A Phase 3 Randomized, Double-blind Study to Evaluate the Efficacy and Safety of Apremilast (CC-10004) in Subjects With Active Behçet's Disease
Behçet's Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02307513 ↗Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Apremilast (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Sep 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Area Under the Curve (AUC) for the Number of Oral Ulcers From Baseline Through Week 12 (AUC W0-12) |
222.14; 129.54 | <0.0001 sig |
| SECONDARY Change From Baseline in Oral Ulcer Pain as Measured by Visual Analog Scale (VAS) at Week 12 |
-15.9; -40.7 | <0.0001 sig |
| SECONDARY Change From Baseline in Disease Activity as Measured by Behçet's Syndrome Activity Score (BSAS) at Week 12 |
-5.41; -17.35 | <0.0001 sig |
| SECONDARY Change From Baseline in Disease Activity as Measured by Behçet's Disease Current Activity Form (BDCAF): Behçet's Disease Current Activity Index (BDCAI) at Week 12 |
-0.4; -0.9 | 0.0335 sig |
| SECONDARY Change From Baseline in Disease Activity as Measured by Behçet's Disease Current Activity Form (BDCAF): Patient's Perception of Disease Activity at Week 12 |
-0.7; -1.7 | <0.0001 sig |
| SECONDARY Change From Baseline in Disease Activity as Measured by Behçet's Disease Current Activity Form (BDCAF): Clinician's Overall Perception of Disease Activity at Week 12 |
-0.7; -1.6 | <0.0001 sig |
| SECONDARY Percentage of Participants Who Achieved an Oral Ulcer Complete Response (Oral Ulcer-Free) by Week 6 and Remained Oral Ulcer-Free for at Least 6 Additional Weeks |
4.9; 29.8 | <0.0001 sig |
| SECONDARY Time to Oral Ulcer Resolution (Complete Response) |
8.1; 2.1 | <0.0001 sig |
| SECONDARY Percentage of Participants Who Experienced an Oral Ulcer Complete Response at Week 12 |
22.3; 52.9 | <0.0001 sig |
| SECONDARY Change From Baseline in Behçet's Disease Quality of Life (BD Qol) Scores at Week 12 |
-0.5; -3.5 | 0.0003 sig |
| SECONDARY Percentage of Participants Who Experienced a Complete Response For Genital Ulcers at Week 12 |
41.2; 70.6 | 0.1100 |
| SECONDARY Percentage of Participants With no Oral Ulcers Following a Complete Response |
13.2; 31.3 | 0.0204 sig |
| SECONDARY Time to Recurrence of Oral Ulcers Following Loss of Complete Response |
2.3; 4.6 | 0.0112 sig |
| SECONDARY Number of Oral Ulcers Following Loss of Complete Response Through Week 12 |
1.5; 1.1 | 0.0683 |
| SECONDARY Change From Baseline in the Total Score of the Static Physician's Global Assessment (PGA) of Skin Lesions of BD at Week 12 |
-0.8; -0.9 | 0.5944 |
| SECONDARY Change From Baseline in Genital Ulcer Pain as Measured by VAS Score at Week 12 |
-24.5; -30.0 | 0.6182 |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) During the Placebo-controlled Treatment Period |
74; 82; 37; 60; 6; 6 | — |
| SECONDARY Number of Participants With TEAEs During the Apremilast-Exposure Period |
70; 90; 29; 64; 4; 17 | — |
Summary
Eligibility Criteria
Inclusion Criteria
- Understand and voluntarily sign an informed consent document prior to any study related assessments/procedures being conducted.
- Male and female subjects ≥ 18 years of age at the time of signing the informed consent document.
- Able to adhere to the study visit schedule and other protocol requirements.
- Diagnosed with Behcet's disease meeting th4 International Study Group (ISG) criteria,
- Oral ulcers that occurred at least 3 times in the previous 12-month period, including oral ulcers at the screening visit.
- Subjects must have at least 2 oral ulcers at Visit 1 (Screening Visit), and:
- At least 2 oral ulcers at Visit 2 (day of randomization), when Visit 2 occurs at least 14 days after Visit 1. OR
- At least 3 oral ulcers at Visit 2 (day of randomization), when Visit 2 occurs at any time between 1 day and 42 days after Visit 1.
- Have prior treatment with at least 1 non-biologic Behçet's disease therapy, such as, but not limited to, topical corticosteroids, or systemic treatment.
- Candidate for systemic therapy, for the treatment of oral ulcers.
a. A candidate for systemic therapy is a subject judged by the study Investigator as someone whose mucocutaneous ulcers are considered inappropriate for topical therapy based on the severity of disease and extent of the affected area, or whose oral ulcers cannot be adequately controlled by topical therapy.
- Laboratory Measures: Must meet the following laboratory measures:
- Hemoglobin > 9 g/dL
- White blood cell (WBC) count ≥ 3000 /L(≥ 3.0 X 10^9/L) and ≤ 14,000/L (≤ 14 X 10^9/L )
- Platelet count ≥ 100, 000 /L (≥ 100 X 10^9/L)
- Serum creatinine ≤ 1.5 mg/dL (≤ 132.6 μmol/L)
- Total bilirubin ≤ 2.0 mg/dL
- Aspartate transaminase (AST [serum glutamic oxaloacetic transaminase, SGOT]) and alanine transaminase (ALT [serum glutamate pyruvic transaminase, SGPT]) ≥ 1.5 X ULN. Subjects who fail screening due to ≥ 1.5 X ULN AST/SGOT and/or ALT/SGPT will be allowed to repeat AST/SGOT and/or ALT/SGPT tests within the screening phase. Repeat test results should be ≤ ULN (within reference range) to be eligible.
Laboratory tests will be allowed to be repeated 1 time if, in the Investigator's clinical judgment, there is a reasonable possibility of the repeat tests not meeting the exclusion values, and with concurrence from the Medical Monitor.
Contraception Requirements:
All Females of Child Bearing Potential (FCBP) must use one of the approved contraceptive options as described below while taking apremilast and for at least 28 days after administration of the last dose of the apremilast.
At the time of study entry, and at any time during the study when a FCBP's contraceptive measures or ability to become pregnant changes, the Investigator will educate the subject regarding contraception options and the correct and consistent use of effective contraceptive methods in order to successfully prevent pregnancy.
All FCBP must have a negative pregnancy test at Visits 1 and 2. All FCBP subjects who engage in activity in which conception is possible must use one of the approved contraceptive options described below:
Option 1: Any one of the following highly effective methods: hormonal contraception (oral, injection, implant, transdermal patch, vaginal ring); intrauterine device (IUD); tubal ligation; or partner's vasectomy; OR Option 2: Male or female condom (latex condom or non-latex condom NOT made out of natural [animal] membrane [for example, polyurethane]); PLUS one additional barrier method: (a) diaphragm with spermicide; (b) cervical cap with spermicide; or (c) contraceptive sponge with spermicide.
Male subjects (including those who have had a vasectomy) who engage in activity in which conception is possible must use barrier contraception (latex or non-latex condoms NOT made out of natural [animal] membrane [for example, polyurethane]) while on IP and for at least 28 days after the last dose of IP.
Exclusion Criteria: The presence of any of the followi
Data sourced from ClinicalTrials.gov (NCT02307513). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.