A Study of PATAS Trifluoroacetate Using Single Ascending Doses in Healthy Volunteers and Multiple Ascending Doses in Subjects With Type 2 Diabetes

NCT ID: NCT07223333

Last Updated: 2025-10-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE1

Total Enrollment

56 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-12-10

Study Completion Date

2026-09-30

Brief Summary

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The primary objective of Part 1 of this study is to evaluate safety and tolerability of single subcutaneous (SC) doses of PATAS in healthy subjects. The secondary objective of Part 1 of this study is to determine the pharmacokinetics (PK) of single SC doses of PATAS in healthy subjects. The primary objectives of Part 2 of this study are to evaluate the safety and tolerability of 4 weekly SC doses of PATAS in subjects with T2D; and to determine the PK and pharmacodynamics (PD) of 4 weekly SC doses of PATAS in subjects with T2D. The secondary objectives of Part 2 of this study are to evaluate the potential effect of multiple SC doses of PATAS on markers of glycemic control, as measured by glucose levels, insulin levels, and other metabolomic biomarkers; and to characterize the adverse event (AE) profiles of the various dose levels of PATAS.

Detailed Description

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Conditions

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Type 2 Diabetes

Keywords

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Type 2 diabetes insulin resistance

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Healthy subjects, Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Excipient only formulation, without active compound

Healthy Subjects, Active

Group Type EXPERIMENTAL

PATAS Trifluoroacetate

Intervention Type DRUG

A drug targeting the interaction between the ALMS1 protein and alpha-PKC

Diabetes Subjects, Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Excipient only formulation, without active compound

Diabetes Subjects, Active

Group Type EXPERIMENTAL

PATAS Trifluoroacetate

Intervention Type DRUG

A drug targeting the interaction between the ALMS1 protein and alpha-PKC

Interventions

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PATAS Trifluoroacetate

A drug targeting the interaction between the ALMS1 protein and alpha-PKC

Intervention Type DRUG

Placebo

Excipient only formulation, without active compound

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Healthy male and female subjects, 18 to 55 years of age, inclusive, at the time of signing the Informed Consent Form (ICF);
2. Willing and able to give written informed consent for participation in the study prior to the initiation of any Screening or study-specific procedures;
3. Body mass index (BMI) within the range of 20.0 to 35.0 kg/m2, inclusive, at Screening;
4. In generally good health, as judged by the Investigator, based upon medical/surgical history and the results of physical examination, vital signs, clinical laboratory assessments, and 12-lead electrocardiogram (ECG) at Screening and at Check-In (Day -1);
5. Female subjects must have a negative serum pregnancy test result at the Screening Visit and a negative urine pregnancy test at Check-In (Day -1) (prior to the first dose of study drug) and must not be pregnant, lactating, or planning a pregnancy from the Screening Visit to 90 days after the last dose of study drug;
6. Negative test result for severe acute respiratory syndrome coronavirus 2 at Check-In (Day -1); and
7. Willing to comply with all study procedures and requirements throughout the duration of the study.


1. Male and female subjects, 18 to 65 years of age, inclusive, at the time of signing the ICF;
2. Willing and able to give written informed consent for participation in the study prior to the initiation of any Screening or study-specific procedures;
3. A diagnosis of T2D \>1 year before Screening;
4. Subjects should be on a stable dose of an oral monotherapy (permitted monotherapies include metformin and dipeptidyl peptidase 4 inhibitors) for at least 90 days before Screening or managing the condition through diet and exercise for at least 90 days before Screening;
5. Glycated hemoglobin ³7.5% and \<9.5% at Screening;
6. BMI within the range of 25.0 to 40.0 kg/m2, inclusive, at Screening;
7. In generally good health, as judged by the Investigator, based upon medical/surgical history and the results of physical examination, vital signs, clinical laboratory assessments, and 12-lead ECG at Screening and at the pre-dose Check-In (Day -1);
8. Female subjects must have a negative serum pregnancy test result at the Screening Visit and a negative urine pregnancy test at the pre-dose Check-In (Day -1) (prior to the first dose of study drug) and must not be pregnant, lactating, or planning a pregnancy from the Screening Visit to 90 days after the last dose of study drug;

Exclusion Criteria

1. Clinically significant history of asthma, eczema, or any other allergic condition or previous severe hypersensitivity; Note: Non-active hay fever is not exclusionary.
2. Liver function tests (alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], alkaline phosphatase \[ALP\], total bilirubin) outside the following upper limits of normal (ULNs) at Screening or at Check-In (Day -1): a. For ALT and AST, measurements \>1.5 × ULN; b. For ALP, measurements \>2 × ULN; or c. For total bilirubin, measurements \>1.5 × ULN.
3. Estimated glomerular filtration rate £60 mL/min/1.73 m2 based on the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation at Screening or at Check-In (Day -1);
4. Thyroid-stimulating hormone (TSH) outside of reference range (e.g., TSH \<1 × lower limit of normal \[LLN\] or TSH \>1 × ULN) at Screening; Note: Abnormal TSH results will reflex to a free thyroxine (T4) test.
5. History of unexplained syncope, cardiac arrest, unexplained cardiac arrythmias or torsades de pointes, or structural heart disease;
6. Personal or family history of long QT syndrome;
7. Clinically significant history of any disease or disorder (i.e., gastrointestinal, cardiovascular, respiratory, renal, hepatic, neurological, dermatological, psychiatric, or metabolic) deemed to be exclusionary, as judged by the Investigator;
8. Abnormal pulse rate or blood pressure (BP) measurements at Screening, defined as: a. Pulse rate \<40 bpm or \>100 bpm; b. Systolic BP \<90 mmHg or \>140 mmHg; or c. Diastolic BP \<50 mmHg or \>90 mmHg.
9. Clinically significant ECG abnormalities at Screening or at Check-In (Day -1), defined as prolongation of the average QTcF interval \>450 ms for males and \>470 ms for females, or other clinically significant ECG abnormalities per Investigator discretion;
10. Positive for hepatitis B surface antigen, HIV antibody, or hepatitis C virus antibody at Screening;
11. Receipt of any investigational product within 30 days prior to first study drug administration (90 days for investigational biologic agents) or 5 half-lives prior to first study drug administration, whichever is greater, or participation in \>3 clinical studies within 12 months; 22. Known or suspected hypersensitivity to PATAS or any components of the formulation used (sodium hydroxide or mannitol);


