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The primary objective of the study is to evaluate the distribution in the central nervous system (CNS) of a microdose 99mTc-MAG3-BIIB067 co-administered with unlabeled BIIB067 (Tofersen). The secondary objective of the study is to assess the safety and tolerability of unlabeled BIIB067 co-administered with a microdose of 99mTc-MAG3-BIIB067 to healthy participants.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BIIB067 High Dose + 99mTc-MAG3-BIIB067 in 15 mL aCSF | Experimental | Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in 15 milliliter (mL) artificial cerebrospinal fluid (aCSF). |
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| BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in 15 mL aCSF | Experimental | Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in 15 mL aCSF. |
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| BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in 5 mL aCSF | Experimental | Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in 5 mL aCSF. |
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| BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in up to 20 mL aCSF | Experimental | Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in up to 20 mL aCSF. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tofersen | Drug | Administered as specified in the treatment arm. |
|
| Measure | Description | Time Frame |
|---|---|---|
| BIIB067 Concentrations Throughout the CNS | BIIB067 concentrations throughout the CNS will be estimated by single-photon emission computed tomography (SPECT)/computed tomography (CT) imaging of 99mTc-MAG3-BIIB067. | 1 to 24 hours post-dose on Day 1 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | 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. An SAE is any untoward medical occurrence that at any dose: results in death, in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event), however, this does not include an event that, had it occurred in a more severe form, might have caused 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. |
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Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Boston | Massachusetts | 02114 | United States | ||
| Research Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41233143 | Derived | Cook BE, McLaren DG, Sullivan JM, El Fakhri G, Yokell DL, Freeman MW, Currier N, Oestergaard ME, Dobson H, Hesterman J, Salem N, Nestorov I, Monine M, Martarello L, Evans KC, Fradette S, Ferguson TA, Graham D, Passamonti L. Central Nervous System Biodistribution and Pharmacokinetics of Radiolabeled Tofersen in Rodents, Nonhuman Primates, and Humans. J Nucl Med. 2026 Jan 2;67(1):139-144. doi: 10.2967/jnumed.125.270731. |
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|
| 99mTc-MAG3-BIIB067 | Drug | Administered as specified in the treatment arm. |
|
| Up to Day 91 |
| Dose of Radiation Absorbed (Dosimetry) in Specific Regions of the CNS | Dose of radiation absorbed (dosimetry) in specific regions of the CNS will be estimated by SPECT/CT imaging of 99mTc-MAG3-BIIB067. | 1 to 24 hours post-dose on Day 1 |
| New York |
| New York |
| 10065 |
| United States |
| ID | Term |
|---|---|
| C000709090 | tofersen |
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