A Study to Evaluate the Safety, Pharmacokinetics and Biodistribution of an Imaging Agent, 18F-OP-801 (18F Hydroxyl Dendrimer) in Patients With Amyotrophic Lateral Sclerosis (ALS) and Healthy Volunteers (HV)


About this study

The purpose of this study is to evaluate the safety, pharmacokinetics and biodistribution of an imaging agent, 18F-OP-801 (18F Hydroxyl Dendrimer), after intravenous administration to patients with Amyotrophic Lateral Sclerosis (ALS) and Healthy Volunteers (HV)

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

1. Adult (Age 18-80, inclusive) at the Screening Visit.

2. 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

3. 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

4. Female subjects of childbearing potential (i.e., ovulating, premenopausal, and not
surgically sterile) and all male subjects must 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.

5. Acceptable methods of contraception for male subjects enrolled in the study include
the following:

1. Condoms or surgical sterilization of subject at least 26 weeks before the
Screening Visit (i.e., vasectomy).

Acceptable methods of contraception for female subjects enrolled in the study
include the following:

2. Surgical sterilization at least 26 weeks before the Screening Visit (includes
hysterectomy or bilateral tubal ligation, bilateral oophorectomy, or

3. Intrauterine device or diaphragm with spermicide for at least 12 weeks before the
Screening Visit; or

4. Hormonal contraception (oral, implant, injection, ring, or patch) for at least 12
weeks before the Screening Visit.

6. If male, subjects must agree to abstain from sperm donation through 90 days after the
Day 1 Visit.

7. Female subjects may not be pregnant, lactating, or breastfeeding.

8. Female subjects of childbearing potential must have negative result for pregnancy test
at Screening and Check-in.

9. Subjects must have an estimated glomerular filtration rate (eGFR) of > 45 mL/min/1.73m^2
at Screening.

10. C-reactive protein level ≤ 10 mg/dL.

11. Subjects must be willing and able to abide by all study requirements and restrictions.

Inclusion Specific to ALS Subjects:

12. Serum NfL concentration at Screening Visit above the 95th percentile of the reference
range for healthy subjects of the same age (using reference data).

13. Sporadic or familial ALS diagnosed as possible, laboratory-supported probable,
probable, or definite as defined by the modified El Escorial criteria.

14. ALS cognitive behavioral screen score > 10 on the cognitive scale and/or > 32 on the
behavioral scale.

15. 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.

16. Medications for ALS subjects should be stable for 30 days prior to the Screening Visit
and remain unchanged during the subject's participation in the study.

Exclusion Criteria:

1. Body weight > 120 kg.

2. 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

3. History of recurrent kidney or liver malignancy.

4. Pacemaker or defibrillator or any non-removable metallic foreign objects in the body
not compatible with MRI.

5. Inability to lie in a PET/CT or PET/MRI scanner for up to 90 minutes.

6. 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 the above 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, with approval from the
Medical Monitor.

7. Resolved acute illness considered clinically significant by the Investigator within 10
days prior to screening.

8. 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.

9. Positive urine drug test, marijuana test, or cotinine test at Screening or Check-In.

10. Any immunizations within the 28 days prior to screening, with the exception of a
COVID-19 vaccine or booster.

11. Received any other investigational medicinal product within 30 days or 5 half-lives of
the investigational medicinal product (whichever is longer) prior to Day 1.

12. Treatment with immunosuppressive agents (e.g., prednisone, solumedrol) within 30 days
of Day 1. Treatment with anti-inflammatory agents including any medications in the
following classes of nonsteroidal anti-inflammatory drugs (NSAIDS): carboxylic acids,
enolic acids, cyclooxygenase (COX) II inhibitors within 14 days of Day 1.

13. Lost or donated > 450 mL of whole blood or blood products within 30 days prior to

14. MRI exclusion criteria include: 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.

15. CT exclusion criteria include any medical device or metallic implant that may
interfere with image acquisition or affect image reconstruction (e.g., CT attenuation

16. Investigator has reason to believe that the subject may be unable to fulfill the
protocol visit schedule or requirements.

17. Has any finding that, in the view of the Investigator and Medical Monitor, would
compromise the subject's safety requirements.

18. Is employed by the Sponsor, the CRO, or the study site (permanent, temporary contract
worker, or designee responsible for the direct conduct of the study), or is a family
member (spouse, parent, sibling, or child) of the Sponsor, CRO, or study site

Exclusion Criteria Specific to HV Subjects:

19. Clinically relevant finding on physical examination at screening.

20. Family history of ALS or frontotemporal dementia.

21. History of any central nervous system disorder or brain trauma (concussions, etc.).

Medical monitor must be consulted for potential inclusion of subject with history
of CNS involvement.

Eligibility last updated 7/20/22. Questions regarding updates should be directed to the study team contact.


Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

Bjorn Oskarsson, M.D.

Open for enrollment

Contact information:

Megan Donahue


More information


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