A Study to Evaluate the Effects of ASP5502 in Healthy Adults and Adults With Primary Sjögren's Syndrome

NCT06544642 | Terminated Phase 1 FDA Drug

• 1 other identifier: 5502-CL-0001
• Study Type: interventional
• Target: 116
• Locations: 1 country
• Sites: 1

Brief Summary

Primary Sjögren's syndrome (pSS) is a condition when the body's immune system attacks glands that produce fluids, such as the tear and saliva glands. This leads to dry eyes and a dry mouth. However, other symptoms may occur such as fatigue, joint pain, and skin problems. These symptoms can be severe. Symptoms can be treated but there is an unmet need to treat the actual condition. In this study, ASP5502 is being given to humans for the first time. The people taking part are healthy adults or adults with pSS. The main aims of the study are to check the safety of ASP5502 and how people tolerate ASP5502. This study will be in 3 parts. In Part 1, healthy men and women will take tablets of ASP5502 or a placebo just once. In this study, the placebo looks like the ASP5502 tablet but doesn't have any medicine in it. Different small groups of people will take a lower to a higher dose of ASP5502 or a placebo. This will happen one group after another. One small group will take tablets of ASP5502 or placebo with and without food. This is to find out if food affects how the body processes ASP5502. After their dose, people will stay in the medical center for a few nights. This is to have blood tests, electrocardiograms (ECGs) to check heart health, and other safety checks, and to report any medical problems. One of these checks is to have their heart continuously tracked during the first night. This is called telemetry. People who take tablets of ASP5502 or placebo with and without food will stay in the medical center for a few extra nights. In Part 2, healthy men and women will take tablets of ASP5502 or a placebo. They will do this once a day for 2 weeks (14 days). Different small groups of people will take a lower to a higher dose of ASP5502 or a placebo. This will happen one group after another. After taking ASP5502 or the placebo, people will stay in the medical center for a few nights. This is to have blood tests, ECGs to check heart health, and other safety checks, and to report any medical problems. Telemetry will also be done continuously during the first night. In Part 3, men and women with pSS will take tablets of ASP5502. They will do this once a day for 4 weeks (28 days). Different small groups of people will take a lower to a higher dose of ASP5502. This will either happen for one group after another, or just for 1 group. The number of groups and the doses taken will be worked out from the results from Part 1 and Part 2 of this study. People will stay in the medical center for a couple of nights. This will happen for their first dose, then again after about 2 weeks and 4 weeks of treatment. As in Parts 1 and 2, this is to have blood tests, ECGs to check heart health, and other safety checks, and to report any medical problems. In all parts of the study, people will return to the medical center about 1 week after their final blood sample is taken, for health check. People in parts 2 and 3 will also receive a telephone call safety check about 4 weeks after their last dose of ASP5502.

Conditions

Healthy Volunteers; Sjögren's Syndrome

Keywords

ASP5502; Safety; Tolerability; Pharmacokinetics; Pharmacodynamics; Food Effect

Outcome Measures

Primary Outcomes (4)

• Number of Participants With Adverse Events (AEs)

  An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study intervention. This includes events related to the comparator, if applicable, and events related to the (study) procedures.

  Up to Day 58

• Number of participants with laboratory value abnormalities and/or AEs

  Number of participants with potentially clinically significant laboratory values.

  Up to Day 38

• Number of participants with vital sign abnormalities and/or AEs

  Number of participants with potentially clinically significant vital sign values.

  Up to Day 38

• Number of participants with 12-lead ECG abnormalities and/or AEs

  Number of participants with potentially clinically significant 12-lead ECG values.

