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Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB101 Administered to Adults With Multiple System Atrophy (HORIZON)

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ClinicalTrials.gov Identifier: NCT04165486
Recruitment Status : Active, not recruiting
First Posted : November 18, 2019
Last Update Posted : April 8, 2021
Sponsor:
Information provided by (Responsible Party):
Biogen

Tracking Information
First Submitted Date  ICMJE November 14, 2019
First Posted Date  ICMJE November 18, 2019
Last Update Posted Date April 8, 2021
Actual Study Start Date  ICMJE July 7, 2020
Estimated Primary Completion Date July 13, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 14, 2019)
  • Number of Participants with Adverse Events (AEs) [ Time Frame: Baseline up to Day 253 ]
    An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment.
  • Number of Participants with Serious Adverse Events (SAEs) [ Time Frame: Screening up to Day 253 ]
    An SAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or is a medically important event.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 14, 2019)
  • Area Under the Concentration-Time Curve From Time Zero to Time of Last Measurable Concentration [ Time Frame: Pre-dose and multiple timepoints up to 24 hours post-dose on Day 1; pre-dose and multiple timepoints up to 6 hours post-dose on 3 subsequent days; post-dose on 6 subsequent days over a 5 ½ month period ]
  • Maximum Observed Concentration (Cmax) [ Time Frame: Pre-dose and multiple timepoints up to 24 hours post-dose on Day 1; pre-dose and multiple timepoints up to 6 hours post-dose on 3 subsequent days; post-dose on 6 subsequent days over a 5 ½ month period ]
  • Time to Reach Maximum Observed Concentration (Tmax) [ Time Frame: Pre-dose and multiple timepoints up to 24 hours post-dose on Day 1; pre-dose and multiple timepoints up to 6 hours post-dose on Days 29, 57 and 85; post-dose on Days 8, 36, 64, 92, 113 and 169 ]
  • Serum Concentration of BIIB101 [ Time Frame: Pre-dose and multiple timepoints up to 24 hours post-dose on Day 1; pre-dose and multiple timepoints up to 6 hours post-dose on 3 subsequent days; post-dose on 6 subsequent days over a 5 ½ month period ]
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 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB101 Administered to Adults With Multiple System Atrophy
Official Title  ICMJE A Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB101 Administered Intrathecally to Adults With Multiple System Atrophy
Brief Summary

The primary objective is to evaluate the safety and tolerability of multiple doses of BIIB101 administered via intrathecal (IT) injection to participants with multiple system atrophy (MSA).

The secondary objective is to evaluate the pharmacokinetic (PK) profile of BIIB101.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Multiple System Atrophy
Intervention  ICMJE
  • Drug: BIIB101
    Administered as specified in the treatment arm.
  • Drug: Placebo
    Administered as specified in the treatment arm.
Study Arms  ICMJE
  • Experimental: BIIB101 Low Dose
    Participants will be administered BIIB101 low dose and matching placebo via intrathecal (IT) injection at regular intervals.
    Interventions:
    • Drug: BIIB101
    • Drug: Placebo
  • Experimental: BIIB101 Medium Dose
    Participants will be administered BIIB101 medium dose and matching placebo via IT injection at regular intervals.
    Interventions:
    • Drug: BIIB101
    • Drug: Placebo
  • Experimental: BIIB101 High Dose
    Participants will be administered BIIB101 high dose and matching placebo via IT injection at regular intervals.
    Interventions:
    • Drug: BIIB101
    • Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: November 14, 2019)
34
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 13, 2022
Estimated Primary Completion Date July 13, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Screening single-photon emission computed tomography (SPECT) with DaTscan™ (ioflupane I123 injection) results demonstrating loss (whether symmetric or asymmetric) of dopamine nerve terminals in the striatum consistent with neurodegenerative parkinsonism, as assessed with qualitative, visual read.
  • Diagnosed with probable or possible MSA, either parkinsonian-type (MSA-P) or cerebellar-type (MSA-C).
  • Must be able to walk unassisted for at least 10 meters (approximately 30 feet)

Key Exclusion Criteria:

  • Presence of cognitive dysfunction (defined as Montreal Cognitive Assessment (MoCA) score <25)
  • Family history of ataxia or parkinsonism and known genetic cause of ataxia or parkinsonism.

NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 40 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Austria,   France,   Germany
Removed Location Countries Portugal,   United Kingdom
 
Administrative Information
NCT Number  ICMJE NCT04165486
Other Study ID Numbers  ICMJE 262SP101
2019-001105-24 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
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/
Responsible Party Biogen
Study Sponsor  ICMJE Biogen
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Biogen
PRS Account Biogen
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP