Study of ATI-450 in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS)

NCT04524858 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: ATI-450-CAPS-201
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 2

Brief Summary

This is a Phase 2 study to investigate the safety and efficacy of ATI-450 for the Maintenance of Remission in Patients with Cryopyrin-Associated Periodic Syndrome (CAPS) Previously Managed with Anti-IL-1 Therapy.

Conditions

Cryopyrin-Associated Periodic Syndrome

Keywords

Cryopyrin-Associated Periodic Syndrome; CAPS

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs)

  Baseline up to week 12

Study Arms (1)

ATI-450

EXPERIMENTAL

Oral, small molecule MK2 inhibitor will be administered twice daily (BID) at a dose of 50 mg

Drug: ATI-450

Interventions

ATI-450 DRUG

Oral, small molecule MK2 inhibitor

ATI-450

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease. Prior agreement between the Investigator and Aclaris for study eligibility is required for patients who do not have a molecular diagnosis of NALP3 mutations available (either testing not performed, or testing performed, but negative) upon study entry. For those patients who have not been molecularly tested for NALP3 mutations, molecular testing should be performed during the study.
• Patients with a PGA score of "minimal" or less and hsCRP and SAA values within the normal range (≤10mg/L), and who are considered to have achieved that response as a result of successful anti-IL-1 therapy.
• Continuous Treatment with anti-IL1 therapy for at least 6 months.
• Able to understand and comply with study procedures and able to provide informed consent.
• Male or non-pregnant, non-nursing female patients at least 18 years of age, inclusive.
• Female patients who are of childbearing potential must use 2 methods of highly effective contraception\* - one of which must be a physical barrier- for the duration of the study and for 30 days after the last dose.
• Male patients of childbearing potential with a female partner of childbearing potential must agree to use a condom plus another highly effective form of birth control for the duration of the study and for 90 days after the last dose.
• Female patients must have a negative serum pregnancy test at screening and a negative urine pregnancy test prior to dosing on Day 1.
• Willing and capable of taking appropriate Covid-19 risk mitigation precautions (e.g. wearing a mask in public, adhering to social distancing, etc.) as required by local, state, or federal guidelines during participation in the study.

You may not qualify if:

• Participation in any clinical study with an investigative agent within 12 weeks prior to entry or within 5 half-lives of the investigational agent.
• Being treated with another immuno-suppressive agent (i.e., in addition to an anti-IL-1 product) for CAPS syndrome (anti- IL-1 therapy will have been used for at least 6 months and will be stopped at study entry).
• Use of any of the following treatments within the indicated washout period prior to the baseline visit:
• Systemic immunosuppressant or immunomodulatory therapy (e.g., etanercept, alefacept, infliximab, methotrexate) within 16 weeks prior to Visit 2 (excluding anti- IL-1 therapy for CAPS).
• Janus Kinase (JAK) inhibitors (systemic or topical) within 4 weeks prior to Visit 2.
• Systemic corticosteroids within 4 weeks prior to Visit 2 (Intranasal, inhaled, and topical ocular corticosteroids are allowed).
• A positive Hepatitis B surface antigen (HBsAg) or Hepatitis C antibody test result.
• Live vaccinations within 3 months prior to the start of the trial, or during the trial.
• History of recurrent and/or evidence of active bacterial, fungal, or viral infections.
• History or evidence of active or latent tuberculosis (TB).
• Tests performed at a central laboratory at screening that meet any of the criteria below (out of range labs may be rechecked one time, after consultation with sponsor or designee, before patient is considered a screen failure):
• White blood cell (WBC) count \<3.0×103 cells/mm3
• Absolute neutrophil count (ANC) \<1.5×103 cells/mm3
• Lymphocyte count \<0.5×103 cells/mm3
• Platelet count \<100×103 cells/mm3

Sponsors & Collaborators

• Aclaris Therapeutics, Inc.: lead

Study Sites (2)

Aclaris Investigational Site

San Diego, California, 92123, United States

Location

Aclaris Investigational Site

San Francisco, California, 94116, United States

Location

MeSH Terms

Conditions

Cryopyrin-Associated Periodic Syndromes

Condition Hierarchy (Ancestors)

Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases; Chronic Inducible Urticaria; Chronic Urticaria; Urticaria; Skin Diseases, Vascular; Cold Urticaria; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Ajay Aggarwal
• Organization: Aclaris Therapeutics, Inc.

Clintrials@aclaristx.com

484-540-6296

Study Officials

• David Gordon

  Aclaris Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open-label, single-arm study

Study Record Dates

• First Submitted: August 19, 2020
• First Posted: August 24, 2020
• Study Start: October 23, 2020
• Primary Completion: February 25, 2021
• Study Completion: February 25, 2021
• Last Updated: September 21, 2023
• Results First Posted: September 21, 2023

Record last verified: 2023-08

Locations

US (2)