Safety, PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy Volunteers

NCT05395624 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: OP-801-001
• Study Type: interventional
• Target: 65
• Locations: 1 country
• Sites: 3

Brief Summary

This is a Phase 1/2 study to evaluate the safety and tolerability of 18F-OP-801 in subjects with ALS, AD, MS, PD and age-matched HVs. 18F-OP-801 is intended as a biomarker for PET imaging of activated microglia and macrophages in regions of neuroinflammation.

Conditions

Amyotrophic Lateral Sclerosis (ALS); Parkinson Disease (PD); Alzheimer Disease (AD); Multiple Sclerosis (MS)

Outcome Measures

Primary Outcomes (1)

• The number of participants with treatment emergent adverse events (Safety and Tolerability)

  Safety of single dose of 18F-OP-801 as measured by treatment-related adverse events as assessed by CTCAE v5.0

  Safety and tolerability of 18F-OP-801 as assessed by the frequency, and severity of treatment-emergent adverse events (TEAEs) from Day 1 to Day 15 or Day 18-29

Secondary Outcomes (6)

• Measurement of biodistribution of 18F-OP-801 for each participant

  Through study completion at Day 15 or Day 18-29

• Measurement of clearance of 18F-OP-801 for each participant

  Through study completion at Day 15 or Day 18-29

• Assess ability of 18F-OP-801 to detect regions of neuroinflammation in ALS, AD, MS, and PD participants

  Through study completion at Day 15 or Day 18-29

• Assess test/retest imaging repeatability

  Through study completion at Day 18-29

• Correlation between plasma NfL levels and degree of 18F-OP-801 uptake in each participant

  Screening and Day 1

Other Outcomes (2)

• Correlation between plasma biomarker levels and PET signal for each participant

  Screening and Day 1

• Evaluate timeframe for optimal CNS tracer uptake

  Day 1

Study Arms (5)

Healthy Volunteer participants

EXPERIMENTAL

Intravenous Administration of 18F-OP-801 (18F Hydroxyl Dendrimer)

Drug: 18F-OP-801

Amyotrophic Lateral Sclerosis participants

EXPERIMENTAL

Intravenous Administration of 18F-OP-801 (18F Hydroxyl Dendrimer)

Drug: 18F-OP-801

Alzheimer's Disease participants

EXPERIMENTAL

Intravenous Administration of 18F-OP-801 (18F Hydroxyl Dendrimer)

Drug: 18F-OP-801

Multiple Sclerosis participants

EXPERIMENTAL

Intravenous Administration of 18F-OP-801 (18F Hydroxyl Dendrimer)

Drug: 18F-OP-801

Parkinson's Disease participants

EXPERIMENTAL

Intravenous Administration of 18F-OP-801 (18F Hydroxyl Dendrimer)

Drug: 18F-OP-801

Interventions

18F-OP-801 DRUG

18F Hydroxyl Dendrimer

Also known as: 18F Hydroxyl Dendrimer

Alzheimer's Disease participants; Amyotrophic Lateral Sclerosis participants; Healthy Volunteer participants; Multiple Sclerosis participants; Parkinson's Disease participants

