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NCT Number: NCT02994147

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

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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Key information

Conditions

Hemophilic Arthropathy

Age range

20 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Tri-Service General Hospital

Taipei, Taiwan

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male age 20 to 65 years, inclusive
  • Diagnosis of Hemophilia A or B
  • Clinical diagnosis of hemophilic arthropathy in the knee(s) for at least 6 months
  • Pettersson score (based on X-ray) of 0 to 4 in at least one knee within 1 year of screening
  • IPSG (2012) overall score of 3 to 9 (based on MRI) in at least one knee at screening
  • Is able to read, understand, and sign the Informed Consent Form (ICF), communicate with the investigator, complete study diaries, and understand and comply with protocol requirements

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:
  • NSAID injection (oral NSAIDs including COX-2 inhibitors allowed)
  • Glucosamine or chondroitin
  • 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

Treatment and study plan

Placebo

Drug

Placebo-only control study medication is the same formulation as active treatment without active ingredient

AC-201CR

Drug

The investigational product is formulated as controlled-release (CR) tablets

Primary outcomes

  1. 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).

Secondary outcomes

  1. Change from baseline in IPSG (MRI) score for non-primary knee

    Time frame: 24 Weeks

  2. Change from baseline in IPSG score by knee

    Time frame: 48 Weeks

  3. Change from baseline in IPSG component scores by knee

    Time frame: 24 Weeks and 48 Weeks

  4. Change from baseline in MRI-measured synovial thickness by knee

    Time frame: 24 Weeks and 48 Weeks

  5. Change from baseline in ultrasonographic synovial thickness by knee

    Time frame: 24 Weeks and 48 Weeks

  6. Change from baseline in ultrasonographic hyperemia score by knee

    Time frame: 24 Weeks and 48 Weeks

  7. Change from baseline in knee pain (by VAS) by knee

    Time frame: 4, 12, 24, 28, 36 and 48 Weeks

  8. Change from baseline in Short Form-36 score

    Time frame: 24 Weeks and 48 Weeks

Sponsors and collaborators

Lead sponsor

TWi Biotechnology, Inc.

Industry

Registry information

Important dates

Study start
2017
Primary completion
2019
Study completion
2019
First posted
Dec 15, 2016
Registry last updated
Jul 9, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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