Healthy VolunteersPhase 1NCT03764488
A Study to Evaluate Safety, Tolerability, and Distribution of a Microdose of Radiolabeled BIIB067 (99mTc-MAG3-BIIB067) Co-administered With BIIB067 (Tofersen) to Healthy Adults
A Phase 1 study testing Tofersen and 99mTc-MAG3-BIIB067 in people with Healthy Volunteers, run by Biogen at 2 sites in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy Volunteers
- What is being tested?
- Tofersen and 99mTc-MAG3-BIIB067
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 2 sitesUnited States
- Study dates
- 20 Dec 2018to 10 Jul 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Must have a body mass index between 18 and 30 kilogram per square meter (kg/m^2), inclusive.
- All men must practice highly effective contraception during the study and for 5 months after their last dose of study treatment.
- All female participants must be of non-childbearing potential and must meet 1 of the following criteria to participate in the study: a. Postmenopausal, b. History of bilateral oophorectomy (performed at least 6 weeks prior to Screening), c. History of hysterectomy (performed at least 6 weeks prior to Screening), or d. History of female surgical sterilization (e.g., bilateral tubal ligation; performed at least 6 weeks prior to Screening).
Who cannot join
- History of or ongoing clinically significant cardiac, endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, or other major disease, as determined by the Investigator.
- Clinically significant abnormal laboratory test values, as determined by the Investigator, at Screening or Check-in.
- Use of any prescription medication, over-the-counter medication (e.g., aspirin and nonsteroidal anti-inflammatory drugs [excluding acetaminophen]), or dietary and herbal supplements (e.g., St. John's wort) within 28 days of Check-in and an unwillingness to refrain from use through end of study (i.e., through the Day 91 visit), unless specifically permitted elsewhere within the protocol.
- Current enrollment in any other drug, biologic, device, or clinical study, or treatment with an investigational drug or approved therapy for investigational use within 30 days (6 months for biologics) or 5 half-lives, whichever is longer, prior to Check-in.
- History or physical examination that could be suggestive of a medical or mechanical condition, disorder, or disease that could represent a relative contraindication to lumbar puncture (LP), including but not limited to: (a) medication use that prolongs coagulation times, including aspirin; (b) known disorders of the coagulation cascade, platelet function, or platelet count; (c) low white blood cell counts; (d) chronic low back pain; (e) prior lumbar spine surgery; (f) anatomical factors at or near the LP site; (g) clinical signs of raised intracranial pressure (e.g., headache, papilledema, or focal neurologic signs).
- Contraindications to radiation exposure for research purposes.
- Exposure to ionizing radiation within the last 12 months that would result in approaching the exposure limits for healthy volunteers.
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Research Site
Boston, Massachusetts, 02114
Research Site
New York, New York, 10065
About this study
Official title: A Phase 1, Safety, Tolerability, and Distribution Study of a Microdose of Radiolabeled BIIB067 Co-administered With BIIB067 to Healthy Adults
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.
- Conditions
- Healthy Volunteers
- Being tested
- Tofersen, 99mTc-MAG3-BIIB067
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TofersenDrug
Also called BIIB067, QALSODY
Administered as specified in the treatment arm.
99mTc-MAG3-BIIB067Drug
Administered as specified in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Drug: Tofersen; Drug: 99mTc-MAG3-BIIB067 |
| 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. Receives: Drug: Tofersen; Drug: 99mTc-MAG3-BIIB067 |
| 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. Receives: Drug: Tofersen; Drug: 99mTc-MAG3-BIIB067 |
| 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. Receives: Drug: Tofersen; Drug: 99mTc-MAG3-BIIB067 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 1 to 24 hours post-dose on Day 1
Secondary outcomes (2)
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.
Measured: 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.
