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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 : Recruiting
First Posted : November 18, 2019
Last Update Posted : July 23, 2020
Sponsor:
Information provided by (Responsible Party):
Biogen

Study Description
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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.


Condition or disease Intervention/treatment Phase
Multiple System Atrophy Drug: BIIB101 Drug: Placebo Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 34 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB101 Administered Intrathecally to Adults With Multiple System Atrophy
Actual Study Start Date : July 7, 2020
Estimated Primary Completion Date : July 13, 2022
Estimated Study Completion Date : July 13, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: BIIB101 Low Dose
Participants will be administered BIIB101 low dose and matching placebo via intrathecal (IT) injection at regular intervals.
 Drug: BIIB101
Administered as specified in the treatment arm.

Drug: Placebo
Administered as specified in the treatment arm.
Experimental: BIIB101 Medium Dose
Participants will be administered BIIB101 medium dose and matching placebo via IT injection at regular intervals.
 Drug: BIIB101
Administered as specified in the treatment arm.

Drug: Placebo
Administered as specified in the treatment arm.
Experimental: BIIB101 High Dose
Participants will be administered BIIB101 high dose and matching placebo via IT injection at regular intervals.
 Drug: BIIB101
Administered as specified in the treatment arm.

Drug: Placebo
Administered as specified in the treatment arm.


Outcome Measures
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Primary Outcome Measures :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. 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 ]
  2. 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 ]
  3. 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 ]
  4. 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 ]

Eligibility Criteria
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Ages Eligible for Study:   40 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Contacts and Locations
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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): NCT04165486


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

Locations
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Austria
Medizinische Universität Innsbruck Recruiting
Innsbruck, Austria, 6020
Contact: Klaus Seppi    +4351250425810    lki.ne.parkinson@tirol-kliniken.at   
Principal Investigator: Klaus Seppi         
France
Hopital Purpan Recruiting
Toulouse Cedex 09, France, 31059
Contact: Olivier Rascol    +33561779103    cic1436@inserm.fr   
Contact: Stephanie Bras    +33561772474    stephanie.bras@inserm.fr   
Principal Investigator: Olivier Rascol         
Sponsors and Collaborators
Biogen
Investigators
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Study Director: Medical Director Biogen
More Information
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Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT04165486    
Other Study ID Numbers: 262SP101
2019-001105-24 ( EudraCT Number )
First Posted: November 18, 2019    Key Record Dates
Last Update Posted: July 23, 2020
Last Verified: July 2020
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: http://www.biogenclinicaldatarequest.com/

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Multiple System Atrophy
Shy-Drager Syndrome
Atrophy
Pathological Conditions, Anatomical
Primary Dysautonomias
Autonomic Nervous System Diseases
Nervous System Diseases
Basal Ganglia Diseases
 Brain Diseases
Central Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases
Hypotension
Vascular Diseases
Cardiovascular Diseases