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Amyotrophic Lateral Sclerosis (ALS)Phase 1/2NCT05395624

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

A Phase 1/2 study testing 18F-OP-801 in people with Amyotrophic Lateral Sclerosis (ALS) and Parkinson Disease (PD), run by Ashvattha Therapeutics, Inc. at 3 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 to 80 yearsHealthy volunteers can take part
What is being tested?
18F-OP-801
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
65 participants
Where
3 sitesUnited States
Study dates
2 Feb 2023to 1 May 2026
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

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 80 years
  • All sexes
  • Healthy volunteers accepted
  • Adult, Older adult

Who may be able to join

  • 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);
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  • 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
  • Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by the modified El Escorial criteria.
  • Forced vital capacity (FVC) of ≥50%; or if in the opinion of the investigator can lay flat for up to 90 minutes. If FVC has been performed within the past 6 months, this data may be used at the discretion of the investigator.
  • For ALS subjects, medication changes within 30 days prior to the Screening Visit should be discussed with the Medical Monitor.
  • Adult (Age 40 to 80, inclusive) at the Screening Visit
  • Clinical diagnosis of early stage dementia, Alzheimer type, plus positive Aβ and tau PET imaging, cerebrospinal fluid (CSF) and/or plasma biomarkers consistent with 2018 NIA-AA criteria
  • MMSE score >20 at Screening
  • AD medication changes within 30 days prior to the Screening Visit should be discussed with the Medical Monitor.
  • Adult (Age 18 to 70, inclusive) at the Screening Visit
  • MS medication changes within 30 days prior to the Screening Visit should be discussed with the Medical Monitor.
  • Diagnosis of RRMS based on 2017 McDonald criteria
  • If not newly diagnosed, subjects should have at least 1 documented relapse in the last 24 months.
  • "Active disease" subjects should have at least 1 Gadolinium-enhancing (Gd+) T1-weighted brain or spinal cord lesion at Screening MRI.
  • "Disease in remission" subjects should have no Gd+ T1-weighted brain or spinal cord lesions at Screening MRI and stable clinical symptoms for at least 3 months prior to Day 1.
  • EDSS score between 2.0 and 5.5 inclusive at Screening
  • Inclusion criteria specific to subjects with progressive MS:
  • Diagnosis of PPMS or SPMS based on 2017 McDonald criteria
  • EDSS score between 3.0 and 6.5 inclusive at Screening
  • No evidence of relapse in the prior 6 months
  • Neurological exam and symptom stability for ≥30 days prior to Day 1
  • Documented evidence of disability progression in the past 24 months not temporally related to a relapse
  • Adult (Age 55 to 80, inclusive) at the Screening Visit
  • Diagnosis of definite, idiopathic Parkinson's disease according to UK Parkinson's Society Brain Bank diagnostic criteria
  • PD medication changes within 30 days prior to the Screening Visit should be discussed with the Medical Monitor.
  • Overall Exclusion Criteria - For All Subjects:
  • 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
  • Laboratory results (serum chemistry, hematology, coagulation and urinalysis) outside the normal range at Screening and Check-In and considered clinically significant in the opinion of the Investigator. Any elevation of aspartate transaminase (AST) and alanine transaminase (ALT) more than 3 times above the upper limit of normal at screening and/or check-in is exclusionary. One retest of an exclusionary laboratory result is allowed at the discretion of the Investigator and with approval from the Medical Monitor.
  • 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
  • MRI exclusion criteria: findings that may interfere with interpretation of the PET imaging, including but not limited to significant cortical/subcortical cerebrovascular disease, infectious disease, space-occupying lesions, hydrocephalus or other abnormalities associated with CNS disease not related to ALS, AD, MS or PD
  • CT exclusion criteria include any medical device or metallic implant that may interfere with image acquisition or affect image reconstruction (e.g., CT attenuation correction).
  • Investigator has reason to believe that the subject may be unable to fulfill the protocol visit schedule or requirements
  • Has any finding that, in the view of the Investigator and Medical Monitor, would compromise the subject's safety in the trial

Who cannot join

  • Clinical signs or laboratory findings suggestive of neuromyelitis optica (NMO) spectrum disorders or myelin oligodendrocyte glycoprotein (MOG)-associated encephalomyelitis (i.e., presence of anti-NMO [aquaporin-4] antibodies or anti-MOG antibodies)
  • Diagnosis of progressive multifocal leukoencephalopathy (PML)
  • Secondary, atypical, or genetic parkinsonism
  • Clinically relevant finding on physical examination at Screening
  • Family history of neurological disease that may confound interpretation of imaging results
  • History of any central nervous system disorder or brain trauma that could cause imaging abnormalities in the opinion of the Principal Investigator and Medical Monitor

Trial locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Julee Cheung contact

408-476-4172

jcheung@avttx.com

United States3 sites, 3 recruiting

  • Mayo Clinic Jacksonville

    Jacksonville, Florida, 32224

    Colette McHugh-Strong, JD, 904-953-4965, McHugh-Strong.Colette@mayo.edu

    Recruiting

  • UCSF

    San Francisco, California, 94107

    Hannah George, 415-501-0671, neuromuscular.research@ucsf.edu

    Recruiting

  • Stanford University

    Stanford, California, 94305

    Sierrah Hoover, 650-723-3223, shoover7@stanford.edu

    Recruiting

Other trials in United States

About this study

Official title: A Phase 1/2 Study to Evaluate Safety, PK and Biodistribution of an Imaging Agent, 18F-OP-801, After Intravenous Administration to Patients With ALS, Alzheimer's Disease, Multiple Sclerosis, Parkinson's Disease and Healthy Volunteers

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.