Measured: 1 to 24 hours post-dose on Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2018
- Study start20 Dec 2018
- Primary completion10 Jun 2021
- Study completion10 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Healthy VolunteersStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03764488
- Sponsor's protocol ID
- 233HV101
- Status last verified
- Jun 2023
- First submitted
- 3 Dec 2018
- Last updated
- 22 Jun 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 41233143
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Safety, Tolerability, and Distribution Study of a Microdose of Radiolabeled BIIB067 Co-administered With BIIB067 to Healthy Adults
- ClinicalTrials.gov ID
- NCT03764488
- Acronym / sponsor ID
- 233HV101
- Conditions
- Healthy Volunteers
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 20 Dec 2018 (actual)
- Primary completion
- 10 Jun 2021 (actual)
- Study completion
- 10 Jul 2021 (actual)
- First posted
- 5 Dec 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| BIIB067 High Dose + 99mTc-MAG3-BIIB067 in 15 mL aCSF | Experimental | Drug: Tofersen, Drug: 99mTc-MAG3-BIIB067Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in 15 milliliter (mL) artificial cerebrospinal fluid (aCSF). |
| BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in 15 mL aCSF | Experimental | Drug: Tofersen, Drug: 99mTc-MAG3-BIIB067Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in 15 mL aCSF. |
| BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in 5 mL aCSF | Experimental | Drug: Tofersen, Drug: 99mTc-MAG3-BIIB067Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in 5 mL aCSF. |
| BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in up to 20 mL aCSF | Experimental | Drug: Tofersen, Drug: 99mTc-MAG3-BIIB067Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in up to 20 mL aCSF. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Must have a body mass index between 18 and 30 kilogram per square meter (kg/m^2), inclusive.
- All men must practice highly effective contraception during the study and for 5 months after their last dose of study treatment.
- All female participants must be of non-childbearing potential and must meet 1 of the following criteria to participate in the study: a. Postmenopausal, b. History of bilateral oophorectomy (performed at least 6 weeks prior to Screening), c. History of hysterectomy (performed at least 6 weeks prior to Screening), or d. History of female surgical sterilization (e.g., bilateral tubal ligation; performed at least 6 weeks prior to Screening).
Key exclusion criteria (8 listed)
- History of or ongoing clinically significant cardiac, endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, or other major disease, as determined by the Investigator.
- Clinically significant abnormal laboratory test values, as determined by the Investigator, at Screening or Check-in.
- Use of any prescription medication, over-the-counter medication (e.g., aspirin and nonsteroidal anti-inflammatory drugs [excluding acetaminophen]), or dietary and herbal supplements (e.g., St. John's wort) within 28 days of Check-in and an unwillingness to refrain from use through end of study (i.e., through the Day 91 visit), unless specifically permitted elsewhere within the protocol.
- Current enrollment in any other drug, biologic, device, or clinical study, or treatment with an investigational drug or approved therapy for investigational use within 30 days (6 months for biologics) or 5 half-lives, whichever is longer, prior to Check-in.
- History or physical examination that could be suggestive of a medical or mechanical condition, disorder, or disease that could represent a relative contraindication to lumbar puncture (LP), including but not limited to: (a) medication use that prolongs coagulation times, including aspirin; (b) known disorders of the coagulation cascade, platelet function, or platelet count; (c) low white blood cell counts; (d) chronic low back pain; (e) prior lumbar spine surgery; (f) anatomical factors at or near the LP site; (g) clinical signs of raised intracranial pressure (e.g., headache, papilledema, or focal neurologic signs).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TofersenAlso: BIIB067, QALSODYAdministered as specified in the treatment arm. | Drug | BIIB067 High Dose + 99mTc-MAG3-BIIB067 in 15 mL aCSF, BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in 15 mL aCSF, BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in 5 mL aCSF, BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in up to 20 mL aCSF |
| 99mTc-MAG3-BIIB067Administered as specified in the treatment arm. | Drug | BIIB067 High Dose + 99mTc-MAG3-BIIB067 in 15 mL aCSF, BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in 15 mL aCSF, BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in 5 mL aCSF, BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in up to 20 mL aCSF |
Primary outcome
BIIB067 Concentrations Throughout the CNS
Time frame: 1 to 24 hours post-dose on Day 1
BIIB067 concentrations throughout the CNS will be estimated by single-photon emission computed tomography (SPECT)/computed tomography (CT) imaging of 99mTc-MAG3-BIIB067.
2 secondary outcomes
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to Day 91
Dose of Radiation Absorbed (Dosimetry) in Specific Regions of the CNS
Time frame: 1 to 24 hours post-dose on Day 1
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2023.
- Derived from this study: PubMed 41233143Cook 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Tofersen and 99mTc-MAG3-BIIB067 in people with Healthy Volunteers, run by Biogen at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
BIIB067 Concentrations Throughout the CNS (time frame: 1 to 24 hours post-dose on Day 1). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03764488, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2023.