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Repeated joint bleeding leads to hemophilic arthropathy (HA), which manifests with chronic synovitis, cartilage damage and bony destruction. Currently available treatments of HA, including analgesics, NSAIDs, and hyaluronic acid, are predominantly directed toward the symptomatic relief of pain and inflammation, with no or little effect on joint cartilage degeneration. AC201CR, a control released formulation of AC-201, demonstrates anti-inflammatory effects by reducing NLRP3 inflammasome assembly. AC-201 is also unique in that it influences both the anabolism and catabolism of chondrocytes in vitro and has shown cartilage-sparing properties in animal studies. The study is designed to evaluate the joint structure-modifying and symptom-relieving effects, safety, and tolerability of AC-201CR in subjects with HA.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator |
|
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| AC-201CR 72mg | Experimental |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo | Drug | Placebo-only control study medication is the same formulation as active treatment without active ingredient |
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| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in IPSG (MRI) score for primary knee | Knee arthropathy will be evaluated according to the 2012 IPSG (International Prophylaxis Study Group) rating scale. This scale is an additive scale with maximum value up to 17 points, comprising 2 subscores of soft tissue changes (maximum 9 points) and osteochondral changes (maximum 8 points). | 24 Weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in IPSG (MRI) score for non-primary knee | 24 Weeks | |
| Change from baseline in IPSG score by knee | 48 Weeks | |
| Change from baseline in IPSG component scores by knee |
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Inclusion Criteria:
Exclusion Criteria:
Total knee replacement in the primary knee
Presence of joint infections in the primary knee
Knee surgery within 6 months prior to screening in the primary knee
Intra-articular hyaluronic acid (IAHA) or platelet-rich plasma (PRP) injection in the primary knee within 6 months prior to screening
Use of any of the following medications after the screening visit:
History of high responder inhibitory antibodies to factor VIII or factor IX clotting factor, with titer >5 Bethesda units (BU), OR clinical evidence for presence of factor inhibitor
History of rheumatoid arthritis or gouty arthropathy
Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) concentration >2x the upper limit of laboratory normal range (ULN), or bilirubin >1.5x ULN at screening
Positive test for human immunodeficiency virus (HIV), hepatitis B virus (HBV), or TB
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Tri-Service General Hospital | Taipei | Taiwan |
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| AC-201CR | Drug | The investigational product is formulated as controlled-release (CR) tablets |
|
| 24 Weeks and 48 Weeks |
| Change from baseline in MRI-measured synovial thickness by knee | 24 Weeks and 48 Weeks |
| Change from baseline in ultrasonographic synovial thickness by knee | 24 Weeks and 48 Weeks |
| Change from baseline in ultrasonographic hyperemia score by knee | 24 Weeks and 48 Weeks |
| Change from baseline in knee pain (by VAS) by knee | 4, 12, 24, 28, 36 and 48 Weeks |
| Change from baseline in Short Form-36 score | 24 Weeks and 48 Weeks |