A Phase 1 Combined Single and Multiple Ascending Oral Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Food Effect and Biomarker Response of ASP5502 in Healthy Adults and Participants With Primary Sjögren's Syndrome

Plain English Summary

A Study to Evaluate the Effects of ASP5502 in Healthy Adults and Adults With Primary Sjögren's Syndrome is a Phase 1 clinical trial sponsored by Astellas Pharma Global Development, Inc. studying Healthy Volunteers, Sjögren's Syndrome. This trial tested the safety and how well ASP5502 is tolerated in healthy adults and those with primary Sjögren's Syndrome. It was designed for adults diagnosed with primary Sjögren's Syndrome or healthy volunteers. Participation involved taking ASP5502 or a placebo (inactive substance) orally, with some participants staying in a medical center for monitoring. The trial was terminated, meaning it did not complete as planned, and no alternative treatments were directly compared within this study. The trial aims to enroll 116 participants.

Official 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, an

Who Can Participate

Here is what you need to know about eligibility for this trial. Adults aged 18 and over can participate if they are healthy or have a confirmed diagnosis of primary Sjögren's Syndrome for at least 6 months. Participants must have a Body Mass Index (BMI) between 18.5 and 30.0 and meet specific weight requirements. Individuals with significant medical conditions, pregnant or breastfeeding women, and those participating in other clinical trials are excluded. Strict contraceptive measures are required for all participants of childbearing potential, both male and female, throughout the study and for a period afterward. This trial is studying Healthy Volunteers, Sjögren's Syndrome, so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcomes measured the safety of ASP5502 by tracking adverse events, and any changes in vital signs, laboratory results, or heart rhythm (ECG) that might be related to the drug. The specific primary outcome measures are: Number of Participants With Adverse Events (AEs) (Up to Day 58); Number of participants with laboratory value abnormalities and/or AEs (Up to Day 38); Number of participants with vital sign abnormalities and/or AEs (Up to Day 38); Number of participants with 12-lead ECG abnormalities and/or AEs (Up to Day 38). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 1, the first major stage of clinical testing. Phase 1 trials typically involve 20-100 participants and focus on safety, dosage levels, and side effects. The primary goal is not to test whether the treatment works but to establish that it is safe enough for further testing. About 70% of Phase 1 drugs advance to Phase 2. If successful, the treatment will proceed to Phase 2 efficacy testing.

Why This Trial Matters

This trial aimed to assess a new drug, ASP5502, for primary Sjögren's Syndrome, addressing an unmet need for treatments that target the condition itself, not just its symptoms. This research targets Healthy Volunteers, Sjögren's Syndrome, where improved treatment options are needed.

Investor Insight

The termination of this Phase 1 trial suggests potential safety or tolerability concerns with ASP5502, impacting its probability of reaching later-stage development and market approval for Sjögren's S Phase 1 trials have approximately a 10% chance of eventually gaining FDA approval.

Is This Trial Right for Me?

Ask your doctor about the specific risks and what is known about ASP5502's side effects. Understand that you may receive either the study drug or a placebo, and your participation will involve regular medical check-ups and tests. Be prepared for potential overnight stays in a medical facility for monitoring and to report any new or worsening symptoms promptly. The trial is being conducted at 1 site. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: SEQUENTIAL
• Masking: DOUBLE
• Enrollment: 116 participants

Interventions

• DRUG: ASP5502 — Oral
• DRUG: Placebo — Oral

Primary Outcomes

• Number of Participants With Adverse Events (AEs) (Up to Day 58)
• Number of participants with laboratory value abnormalities and/or AEs (Up to Day 38)
• Number of participants with vital sign abnormalities and/or AEs (Up to Day 38)
• Number of participants with 12-lead ECG abnormalities and/or AEs (Up to Day 38)

Secondary Outcomes

• 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)

Full Eligibility Criteria

Inclusion Criteria:

Inclusion Criteria for Healthy Participants

* 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.

Inclusion Criteria for Participants with pSS

* 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.
  * WOCBP 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 BMI range of 18.5 to 30.0 kg/m\^2 inclusive and weighs at least 50 kg for male and 40 kg for fe

Trial Locations

• Fortrea Clinical Research Unit Inc, Daytona Beach, Florida, United States

Frequently Asked Questions

Related Conditions

• Sjögren's Syndrome
• Autoimmune Diseases
• Rheumatology
• Ophthalmology (Dry Eye)
• Gastroenterology (Dry Mouth)
• Immunology
• Inflammatory Conditions
• Fatigue
• Joint Pain
• Skin Conditions

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