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Completed

NCT Number: NCT06454721

A Study to Determine the Biodistribution, Safety, and Tolerability of a Microdose of Radiolabeled Diranersen (BIIB080) Co-administered With Diranersen in Healthy Adults

In this study, researchers will learn more about a study drug called diranersen (BIIB080). Diranersen is currently a drug under investigation for treatment of Alzheimer's disease. The main question researchers are trying to answer in this study is how radiolabeled diranersen distributes in the brain and spinal cord. To help answer this question, researchers will use positron emission tomography (PET) scanner that can detect radiolabeled diranersen after a single injection of a small dose of radiolabeled diranersen ([89Zr]Zr-DFO-diranersen) and a dose of diranersen together via an intrathecal (IT) injection in healthy volunteers. Researchers will also learn about the safety of injecting radiolabeled diranersen and diranersen together.

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Key information

Conditions

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

UC Davis

Sacramento, California, 95817, United States

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Must be healthy adult and have a body mass index (BMI) 18 through 30 kilograms per square meter (kg/m^2).
  • Males must practice highly effective contraception and females must be of no childbearing potential.

Key Exclusion Criteria:

  • Conditions that influence cerebrospinal fluid (CSF) kinetics e.g., significant scoliosis or a physical disability that limits mobility.
  • Contraindication to:
  • Magnetic resonance imaging (MRI) scan: e.g., indwelling ferrous metal, implanted medical devices, or claustrophobia.
  • Radiation/PET scan: e.g., inability to lie flat or still for the duration of the scan) or intolerance to previous nuclear medicine scans.
  • Lumbar Puncture (LP, spinal tap): e.g., current use of a medication that prolongs coagulation times, including aspirin, known disorders of the coagulation cascade, platelet function, or platelet count, low white blood cell counts, chronic low back pain; prior lumbar spine surgery, anatomical factors at or near the spinal tap site, clinical signs of raised intracranial pressure (e.g., headache, or focal neurologic signs).
  • Findings from the screening MRI that may interfere with participant safety or the scientific integrity of the study e.g., Chiari malformation, high grade spinal stenosis, hydrocephalus, or neural tube defect.
  • Abnormal Laboratory Values e.g., liver functions, anemia, abnormal blood coagulation profile.
  • Cardiac, endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, or malignant disease.
  • Severe allergic or anaphylactic reactions/ systemic hypersensitivity to diranersen or any component of the study treatment.
  • Alcohol or substance abuse.
  • Plans to undergo elective procedures or surgeries during the study.
  • Enrollment in any other drug, biologic, device, or clinical study within 30 days or 5 half-lives, whichever is longer, prior to Check-in.
  • Use of any prescription medication (except hormone therapy), over-the-counter medication (excluding acetaminophen), or dietary and herbal supplements (e.g., St. John's wort) within 28 days of the of dosing and during the study.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

[89Zr]Zr-DFO-Diranersen

Drug

Administered as specified in the treatment arm.

Diranersen

Drug

Administered as specified in the treatment arm.

Other names: ISIS 814907, BIIB080

Primary outcomes

  1. CNS Biodistribution of [89Zr]Zr-DFO-Diranersen Assessed as the Percentage of Dose Taken up by Each Region of Interest (ROI)

    Time frame: Post-dose on Days 1, 2, 5, and 8

    CNS biodistribution of [89Zr]Zr-DFO-Diranersen will be measured using the Positron Emission tomography (PET) imaging. The mean radioactive counts per voxel will be measured. These counts will be converted to the percentage of dose that is taken up by each ROI (% of injected tracer dose per gram of tissue) to quantify the biodistribution in measurable tissues and CSF spaces at relative distances from the lumbar injection using quantitative scaling algorithms.

Secondary outcomes

  1. Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Up to 197 days

    An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. 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, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect and is a medically important event.

  2. Dosimetry: Dose of Radiation Absorbed as Measured by PET Imaging Test

    Time frame: Post-dose on Days 1, 2, 5, and 8

    Absorbed dose for the various organs will be estimated from the PET scans.

Sponsors and collaborators

Lead sponsor

Biogen

Industry

Registry information

Official study title

A Phase 1, Open-Label Study to Determine the Biodistribution, Safety, and Tolerability of a Microdose of Radiolabeled BIIB080 Co-administered With BIIB080 in Healthy Adults

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Jun 12, 2024
Registry last updated
Jul 10, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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