Read the detailed description

Microglia and macrophages have emerged as key players in neurodegenerative and neuroinflammatory disorders of the central nervous system (CNS) such as amyotrophic lateral sclerosis, Alzheimer's disease (AD), multiple sclerosis (MS) and Parkinson's disease (PD). Treatments that selectively target these cells will need to cross the blood-brain barrier (BBB) at levels high enough to produce therapeutic effects. Unfortunately, it is difficult to directly measure the amount of a therapeutic that actually reaches the CNS target tissue. Development of biomarkers that allow direct visualization of cellular targeting across the BBB could offer profound insight into drug actions on innate immune cells in the brain. Furthermore, the ability to track accumulation of activated microglia in the brain could allow early identification of patients at risk for neurodegenerative or neuroinflammatory disease, precise stratification of patients for clinical trials and an efficacy measure for therapies that target neuroinflammation.

Positron emission tomography (PET) is a noninvasive imaging technology that can provide quantitative biological information in vivo, and it plays an important role in disease diagnosis, therapy assessment, and drug development. PET allows evaluation of the biological process without pharmacological effects because the amount of radiotracer used in imaging studies is very low. Several PET diagnostics track neuroinflammation in the brain, but current methods are limited by high background signal in healthy tissues.

18F-OP-801 is selectively taken up only by activated but not resting microglia, offering the potential to detect neuroinflammation at lower levels and earlier stages of disease than any current clinical PET radiotracer. We propose to use 18F-OP-801 to image activated microglia and brain macrophages in subjects with ALS, AD, MS, and PD to assess the compound's utility as a biomarker of neuroinflammation

Being tested
18F-OP-801
Medical subject headings
Amyotrophic Lateral Sclerosis, Parkinson Disease, Alzheimer Disease, Multiple Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 18F-OP-801Drug

    Also called 18F Hydroxyl Dendrimer

    18F Hydroxyl Dendrimer

Study groups in NCT05395624
Study groupRoleWhat they receive
Healthy Volunteer participantsExperimentalIntravenous Administration of 18F-OP-801 (18F Hydroxyl Dendrimer)
Receives: Drug: 18F-OP-801
Amyotrophic Lateral Sclerosis participantsExperimentalIntravenous Administration of 18F-OP-801 (18F Hydroxyl Dendrimer)
Receives: Drug: 18F-OP-801
Alzheimer's Disease participantsExperimentalIntravenous Administration of 18F-OP-801 (18F Hydroxyl Dendrimer)
Receives: Drug: 18F-OP-801
Multiple Sclerosis participantsExperimentalIntravenous Administration of 18F-OP-801 (18F Hydroxyl Dendrimer)
Receives: Drug: 18F-OP-801
Parkinson's Disease participantsExperimentalIntravenous Administration of 18F-OP-801 (18F Hydroxyl Dendrimer)
Receives: Drug: 18F-OP-801

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: 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)

  1. Measurement of biodistribution of 18F-OP-801 for each participant

    Measure biodistribution of 18F-OP-801 using whole body PET/MRI or PET/computed tomography (CT) scans

    Measured: Through study completion at Day 15 or Day 18-29

  2. Measurement of clearance of 18F-OP-801 for each participant

    Measure clearance of 18F-OP-801 using whole body PET/MRI or PET/computed tomography (CT) scans

    Measured: Through study completion at Day 15 or Day 18-29

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

    Determine correlation between MRI and PET images in each subject, quantifying the extent of 18F-OP-801 uptake in the region of neuroinflammation relative to normal brain section in the same subject as measured by whole body PET/MRI or PET/CT scans

    Measured: Through study completion at Day 15 or Day 18-29

  4. Assess test/retest imaging repeatability

    Compare regional and global brain uptake of 18F-OP-801 on two separate occasions

    Measured: Through study completion at Day 18-29

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

    Correlation of plasma NfL levels with brain PET signal

    Measured: Screening and Day 1

  6. Correlation between relevant clinical scales and degree of 18F-OP-801 uptake in each participant

    Correlation of ALSFRS-R/PUMNS, MMSE, EDSS or SE-ADL scores with brain PET signal

    Measured: Screening and Day 1

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 May 2022
  2. Study start2 Feb 2023
  3. Primary completion1 Jan 2026 (estimated)
  4. Study completion1 May 2026 (estimated)

Resources

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 1/2
Primary purpose
Diagnosis
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
65 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05395624
Sponsor's protocol ID
OP-801-001
Status last verified
Apr 2025
First submitted
13 May 2022
Last updated
2 May 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 37310119

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing 18F-OP-801 in people with Amyotrophic Lateral Sclerosis (ALS) and Parkinson Disease (PD), run by Ashvattha Therapeutics, Inc. at 3 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 58 inclusion and 6 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: looking for participants now, as of the sponsor's last update on 2 May 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Julee Cheung (408-476-4172, jcheung@avttx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The number of participants with treatment emergent adverse events (Safety and Tolerability) (time frame: 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). 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 NCT05395624, 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 2 May 2025.

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