that are transported by Breast Cancer Resistance Protein (BCRP) and P-glycoprotein (P-gp) including, but not limited to, rosuvastatin, sulfasalazine, dabigatran, digoxin and fexofenadine.
- Current enrollment or plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy for investigational use is administered within 30 days or 5 half-lives of the agent, whichever is longer, prior to the Baseline Visit (pre-dose on Day 1). Participation in a noninterventional study focused on ALS natural history may be allowed at the discretion of the Investigator and after consultation with the Sponsor.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Contacts and Locations
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, Arizona | |
| Barrow Neurological Institute | Recruiting |
| Phoenix, Arizona, United States, 85013 | |
| Contact Fulton.Research@DignityHealth.org | |
| United States, California | |
| University of California San Diego Medical Center | Recruiting |
| San Diego, California, United States, 92121 | |
| Contact: Rosemarie Previte 858-246-1319 rprevite@ucsd.edu | |
| United States, Florida | |
| Mayo Clinic Hospital | Recruiting |
| Jacksonville, Florida, United States, 32224 | |
| Contact 904-953-6912 mayofloridaALSresearch@mayo.edu | |
| University of South Florida | Recruiting |
| Tampa, Florida, United States, 33612 | |
| United States, Maryland | |
| Johns Hopkins University, Dept of Neurology | Recruiting |
| Baltimore, Maryland, United States, 21205 | |
| Contact 410-502-0495 emosmil1@jhmi.edu | |
| United States, Massachusetts | |
| Research Site | Recruiting |
| Boston, Massachusetts, United States, 21219 | |
| United States, Missouri | |
| Research Site | Recruiting |
| Saint Louis, Missouri, United States, 63110 | |
| United States, Nebraska | |
| Research Site | Recruiting |
| Lincoln, Nebraska, United States, 68506-2960 | |
| United States, Tennessee | |
| Alliance for Multispecialty Research NOCCR/VRG | Recruiting |
| Knoxville, Tennessee, United States, 37920 | |
| Contact: Alliance for Multispeciality Research NOCCR/VRG 865-305-9100 ext 302 or 303 | |
Sponsors and Collaborators
Biogen
Investigators
| Study Director: | Medical Director | Biogen |
| Tracking Information | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| First Submitted Date ICMJE | May 8, 2019 | ||||||||
| First Posted Date ICMJE | May 10, 2019 | ||||||||
| Last Update Posted Date | March 22, 2021 | ||||||||
| Actual Study Start Date ICMJE | May 30, 2019 | ||||||||
| Estimated Primary Completion Date | June 14, 2021 (Final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures ICMJE |
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Screening (Day -28 ) up to Day 15 ] An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, or is a medically important event.
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| Original Primary Outcome Measures ICMJE | Same as current | ||||||||
| Change History | |||||||||
| Current Secondary Outcome Measures ICMJE |
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| Original Secondary Outcome Measures ICMJE | Same as current | ||||||||
| Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title ICMJE | A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis | ||||||||
| Official Title ICMJE | A Phase 1, Double-Blind, Placebo-Controlled, Single-Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adult Participants With Amyotrophic Lateral Sclerosis | ||||||||
| Brief Summary | The primary objective of this study is to evaluate the safety, tolerability of single-ascending doses of BIIB100 in adults with amyotrophic lateral sclerosis (ALS). The secondary objective of the study is to characterize the pharmacokinetic profile of BIIB100. | ||||||||
| Detailed Description | Not Provided | ||||||||
| Study Type ICMJE | Interventional | ||||||||
| Study Phase ICMJE | Phase 1 | ||||||||
| Study Design ICMJE | Allocation: Randomized Intervention Model: Sequential Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
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| Condition ICMJE | Amyotrophic Lateral Sclerosis | ||||||||
| Intervention ICMJE |
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| Study Arms ICMJE |
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| Publications * | Not Provided | ||||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status ICMJE | Recruiting | ||||||||
| Estimated Enrollment ICMJE |
48 | ||||||||
| Original Estimated Enrollment ICMJE |
40 | ||||||||
| Estimated Study Completion Date ICMJE | June 14, 2021 | ||||||||
| Estimated Primary Completion Date | June 14, 2021 (Final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria ICMJE |
Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other protocol defined Inclusion/Exclusion criteria may apply. |
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| Sex/Gender ICMJE |
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| Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||||||||
| Accepts Healthy Volunteers ICMJE | No | ||||||||
| Contacts ICMJE |
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| Listed Location Countries ICMJE | United States | ||||||||
| Removed Location Countries | |||||||||
| Administrative Information | |||||||||
| NCT Number ICMJE | NCT03945279 | ||||||||
| Other Study ID Numbers ICMJE | 261AS101 | ||||||||
| Has Data Monitoring Committee | Yes | ||||||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement ICMJE |
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| Responsible Party | Biogen | ||||||||
| Study Sponsor ICMJE | Biogen | ||||||||
| Collaborators ICMJE | Not Provided | ||||||||
| Investigators ICMJE |
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| PRS Account | Biogen | ||||||||
| Verification Date | March 2021 | ||||||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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