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Hepatic InsufficiencyPhase 1NCT06390410

A Pharmacokinetic Study of Simufilam in Subjects With Impaired Hepatic Function

A Phase 1 study testing Simufilam in people with Hepatic Insufficiency, run by Cassava Sciences, Inc. at 3 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Evaluate simufilam levels in the blood of hepatically impaired individuals compared to Healthy individuals of similar demographics Read more about this study

Who is it for?
Ages 18 to 75 yearsHealthy volunteers can take part
Conditions the study is for
Hepatic Insufficiency
What is being tested?
Simufilam
Could I receive a placebo?
No, everyone receives an active treatment
How many people
27 participants
Where
3 sitesUnited States
Study dates
26 Jun 2024to 7 Dec 2024
Phase
Phase 1
Are there results?
None posted in the registry

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

Who may be able to join

  • The subject is willing and able to speak, read, and understand English and is willing to provide written informed consent.
  • Male or female subjects between 18 and 75 years of age, inclusive.
  • The subject has a body mass index (BMI) within 18-40 kg/m2.
  • The subject must have renal function that is normal or consistent with mild renal insufficiency.
  • The subject has normal/acceptable mental status for study participation in the opinion of the Investigator.
  • Male subjects and female subjects of childbearing potential must agree to their respective birth control requirements. Females of non-childbearing potential must satisfy the definition of permanent sterility or postmenopausal status
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  • The subject must agree to comply with the drawing of blood samples for the pharmacokinetics (PK) assessments.
  • The subject is willing and able to comply with all testing and requirements defined in the protocol.
  • The subject is willing and able to remain at the study site unit for the duration of the study.
  • The subject must be negative for the COVID virus at both the Screening Visit and the Check-In Visit.
  • Additional inclusion criteria specific for subjects with normal hepatic function:
  • The subject with normal hepatic function is in reasonably good health, determined by no clinically significant findings from medical history, physical examination, 12-lead electrocardiogram (ECG), vital sign measurements, and clinical laboratory evaluations in the opinion of the Investigator and/or Medical Monitor.
  • Additional inclusion criteria specific for subjects with hepatic impairment:
  • Subjects with hepatic impairment with other stable, chronic medical conditions (e.g., hypertension, hyperlipidemia, diabetes) may be included if, in the opinion of the Investigator and Medical Monitor, the abnormality will not significantly alter the disposition of the drug and will not interfere with interpretation of the data.
  • The subject with moderate or mild hepatic impairment has been diagnosed at least 6 months prior to Screening.
  • Moderate: Child-Pugh Class B (7 to 9 points)
  • Mild: Child-Pugh Class A (5 to 6 points) (if a decision is taken to enroll)
  • The subject with hepatic impairment has clinical laboratory tests that are considered clinically stable and consistent with the subject's disease, in the opinion of the investigator.

Who cannot join

  • The subject has a clinically significant electrocardiogram (ECG) abnormality including, but not necessarily limited to, a confirmed QT interval by the Fridericia correction formula (QTcF) > 470 msec (females) or > 450 msec (males) based on WHO 2016 guidelines.
  • The subject has had a clinically significant illness within 30 days prior to the Screening Visit.
  • The subject has a medically significant comorbid illness that would impact the successful conduct of the study or achieving its objectives in the opinion of the Investigator and/or Medical Monitor.
  • The subject has used a known strong CYP2C19 inhibitor (fluconazole, fluoxetine, fluvoxamine or ticlopidine) or inducer (rifampin) within 28 days prior to admission, or 5 half-lives (whichever is longer).
  • The subject has consumed alcohol, grapefruit, grapefruit juice, caffeine- or xanthine-containing products within 72 hours before dosing or intends to use any of these products during the study.
  • The subject has smoked more than 10 cigarettes per day on average for the past 6 months.
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  • The subject has a history of substance abuse within 12 months of the Screening Visit.
  • The subject tests positive on the urine drug screen for drugs of abuse at either the Screening Visit or the Check-In Visit (Note - Subjects with hepatic impairment who are prescribed medications assessed in the urine drug screen [e.g, opiates, benzodiazepines] need not be excluded based on a positive urine drug screen).
  • The subject has a positive ethanol breath test at either the Screening Visit or the Check-In Visit.
  • The subject has a history of regular alcohol consumption defined as greater than 7 drinks per week for women and 14 drinks per week for men within six months prior to the Screening Visit.
  • The subject has a positive serum hepatitis B surface antigen test at the Screening Visit.
  • The subject has a positive human immunodeficiency virus (HIV) test at the Screening Visit.
  • The subject is pregnant or breastfeeding.
  • The subject has received an investigational drug within 30 days or five half-lives - whichever is longer - prior to Study Day 1.
  • The subject has previously received simufilam.
  • The subject has donated or lost a significant volume of blood (>500 mL) within a 90 day period prior to the Screening Visit. This does not include plasma donation.
  • Subjects with poor peripheral venous access.
  • Subjects who, in the opinion of the Investigator (or designee), should not participate in this study.
  • Additional exclusion criteria specific for subjects with normal hepatic function:
  • The subject with normal hepatic function has or has had hepatitis C, as confirmed by a positive hepatitis C virus (HCV) antibody test at the Screening Visit.
  • The subject with normal hepatic function has used prescription or over the counter (OTC) medication within 7 days prior to the Check-In visit, or intends to use any prescription or OTC medication during the study, with the following exceptions:
  • Hormonal contraceptives,
  • Over-the-counter analgesics, stool softeners, and/or multivitamins up to 48 hours prior to study drug administration.
  • Additional exclusion criteria specific for subjects with hepatic impairment:
  • The subject with hepatic impairment has used prescription or OTC medication within 7 days prior to the Check-In Visit except for the management of their hepatic impairment or stable, chronic medical conditions. Such medications must have a stable dose regiment and be approved by the Medical Monitor (Note - over-the-counter analgesics, stool softeners, and/or multivitamins up to 48 hours prior to study drug administration are acceptable).
  • The subject with hepatic impairment has a hemoglobin level < 9.0 (g/dL) or a platelet count < 35×109/L.
  • The hepatically impaired subject with type 2 diabetes on standard oral medication has a HbA1C > 9.5% at either the Screening Visit or the Check-In Visit (Note - use of insulin is not acceptable).
  • The subject with hepatic impairment who has a positive hepatitis C virus (HCV) antibody test also has a positive HCV RNA test (that confirms chronic infection upon reflex testing).
  • The subject with hepatic impairment has evidence of active hepatic encephalopathy > Grade 2 or history of >Grade 2 within the last 6 months. Subject with a history of > Grade 2 hepatic encephalopathy more than 6 months prior to Screening and who currently receives medication for treatment may be allowed, but should be assigned the pre-treatment Child-Pugh score of hepatic encephalopathy.
  • The subject with hepatic impairment has acute hepatitis, severe ascites (defined by the need for frequent paracentesis), hepatocellular carcinoma, current unstable hematological condition, recent upper gastrointestinal bleed, surgical portosystemic shunts, or evidence of hepatorenal syndrome.
  • The subject with hepatic impairment has history of pancreatitis within 4 months of Screening.
  • The subject with hepatic impairment has had a liver transplant prior to Screening.