  Up to Day 38

Secondary Outcomes (7)

• Pharmacokinetics (PK) of ASP5502 in plasma: maximum concentration (Cmax)

  Up to Day 29

• PK of ASP5502 in plasma: area under the concentration-time curve from the time of dosing to the last measurable concentration (AUClast) [Part 1]

  Up to Day 7

• PK of ASP5502 in plasma: area under the concentration-time curve from the time of dosing extrapolated to time infinity (AUCinf) [Part 1]

  Up to Day 7

• PK of ASP5502 in plasma: area under the concentration-time curve from time of dosing to 24 hours post dose (AUC24) [Part 2 & Part 3]

  Up to Day 2

• PK of ASP5502 in plasma: area under the concentration-time curve during a dosing interval, where tau is the length of the dosing interval (AUCtau) [Part 2 & Part 3]

  Up to Day 29

Study Arms (4)

Part 1 - Single Ascending Dose in Healthy participants (Dose Escalation Cohort)

EXPERIMENTAL

Participants will receive a single oral dose of ASP5502 or Placebo on Day 1.

Drug: ASP5502; Drug: Placebo

Part 1 - Single Ascending Dose in Healthy participants (Food Effect Cohort)

EXPERIMENTAL

Participants will receive a single oral dose of ASP5502 or Placebo under fasting conditions on period 1 Day 1 and single dose of ASP5502 or Placebo under fed conditions on period 2 Day 1.

Drug: ASP5502; Drug: Placebo

Part 2 - Multiple Ascending Dose in Healthy participants

EXPERIMENTAL

Participants will receive daily oral doses of ASP5502 or placebo for 14 days.

Drug: ASP5502; Drug: Placebo

Part 3 - Repeated Dose in Participants with pSS

EXPERIMENTAL

Participants will receive daily oral doses of ASP5502 for 28 days.

Drug: ASP5502

Interventions

ASP5502 DRUG

Oral

Part 1 - Single Ascending Dose in Healthy participants (Dose Escalation Cohort); Part 1 - Single Ascending Dose in Healthy participants (Food Effect Cohort); Part 2 - Multiple Ascending Dose in Healthy participants; Part 3 - Repeated Dose in Participants with pSS

Placebo DRUG

Oral

Part 1 - Single Ascending Dose in Healthy participants (Dose Escalation Cohort); Part 1 - Single Ascending Dose in Healthy participants (Food Effect Cohort); Part 2 - Multiple Ascending Dose in Healthy participants

Eligibility Criteria

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

You may qualify if:

• Participant is healthy and has no clinically significant medical conditions based on the review of the physical examination, ECG and protocol-defined clinical laboratory tests at screening or on day -2 or day -1.
• Female participant is not pregnant and at least 1 of the following conditions apply:
• Not a women of childbearing potential (WOCBP)
• WOCBP who has a negative blood pregnancy test at screening and a urine pregnancy test on day -2 and agrees to follow the contraceptive guidance from the time of informed consent through at least 5 half-lives or 30 days, whichever is longer, after final study intervention administration. Female participants on hormonal contraceptives must also be using a double barrier method as defined in Contraceptive Requirements.
• Female participant must not be breastfeeding or lactating starting at screening and throughout the investigational period and for 5 half-lives or 30 days, whichever is longer, after final study intervention administration.
• Female participant must not donate ova starting at first administration of study intervention and throughout the investigational period and for 5 half-lives or 30 days, whichever is longer, after final study intervention administration.
• Male participant must agree to use contraception with female partner(s) of childbearing potential (including breastfeeding partner) throughout the treatment period and for 5 half-lives or 30 days, whichever is longer, after final study intervention administration.
• Male participant must agree to remain abstinent or use a condom with pregnant partner(s) for the duration of the pregnancy throughout the investigational period and for 5 half-lives or 30 days, whichever is longer, after final study intervention administration.
• Male participant must not donate sperm during the treatment period and for 5 half-lives or 30 days, whichever is longer, after final study intervention administration.
• Participant agrees not to participate in another interventional study while participating in the present study from the time of signing informed consent through the end of study visit.
• Participant has a body mass index (BMI) range of 18.5 to 30.0 kg/m\^2 inclusive and weighs at least 50 kg for male and 40 kg for female at screening.
• Participant is diagnosed based on the 2016 American College of Rheumatology (ACR)- European Alliance of Associations for Rheumatology (EULAR) Classification Criteria for pSS. Diagnosis should have been established at least 6 months prior to day -1 and no clinically significant medical condition is present on the physical examination, ECG and protocol-defined clinical laboratory tests at screening or on day -1.
• Female participant is not pregnant and at least 1 of the following conditions apply:
• Not a WOCBP
• WOCBP who has a negative blood pregnancy test at screening and a urine pregnancy test on day -1 and agrees to follow the contraceptive guidance from the time of informed consent through at least 5 half-lives or 30 days, whichever is longer, after final study intervention administration. Female participants on hormonal contraceptives are allowed ONLY if the participant also agrees to use a double barrier method (condom and spermicide) and the results from the 4β hydroxycholesterol assessment in Part 2 are negative.