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Has the ability to understand and sign the written ICF and local medical privacy authorization forms, which must be obtained prior to the conduct of any study related procedures.
• Female subjects of non-childbearing potential must be either surgically sterile (hysterectomy, bilateral tubal ligation, salpingectomy, and/or bilateral oophorectomy at least 26 weeks before the Screening Visit) or postmenopausal, defined as spontaneous amenorrhea for at least 2 years, with follicle-stimulating hormone (FSH) in the postmenopausal range at screening, based on the local laboratory's defined ranges.
• Female subjects of childbearing potential (i.e., ovulating, premenopausal, and not surgically sterile) and all male subjects must agree to practice abstinence from sexual intercourse or use a medically accepted contraceptive regimen (including hormonal contraceptives) during their participation in the study and for 90 days (males) or 6 months (females) after Day 1. Medically accepted contraceptive methods are defined as those with 90% or greater efficacy and are as follows:
• Male subjects: condoms or surgical sterilization of subject at least 26 weeks before the Screening Visit (i.e., vasectomy).
• Female subjects:
• Surgical sterilization at least 26 weeks before the Screening Visit (includes hysterectomy or bilateral tubal ligation, bilateral oophorectomy, or salpingectomy);
• Intrauterine device or diaphragm with spermicide for at least 12 weeks before the Screening Visit; or
• Hormonal contraception (oral, implant, injection, ring, or patch) for at least 12 weeks before the Screening Visit.
• If male, subjects must agree to abstain from sperm donation through 90 days after the Day 1 Visit.
• Female subjects may not be pregnant, lactating, or breastfeeding.
• Female subjects of childbearing potential must have negative result for pregnancy test at Screening and Check-in.
• Subjects must have an estimated glomerular filtration rate (eGFR) of \>45 mL/min/1.73m2 at Screening.
• C-reactive protein level ≤10 mg/dL.
• Subjects must be willing and able to abide by all study requirements and restrictions.
• Adult (Age 18 to 80, inclusive) at the Screening Visit

You may not qualify if:

• Subjects meeting any of the following criteria will be excluded from this study:
• Body weight \>120 kg
• Evidence of clinically significant or past medical history of hematologic, renal, endocrine, pulmonary, cardiac, gastrointestinal, hepatic, psychiatric, neurologic, immunologic, allergic disease (including multiple or clinically significant drug allergies) or any other condition that, in the opinion of the Investigator, might significantly interfere with the absorption, distribution, metabolism or excretion of study drug or place the subject at an unacceptable risk as a participant in this study
• History of recurrent kidney or liver malignancy
• Pacemaker or defibrillator or any non-removable metallic foreign objects in the body not compatible with MRI
• Inability to lie in a PET/CT or PET/MRI scanner for up to 90 minutes at a time
• Resolved acute illness considered clinically significant by the Investigator within 10 days prior to Screening
• History of alcoholism or drug abuse within 2 years prior to Screening. No cannabinoid drug use for at least 10 days prior to Day 1.
• Positive urine drug test, marijuana test or cotinine test at Screening or Check-In
• Any immunizations within the 28 days prior to screening
• Received any other investigational medicinal product within 30 days or 5 half-lives (whichever is longer) prior to Day 1
• Corticosteroid treatment (e.g., prednisone, solumedrol) within 30 days of Baseline
• Treatment with any of the following classes of nonsteroidal anti-inflammatory drugs (NSAIDS): carboxylic acids, enolic acids, cyclooxygenase (COX) II inhibitors within 14 days of Day 1
• Lost or donated \>450 mL of whole blood or blood products within 30 days prior to Screening
• Investigator has reason to believe that the subject may be unable to fulfill the protocol visit schedule or requirements

Sponsors & Collaborators

• Ashvattha Therapeutics, Inc.: lead

Study Sites (3)

UCSF

San Francisco, California, 94107, United States

RECRUITING

Stanford University

Stanford, California, 94305, United States

RECRUITING

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224, United States

RECRUITING

Related Publications (1)

• Jackson IM, Carlson ML, Beinat C, Malik N, Kalita M, Reyes S, Azevedo EC, Nagy SC, Alam IS, Sharma R, La Rosa SA, Moradi F, Cleland J, Shen B, James ML. Clinical Radiosynthesis and Translation of [18F]OP-801: A Novel Radiotracer for Imaging Reactive Microglia and Macrophages. ACS Chem Neurosci. 2023 Jul 5;14(13):2416-2424. doi: 10.1021/acschemneuro.3c00028. Epub 2023 Jun 13.

  PMID: 37310119 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Parkinson Disease; Alzheimer Disease; Multiple Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Movement Disorders; Synucleinopathies; Dementia; Tauopathies; Neurocognitive Disorders; Mental Disorders; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Farshad Moradi, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Julee Cheung

CONTACT

408-476-4172; jcheung@avttx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 13, 2022
• First Posted: May 27, 2022
• Study Start: February 2, 2023
• Primary Completion: January 1, 2026
• Study Completion: May 1, 2026
• Last Updated: May 2, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)