A Study to Evaluate AEVI-007 in Participants With Adult Onset Still's Disease

• ClinicalTrials.gov Identifier: NCT04752371
• Recruitment Status: Recruiting
• First Posted: February 12, 2021
• Last Update Posted: February 18, 2022
• Sponsor: Cerecor Inc
• Information provided by (Responsible Party): Cerecor Inc

Study Description

Brief Summary:

The main purpose of the study is to evaluate the safety and tolerability of AEVI-007 in participants with Adult Onset Still's Disease (AOSD).

Condition or disease	Intervention/treatment	Phase
Adult Onset Still's Disease	Drug: AEVI-007 (CERC-007, AVTX-007)	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 12 participants
• Allocation: Non-Randomized
• Intervention Model: Sequential Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase 1b, Multicenter, Open-Label Study to Evaluate the Safety and Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AEVI-007 in Subjects With Adult Onset Still's Disease
• Actual Study Start Date: March 25, 2021
• Estimated Primary Completion Date: March 30, 2022
• Estimated Study Completion Date: March 30, 2022

Arms and Interventions

Arm	Intervention/treatment
Experimental: Cohort 1: AEVI-007 7 mg/kg; 6 participants will be administered AEVI-007 at a dose of 7 mg/kg (500 mg maximum) at Baseline, Week 4 and Week 8. Based on safety results in first 6 participants, additional participants may be enrolled in this cohort.	Drug: AEVI-007 (CERC-007, AVTX-007); Intravenous (IV) Infusion
Experimental: Cohort 2: AEVI-007 Dose escalation/reduction; Based on safety results in Cohort 1, 6 participants will be administered AEVI-007 at a dose of 14 mg/kg (1000 mg maximum) at Baseline, Week 4 and Week 8, or may receive a lower dose (4 mg/kg, maximum 300 mg). Dose in participants receiving 14 mg/kg may be reduced based on safety results.	Drug: AEVI-007 (CERC-007, AVTX-007); Intravenous (IV) Infusion

Outcome Measures

Primary Outcome Measures :

1. Incidence of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Baseline to Week 12 ]
2. Incidence of Clinically Significant Changes from Baseline in Vital Signs [ Time Frame: Baseline to Week 12 ]
3. Incidence of Clinically Significant Changes from Baseline in Clinical Laboratory Results [ Time Frame: Baseline to Week 12 ]

Secondary Outcome Measures :

1. Number of Participants Who Achieved Resolution of Fever [ Time Frame: Baseline to Week 12 ]
   Resolution of fever is defined as no temperature >38°C for 48 hours
2. Number of Participants Whose C-Reactive Protein (CRP) Levels Reduced by at Least 50% by Week 4 [ Time Frame: Baseline to Week 4 ]
3. Number of Participants Whose C-Reactive Protein (CRP) Levels Reduced by at Least 50% by Week 12 [ Time Frame: Baseline to Week 12 ]
4. Change from Baseline to Week 4 in the Physician Global Assessment of Disease Activity [ Time Frame: Baseline to Week 4 ]
   Physician global assessment of disease activity will be assessed using a Visual Analogue Scale (VAS) where 0 mm indicates no evidence of disease and 100 mm indicates very severe disease activity.
5. Change from Baseline to Week 12 in the Physician Global Assessment of Disease Activity [ Time Frame: Baseline to Week 12 ]
   Physician global assessment of disease activity will be assessed using a Visual Analogue Scale (VAS) where 0 mm indicates no evidence of disease and 100 mm indicates very severe disease activity.
6. Change from Baseline to Week 4 in the Patient Global Assessment of Disease Activity [ Time Frame: Baseline to Week 4 ]
   Patient global assessment of disease activity will be assessed using a Visual Analogue Scale (VAS) where 0 mm indicates no evidence of disease and 100 mm indicates very severe disease activity.
7. Change from Baseline to Week 12 in the Patient Global Assessment of Disease Activity [ Time Frame: Baseline to Week 12 ]
   Patient global assessment of disease activity will be assessed using a Visual Analogue Scale (VAS) where 0 mm indicates no evidence of disease and 100 mm indicates very severe disease activity.
8. Change from Baseline to Week 4 in the Modified Pouchot Score [ Time Frame: Baseline to Week 4 ]
9. Change from Baseline to Week 12 in the Modified Pouchot Score [ Time Frame: Baseline to Week 12 ]
10. Change from Baseline to Week 4 in the Disease Activity Score-28 with C-Reactive Protein (DAS28-CRP) [ Time Frame: Baseline to Week 4 ]
11. Change from Baseline to Week 12 in the Disease Activity Score-28 with C-Reactive Protein (DAS28-CRP) [ Time Frame: Baseline to Week 12 ]
12. Change from Baseline to Week 4 in C-Reactive Protein Levels [ Time Frame: Baseline to Week 4 ]
13. Change from Baseline to Week 12 in C-Reactive Protein Levels [ Time Frame: Baseline to Week 12 ]
14. Change from Baseline to Week 4 in Ferritin Levels [ Time Frame: Baseline to Week 4 ]
15. Change from Baseline to Week 12 in Ferritin Levels [ Time Frame: Baseline to Week 12 ]
16. Change from Baseline to Week 4 in Erythrocyte Sedimentation Rate (ESR) [ Time Frame: Baseline to Week 4 ]
17. Change from Baseline to Week 12 in Erythrocyte Sedimentation Rate (ESR) [ Time Frame: Baseline to Week 12 ]
18. Serum Concentration of AEVI-007 [ Time Frame: Baseline and Weeks 1, 2, 4, 8 and 12 ]

