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 : January 29, 2021
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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 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 |

Arm | Intervention/treatment |
---|---|
Experimental: BIIB101 Low Dose
Participants will be administered BIIB101 low dose and matching placebo via intrathecal (IT) injection at regular intervals.
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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. |
- 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.
- 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 ]

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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 |
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.

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
Contact: US Biogen Clinical Trial Center | 866-633-4636 | clinicaltrials@biogen.com | |
Contact: Global Biogen Clinical Trial Center | clinicaltrials@biogen.com |
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, Haute Garonne, France, 31059 | |
Contact: Olivier Rascol +33561779103 cic1436@inserm.fr | |
Contact: Stephanie Bras +33561772474 stephanie.bras@inserm.fr | |
Principal Investigator: Olivier Rascol | |
Hopital Roger Salengro - CHU Lille | Recruiting |
Lille Cedex, Nord, France, 59037 | |
Contact: Caroline Moerau +33320446752 caroline.moreau@chru-lille.fr | |
Principal Investigator: Caroline Moerau | |
Groupe Hospitalier Pitie-Salpetriere | Recruiting |
Paris, France, 75013 | |
Contact: Jean-Christophe Corvol | |
Germany | |
Universitaetsklinikum Ulm | Recruiting |
Ulm, Baden Wuerttemberg, Germany, 89081 | |
Contact: Albert Ludolf Albert.Ludolph@rku.de | |
Principal Investigator: Albert Ludolf | |
Universitaetsklinikum Giessen und Marburg GmbH Standort Marburg | Recruiting |
Marburg, Hessen, Germany, 35043 | |
Medizinische Hochschule Hannover | Recruiting |
Hannover, Niedersachsen, Germany, 30625 | |
Principal Investigator: Guenter Hoeglinger | |
Universitaetsklinikum Duesseldorf AoeR | Recruiting |
Duesseldorf, Nordrhein Westfalen, Germany, 40225 |
Study Director: | Medical Director | Biogen |
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: | January 29, 2021 |
Last Verified: | January 2021 |
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/ |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
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 |