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

NCT ID: NCT03764488

Last Updated: 2023-06-22

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-12-20
• Study Completion Date: 2021-07-10

Brief 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). 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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: BASIC_SCIENCE
• Blinding Strategy: NONE

Study Groups

BIIB067 High Dose + 99mTc-MAG3-BIIB067 in 15 mL aCSF

Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in 15 milliliter (mL) artificial cerebrospinal fluid (aCSF).

Group Type: EXPERIMENTAL

Tofersen

Intervention Type: DRUG

Administered as specified in the treatment arm.

99mTc-MAG3-BIIB067

Intervention Type: DRUG

Administered as specified in the treatment arm.

BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in 15 mL aCSF

Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in 15 mL aCSF.

Group Type: EXPERIMENTAL

Tofersen

Intervention Type: DRUG

Administered as specified in the treatment arm.

99mTc-MAG3-BIIB067

Intervention Type: DRUG

Administered as specified in the treatment arm.

BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in 5 mL aCSF

Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in 5 mL aCSF.

Group Type: EXPERIMENTAL

Tofersen

Intervention Type: DRUG

Administered as specified in the treatment arm.

99mTc-MAG3-BIIB067

Intervention Type: DRUG

Administered as specified in the treatment arm.

BIIB067 Low Dose + 99mTc-MAG3-BIIB067 in up to 20 mL aCSF

Participants will receive intrathecal injection consisting of unlabeled BIIB067 and 99mTc-MAG3-BIIB067 in up to 20 mL aCSF.

Group Type: EXPERIMENTAL

Tofersen

Intervention Type: DRUG

Administered as specified in the treatment arm.

99mTc-MAG3-BIIB067

Intervention Type: DRUG

Administered as specified in the treatment arm.

Interventions

Tofersen

Administered as specified in the treatment arm.

Intervention Type: DRUG

99mTc-MAG3-BIIB067

Administered as specified in the treatment arm.

Intervention Type: DRUG

Other Intervention Names

BIIB067; QALSODY

Eligibility Criteria

Inclusion Criteria

• 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).

Exclusion Criteria

• 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Biogen

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Biogen

Locations

Research Site

Boston, Massachusetts, United States

Research Site

New York, New York, United States

Countries

United States

Other Identifiers

233HV101

Identifier Type: -

Identifier Source: org_study_id