Clinical trial NCT04684511: Rofecoxib Efficacy and Safety Evaluation Trial in Hemophilic Arthropathy
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Hemophilic Arthropathy
Keywords: Hemophilia
Pain
HA
Joint Pain
Lead sponsor: Tremeau Pharmceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2020-12-24
Enrollment: 2
Sex: ALL
Minimum age (years): 12
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: 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.
timeFrame: 12 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Orthopedic Hospital DBA Orthopedic Hemophilia Treatment Center
state: California
city: Orange
country: United States
facility: Center for Inherited Blood Disorders
state: California
city: Sacramento
country: United States
facility: UC Davis Children's Hospital
state: California
city: Aurora
country: United States
facility: University of Colorado Hemophilia & Thrombosis Center
state: Colorado
city: Washington
country: United States
facility: Georgetown University Hospital - Medstar
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida - Shands
state: Florida
city: Miami
country: United States
facility: Clinical Trial Services, Corp
state: Florida
city: Miami
country: United States
facility: Anchor Medical Research, LLC
state: Florida
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa Hospitals & Clinics
state: Iowa
city: New Orleans
country: United States
facility: Louisiana Center for Bleeding and Clotting Disorders
state: Louisiana
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Buffalo
country: United States
facility: ECMC Hospital
state: New York
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai.
state: New York
city: Greenville
country: United States
facility: East Carolina University
state: North Carolina
city: Cleveland
country: United States
facility: Rainbow Babies and Children's Hospital
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Dallas
country: United States
facility: North Texas Comprehensive Hemophilia Center
state: Texas
city: Houston
country: United States
facility: Gulf States Hemophilia and Thrombophilia Center
state: Texas
city: Waratah
country: Australia
facility: Calvary Mater Newcastle
state: New South Wales
city: Murdoch
country: Australia
facility: Fiona Stanley Hospital
state: Western Australia
city: Melbourne
country: Australia
facility: The Alfred Hospital
city: Saint John's
country: Canada
facility: Health Sciences Center
state: Newfoundland and Labrador
city: Hamilton
country: Canada
facility: Hamilton Health Sciences Centre
state: Ontario
city: Bologna
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesù
city: Poznań
country: Poland
facility: Centrum Medyczne Pratia Poznan
city: Wrocław
country: Poland
facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu
city: Oviedo
country: Spain
facility: Hospital Universitario Central de Asturias
state: Asturias
city: Adana
country: Turkey
facility: Acibadem Adana Hospital
city: Antalya
country: Turkey
facility: Akdeniz University Medical Faculty
city: Edirne
country: Turkey
facility: Erciyes University Medical Faculty
city: Gaziantep
country: Turkey
facility: Gaziantep University Medical Faculty Sahinbey Educational Research Hospital
city: Istanbul
country: Turkey
facility: Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty
city: İzmir
country: Turkey
facility: Ege University Medical Faculty
city: İzmir
country: Turkey
facility: Ege University Medical Faculty
city: Samsun
country: Turkey
facility: Ondokuz Mayis Univ. Med. Fac.
city: Dnipro
country: Ukraine
facility: CI Dnipropetrovsk CMCH #4 of Dnipropetrovsk RC
city: Kharkiv
country: Ukraine
facility: CI of Healthcare Kharkiv City Clin Children's Hosp #16 Kharkiv NMU
city: Kyiv
country: Ukraine
facility: Communal Institution of Kyiv Regional Council Kyiv Regional Clinical Hospital
city: Kyiv
country: Ukraine
facility: Kyiv CCH #9 City SPC of Diagnostics & Treatment of Patients with HP
city: Kyiv
country: Ukraine
facility: Nat.Children Specialized Hosp.OKHMATDYT of the Ministry of Health of Ukraine
city: Lviv
country: Ukraine
facility: SI Institute of Blood Pathology and Transfusion Medicine of NAMSU
city: Poltava
country: Ukraine
facility: M.V. Sklifosovskyi Poltava RCH Dept of Pulmonology HSEIU Ukrainian Medical Stomatological Academy
city: Ternopil'
country: Ukraine
facility: CI of TRC Ternopil UH
city: Zaporizhzhya
country: Ukraine
facility: CI Zaporizhzhya Regional Clinical Hospital of ZRC
Study officials:
affiliation: Tremeau Pharmaceuticals
name: Judith Boice, PhD
role: STUDY_DIRECTOR
References: