Clinical Trial: A Proof-of-Concept Study of AC-201 Controlled-Release Tablet (CR Tablet) in Patients With Hemophilic Arthropathy

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Proof-of-Concept Study of AC-201 Controlled-Release Tablet (CR Tablet) in Patients With Hemophilic Arthropathy

Brief Summary: 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.

Detailed Summary:
Sponsor: TWi Biotechnology, Inc.

Current Primary Outcome: Change from baseline in IPSG (MRI) score for primary knee [ Time Frame: 24 Weeks ]

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).


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change from baseline in IPSG (MRI) score for non-primary knee [ Time Frame: 24 Weeks ]
  • Change from baseline in IPSG score by knee [ Time Frame: 48 Weeks ]
  • Change from baseline in IPSG component scores by knee [ Time Frame: 24 Weeks and 48 Weeks ]
  • Change from baseline in MRI-measured synovial thickness by knee [ Time Frame: 24 Weeks and 48 Weeks ]
  • Change from baseline in ultrasonographic synovial thickness by knee [ Time Frame: 24 Weeks and 48 Weeks ]
  • Change from baseline in ultrasonographic hyperemia score by knee [ Time Frame: 24 Weeks and 48 Weeks ]
  • Change from baseline in knee pain (by VAS) by knee [ Time Frame: 4, 12, 24, 28, 36 and 48 Weeks ]
  • Change from baseline in Short Form-36 score [ Time Frame: 24 Weeks and 48 Weeks ]


Original Secondary Outcome: Same as current

Information By: TWi Biotechnology, Inc.

Dates:
Date Received: December 9, 2016
Date Started: August 2016
Date Completion: December 2018
Last Updated: December 13, 2016
Last Verified: December 2016