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 75 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

1. Participant is 18 to 75 years of age (inclusive) at the time of consent.

2. Participant has been diagnosed with AOSD based on classification criteria (according to Yamaguchi et al, 1992) defined as having 5 or more of the following criteria, 2 of which are major:

   1. Major Criteria

      • Fever >39°C, lasting 1 week or longer
      • Arthralgia or arthritis, lasting 2 weeks or longer
      • Typical rash
      • Leukocytes >10,000 mm^3 with >80% polymorphonuclear cells

   2. Minor Criteria

      • Sore throat
      • Recent development of significant lymphadenopathy
      • Hepatomegaly or splenomegaly
      • Abnormal liver function tests
      • Negative tests for antinuclear antibody and rheumatoid factor
3. Participant has reported a recurring fever >38°C, consistent with active disease, within the last 5 days of the Screening and Baseline visits.
4. If undergoing treatment with nonsteroidal anti-inflammatory drugs (NSAIDs), participant is on a stable dose for at least 48 hours prior to the Baseline Visit (Visit 2).
5. If undergoing treatment with glucocorticoids, participant is on a stable dose for at least 48 hours prior to the Baseline Visit (Visit 2).
6. If undergoing treatment with conventional disease-modifying antirheumatic drugs (DMARDs), participant is on a stable dose for at least 4 weeks prior to the Baseline Visit (Visit 2).

7. For participants who have received treatment with biological DMARDs, participant has the required washout (normalization) period prior to the Baseline Visit (Visit 2). The washout (normalization) period for biological DMARDs is as follows:

   1. Anakinra - 1 week
   2. Etanercept, rilonacept - 4 weeks
   3. Adalimumab, certolizumab, infliximab, golimumab, abatacept, tocilizumab and canakinumab - 8 weeks
   4. Rituximab - 36 weeks

Exclusion Criteria:

1. Participant has another serious chronic-inflammatory disease.
2. Participant has a relevant, active infection or another disease, which entails a tendency towards infection.
3. Participant has active macrophage activation syndrome.

4. Participant has the following abnormal values:

   1. Serum creatinine concentration >1.5 mg/dl.
   2. Hemoglobin ≤ 10 g/dl, neutrophils ≤1,500 /μl and/or thrombocytes ≤75,000 /μl.

Contacts and Locations

Contacts

Contact: Barbara Sabatino; 610-977-2772; bsabatino@avalotx.com

Locations

United States, California

Sponsor Investigative Site; Recruiting

La Mesa, California, United States, 91942

Sponsor Investigative Site; Recruiting

San Diego, California, United States, 92108

United States, Florida

Sponsor Investigative Site; Recruiting

Gainesville, Florida, United States, 32610

United States, Maryland

Sponsor Investigative Site; Recruiting

Columbia, Maryland, United States, 21046

United States, Michigan

Sponsor Investigative Site; Recruiting

Ann Arbor, Michigan, United States, 48109

Belgium

Sponsor Investigative Site; Recruiting

Gent, Oost-Vlaanderen, Belgium, 9000

Poland

Sponsor Investigative Site; Recruiting

Elbląg, Poland, 82-300

Sponsor Investigative Site; Recruiting

Pomorskie, Poland, 85-168

Sponsor Investigative Site; Recruiting

Poznań, Poland, 61-113

Sponsor Investigative Site; Recruiting

Poznań, Poland, 61-545

Ukraine

Sponsor Investigative Site; Recruiting

Kyiv, Ukraine, 03151

Sponsor Investigative Site; Recruiting

Poltava, Ukraine, 36011

Sponsor Investigative Site; Recruiting

Ternopil', Ukraine, 46002

Sponsor Investigative Site; Recruiting

Vinnitsa, Ukraine, 21018

Sponsors and Collaborators

Cerecor Inc

Investigators

• Principal Investigator: Isabelle Peene, MD, PhD; University Hospital, Ghent

More Information

• Responsible Party: Cerecor Inc
• ClinicalTrials.gov Identifier: NCT04752371
• Other Study ID Numbers: AEVI-007-AOSD-101; 2020-004099-16 ( EudraCT Number )
• First Posted: February 12, 2021
• Last Update Posted: February 18, 2022
• Last Verified: February 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Cerecor Inc:

Adult Onset Still's Disease; AEVI-007

Additional relevant MeSH terms:

Arthritis, Juvenile; Still's Disease, Adult-Onset; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Arthritis, Rheumatoid