Rofecoxib Efficacy and Safety Evaluation Trial in Hemophilic Arthropathy

Study Overview
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Brief Summary
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This is a multicenter, randomized, double-blind study to evaluate the efficacy and safety of TRM-201 (rofecoxib) versus Placebo in the treatment of patients with hemophilic arthropathy (HA) over a 12-week period (Part I) that is followed by a year-long (52 week) open-label extension (Part II) to further evaluate the safety and maintenance of efficacy of TRM-201.

Official Title
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A Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Phase III Study With a Long-Term, Open-Label Extension to Evaluate the Efficacy and Safety of TRM-201 (Rofecoxib) in Patients With Hemophilic Arthropathy

Conditions
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Hemophilic Arthropathy

Intervention / Treatment
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* Drug: TRM-201 (Rofecoxib)
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of hemophilia A or B Either on a stable prophylaxis regimen for their bleeding disorder (factor, bypassing agent, or nonfactor product therapy) OR currently taking or agree to initiate a gastroprotective agent (esomeprazole) for the duration of the trial Diagnosis of Hemophilic Arthopathy for at least 6 months prior to screening Chronic symptomatic pain in one or more joint(s) on 20 of the 30 days prior to screening. Able and willing to wash out of non-study analgesic medications/agents for at least 7 days prior to Randomization including: acetaminophen (paracetamol), NSAIDs, opioids, cannabinoids, topical analgesics, benzodiazepines, gabapentin/pregabalin-containing products and other antiepileptic drugs used for pain Primary source of pain is due to Hemophilic Arthropathy Exclusion Criteria: Taking opioids for greater than 4 days per week prior to screening Has a history of advanced renal disease or severe liver disease (within the last 6 months) Receiving emicizumab while also receiving activated prothrombin complex concentrate (FEIBA) Uncontrolled or poorly controlled hypertension History of major cardiac or cerebrovascular disease History of an upper GI perforation, obstruction, or major GI bleed or current evidence of GI bleeding Has active hepatitis C or hepatitis B infection or uncontrolled HIV. Note: Patients who are HIV positive are allowed to participate if considered to be controlled. Has a positive drug screen for all prohibited drugs of potential abuse at screening Has had an intra-articular injection (within 3 months), initiated physical therapy (within 30 days) or has had orthopedic surgery (within 4 months) prior to screening

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: TRM-201 (Rofecoxib)<br>1 TRM-201 tablet taken orally once daily for 12 weeks in Part I and orally once daily for an additional 52 weeks in Part II | Drug: TRM-201 (Rofecoxib)<br>* Eligible patients will be randomized to receive TRM-201 or placebo<br>* Other names: Placebo;|
| Placebo Comparator: Placebo<br>1 placebo tablet (to match TRM-201) taken orally once daily for 12 weeks in Part I and then 1 TRM-201 tablet taken orally once daily for an additional 52 weeks in Part II | Drug: Placebo<br>* Matching Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The change from baseline in the patient assessment of the daily Average Hemophilic arthropathy Pain Intensity score on a 0- to 10-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine. |  | 12 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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HA, Pain, Hemophilia, Joint Pain