1. Poorly controlled diabetes with fluctuating blood sugars, as judged by the Investigator;
2. History of diabetic ketoacidosis or hyperosmolar coma in the 6 months prior to Screening;
3. History of level 3 hypoglycemia in the 6 months prior to Screening or a history of hypoglycemia unawareness;
4. History or current evidence of type 1 diabetes or any other form of diabetes (e.g., latent autoimmune diabetes in adults, maturity onset diabetes of the young, or secondary diabetes);
5. History of active or uncontrolled diabetic complications (i.e., neuropathy, retinopathy, nephropathy, gastroparesis); Note: Diabetic complications that are, in the opinion of the Investigator, stable in condition are permitted.
6. Unwilling or unable to use the study-provided continuous glucose monitor (CGM); Note: Subjects currently utilizing a personal CGM device for monitoring their blood glucose levels and are unwilling to discontinue use or planning to utilize a personal CGM device for monitoring their blood glucose levels are excluded from the study. Note: Subjects using a glucometer to monitor blood glucose levels should continue using the device during the study, per standard of care.
7. Clinically significant history of asthma, eczema, or any other allergic condition or previous severe hypersensitivity; Note: Non-active hay fever is not exclusionary.
8. Has an active or untreated malignancy or has been in remission from malignancy for £5 years except well-treated basal cell skin cancer or cervical cancer in situ;
9. Liver function tests (ALT, AST, ALP, total bilirubin) outside the following ULNs at Screening or at Check-In (Day -1): a. For ALT and AST, measurements \>1.5 × ULN; b. For ALP, measurements \>2 × ULN; or c. For total bilirubin, measurements \>1.5 × ULN.
10. TSH \<1 × LLN or TSH \>1.5 × ULN at Screening; Note: Abnormal TSH results will reflex to a T4 test.
11. Uncontrolled hypothyroidism or hyperthyroidism, as judged by the Investigator; Note: If a subject is on levothyroxine therapy, the subject should be on a stable dose for at least 6 weeks prior to Screening.
12. Estimated glomerular filtration rate £60 mL/min/1.73 m2 based on the CKD-EPI equation at Screening or at the pre-dose Check-In (Day -1);
13. History of unexplained syncope, cardiac arrest, unexplained cardiac arrythmias or torsades de pointes, or structural heart disease;
14. Personal or family history of long QT syndrome;
15. Clinically significant history of any disease or disorder (i.e., gastrointestinal, cardiovascular, respiratory, renal, hepatic, dermatological, or psychiatric) deemed to be exclusionary, as judged by the Investigator;
16. Abnormal pulse rate or BP measurements at Screening, defined as: a. Pulse rate \<40 bpm or \>100 bpm; b. Systolic BP \<90 mmHg or \>150 mmHg; or c. Diastolic BP \<50 mmHg or \>90 mmHg.
17. Clinically significant ECG abnormalities at Screening or at Check-In (Day -1), defined as prolongation of the average QTcF interval \>450 ms for males and \>470 ms for females, or other clinically significant ECG abnormalities per Investigator discretion;
18. Any other clinically significant laboratory abnormality deemed to be exclusionary, as judged by the Investigator;
19. Use of insulin, sulfonylureas, peroxisome proliferator activated receptor gamma agonists, sodium-glucose transport protein 2 inhibitors, pramlintide, or glucagon-like peptide-1 receptor agonists within 90 days prior to Screening;
20. Use of any immunosuppressants or systemic steroids for ³7 days in the 90 days prior to the pre-dose Check-In (Day -1);
21. Positive urine drug screen (drugs of abuse) or urine cotinine test at Screening and/or the pre-dose Check-In (Day -1) or a history of drug/chemical abuse within 1 year prior to Screening;
22. Use of tobacco- or nicotine-containing products within 90 days prior to Screening or an unwillingness to abstain from the use of tobacco- or nicotine-containing products during the study;
23. Have typical weekly alcohol consumption of ³14 alcoholic drinks. One drink
24. Positive for hepatitis B surface antigen, HIV antibody, or hepatitis C virus antibody at Screening;
Minimum Eligible Age

18 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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AdipoPharma LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Vincent Marion, Ph.D.

Role: STUDY_CHAIR

AdipoPharma LLC

Locations

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Medpace Clinical Pharmaology Unit

Cincinnati, Ohio, United States

Site Status

Countries

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United States

Central Contacts

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Tarek Hiwot, M.D.

Role: CONTACT

Phone: 44 7391 537 158

Email: [email protected]

Michael J Fare

Role: CONTACT

Phone: 203-671-4351

Email: [email protected]

Other Identifiers

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PATAS-CL-001

Identifier Type: -

Identifier Source: org_study_id