You may not qualify if:

• Participant has been pregnant within 6 months prior to screening.
• Participant has any history or evidence of any clinically significant cardiovascular, gastrointestinal, endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal and/or other major disease or malignancy.
• Participant has a history of malignancy within 2 years before screening (exceptions are squamous and basal cell carcinomas of the skin and carcinoma in situ of the cervix, or malignancy that is considered cured with minimal risk of recurrence).
• Participant has had major surgery (e.g., requiring general anesthesia) within 90 days before screening, or will not have fully recovered from surgery, or has surgery planned during the time the participant is expected to participate in the study or within 5 half-lives after the last dose of study intervention administration or end of study visit, whichever is longer.
• Participant has/had febrile illness or symptomatic, viral, bacterial (including upper respiratory infection) or fungal (noncutaneous) infection within 28 days prior to day -1.
• Participant has a positive QuantiFERON®-TB Gold test at screening.
• Participant has a history of drug or alcohol abuse according to Diagnostic and Statistical Manual of Mental Disorders (5th edition) (DSM-5) criteria within 2 years before screening.
• Participant has a history of unexplained syncope, cardiac arrest, unexplained cardiac arrhythmias or torsades de pointes, structural heart disease, a family history of long QT syndrome or taking any QT prolonging medication.
• Participant has used any prescribed or nonprescribed drugs (including vitamins and natural and herbal remedies, e.g., St. John's Wort) in the 28 days prior to study intervention administration, except for contraceptive use, hormone replacement therapy (HRT) use and occasional use of acetaminophen (up to 2 g/day).
• Participant has used any inducer of metabolism (e.g., barbiturates and rifampin) in the 3 months prior to day -1.
• Participant has received a vaccine within the 2 weeks prior to day -1 or will have a vaccine dose before the follow-up visit.
• Participant has received any investigational therapy within 28 days or 5 half-lives, whichever is longer, prior to screening.
• Participant has any of the liver safety monitoring panel (alkaline phosphatase \[ALP\], alanine transaminase \[ALT\], aspartate aminotransferase \[AST\] and total bilirubin \[TBL\]) above the upper limit of normal (ULN) on day -2. In such a case, the assessment may be repeated once.
• Participant has creatinine level outside normal limits on day -2. In such a case, the assessment may be repeated once.
• Participant has a mean pulse \< 45 or \> 90 bpm; mean systolic blood pressure \> 140 mmHg; mean diastolic blood pressure \> 90 mmHg (measurements taken in triplicate after participant has been resting in the supine position for at least 5 minutes; pulse will be measured automatically) on day -2. If the mean blood pressure exceeds the limits above, 1 additional triplicate may be taken.

Sponsors & Collaborators

• Astellas Pharma Global Development, Inc.: lead

Study Sites (1)

Fortrea Clinical Research Unit Inc

Daytona Beach, Florida, 32117, United States

Location

MeSH Terms

Conditions

Sjogren's Syndrome

Condition Hierarchy (Ancestors)

Arthritis, Rheumatoid; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Xerostomia; Salivary Gland Diseases; Mouth Diseases; Stomatognathic Diseases; Dry Eye Syndromes; Lacrimal Apparatus Diseases; Eye Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Medical Director

  Astellas Pharma Global Development, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: BASIC SCIENCE
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 6, 2024
• First Posted: August 9, 2024
• Study Start: August 21, 2024
• Primary Completion: March 5, 2026
• Study Completion: March 5, 2026
• Last Updated: March 25, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)