Trial locations and contacts

3 sites in 1 country.

United States3 sites
  • Orange County Research Center

    Lake Forest, California, 92630

  • Clinical Pharmacology of Miami

    Miami, Florida, 33014

  • Orlando Clinical Research Center

    Orlando, Florida, 32809

Other trials in United States

About this study

Evaluate simufilam levels in the blood of hepatically impaired individuals compared to Healthy individuals of similar demographics

Read the detailed description

This is a Phase I, open label, single-dose study of simufilam 100 mg. Up to 34 subjects may be enrolled; 10 subjects with moderate hepatic impairment, 10 healthy volunteers with normal hepatic function (with the potential of 4 more), and if needed 10 subjects with mild hepatic impairment. Both males and females will be enrolled. The study will be conducted in 2 groups (with the potential of a third) assigned based on degree of hepatic impairment as follows:

up to 10 subjects with moderate hepatic impairment (Child-Pugh score of 7-9) up to 10 health volunteers (matched to each hepatic impairment severity group)

If needed up to 10 subjects with mild hepatic impairment (Child-Pugh score of 5-6), and an additional 4 healthy volunteers if needed to match the mild group

Being tested
Simufilam
Medical subject headings
Hepatic Insufficiency

What participants receive

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

  • SimufilamDrug

    100 mg PTI-125

Study groups in NCT06390410
Study groupRoleWhat they receive
Moderate Hepatic ImpairmentExperimentalNot described
Receives: Drug: Simufilam
Healthy VolunteerExperimentalNot described
Receives: Drug: Simufilam
Mild Hepatic ImpairmentExperimentalNot described
Receives: Drug: Simufilam

What is being measured

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

  1. Area Under the Curve; moderate vs. normal and mild vs. normal

    Area Under the Curve of simufilam concentration in plasma in Moderate and Mild Impairment vs. Normal (each separately)

    Measured: 5 Days

  2. Concentration max; moderate vs. normal and mild vs. normal

    Maximum simufilam concentration in plasma in Moderate and Mild Impairment vs. Normal (each separately)

    Measured: 5 Days

  3. Time max; moderate vs. normal and mild vs. normal

    Time to maximum simufilam concentration in plasma in Moderate and Mild Impairment vs. Normal (each separately)

    Measured: 5 Days

  4. Half-life; moderate vs. normal and mild vs. normal

    Half-life of simufilam concentration in plasma in Moderate and Mild Impairment vs. Normal (each separately)

    Measured: 5 Days

  5. Elimination rate constant; moderate vs. normal and mild vs. normal

    Elimination rate constant of simufilam concentration in plasma in Moderate and Mild Impairment vs. Normal (each separately)

    Measured: 5 Days

Timeline

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

  1. First posted30 Apr 2024
  2. Study start26 Jun 2024
  3. Primary completion7 Dec 2024
  4. Study completion7 Dec 2024

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
Primary purpose
Basic science
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
27 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06390410
Sponsor's protocol ID
PTI-125-11
Status last verified
Jan 2025
First submitted
25 Apr 2024
Last updated
7 Jan 2025
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Simufilam in people with Hepatic Insufficiency, run by Cassava Sciences, 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 18 inclusion and 32 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: finished, as of the sponsor's last update on 7 Jan 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 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.

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

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Area Under the Curve; moderate vs. normal and mild vs. normal (time frame: 5 Days). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
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 7 Jan 2025.

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