Rofecoxib Efficacy and Safety Evaluation Trial in Hemophilic Arthropathy

NCT04684511 | Terminated Phase 3 DMC FDA Drug

• 1 other identifier: TRM-201-HA-301
• Study Type: interventional
• Target: 2
• Locations: 8 countries
• Sites: 46

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.

Conditions

Hemophilic Arthropathy

Keywords

Hemophilia; Pain; HA; Joint Pain

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (2)

TRM-201 (Rofecoxib)

EXPERIMENTAL

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)

Placebo

PLACEBO COMPARATOR

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

Interventions

TRM-201 (Rofecoxib) DRUG

Eligible patients will be randomized to receive TRM-201 or placebo

Also known as: Placebo

TRM-201 (Rofecoxib)

Placebo DRUG

Matching Placebo

Placebo

Eligibility Criteria

• Age: 12 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Tremeau Pharmceuticals, Inc.: lead

Study Sites (46)

Orthopedic Hospital DBA Orthopedic Hemophilia Treatment Center

Los Angeles, California, 90007, United States

Location

Center for Inherited Blood Disorders

Orange, California, 92868, United States

Location

UC Davis Children's Hospital

Sacramento, California, 95817, United States

Location

University of Colorado Hemophilia & Thrombosis Center

Aurora, Colorado, 80045, United States

Location

Georgetown University Hospital - Medstar

Washington D.C., District of Columbia, 20007, United States

Location

University of Florida - Shands

Gainesville, Florida, 32610, United States

Location

Clinical Trial Services, Corp

Miami, Florida, 33144, United States

Location

Anchor Medical Research, LLC

Miami, Florida, 33176, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

University of Iowa Hospitals & Clinics

Iowa City, Iowa, 52242, United States

Location

Louisiana Center for Bleeding and Clotting Disorders

New Orleans, Louisiana, 70112, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

ECMC Hospital

Buffalo, New York, 14202, United States

Location

Icahn School of Medicine at Mount Sinai.

New York, New York, 10029, United States

Location

East Carolina University

Greenville, North Carolina, 27858, United States

Location

Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

North Texas Comprehensive Hemophilia Center

Dallas, Texas, 75390, United States

Location

Gulf States Hemophilia and Thrombophilia Center

Houston, Texas, 77030, United States

Location

Calvary Mater Newcastle

Waratah, New South Wales, Australia

Location

Fiona Stanley Hospital

Murdoch, Western Australia, Australia

Location

The Alfred Hospital

Melbourne, 3004, Australia

Location

Health Sciences Center

St. John's, Newfoundland and Labrador, NL A1B3V6, Canada

Location

Hamilton Health Sciences Centre

Hamilton, Ontario, Canada

Location

Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi

Bologna, Italy

Location

Ospedale Pediatrico Bambino Gesù

Roma, Italy

Location

Centrum Medyczne Pratia Poznan

Poznan, Poland

Location

Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu

Wroclaw, Poland

Location

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, 33011, Spain

Location

Acibadem Adana Hospital

Adana, Turkey (Türkiye)

Location

Akdeniz University Medical Faculty

Antalya, Turkey (Türkiye)

Location

Erciyes University Medical Faculty

Edirne, Turkey (Türkiye)

Location

Gaziantep University Medical Faculty Sahinbey Educational Research Hospital

Gaziantep, 27310, Turkey (Türkiye)

Location

Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty

Istanbul, Turkey (Türkiye)

Location

Ege University Medical Faculty

Izmir, 35100, Turkey (Türkiye)

Location

Ege University Medical Faculty

Izmir, Turkey (Türkiye)

Location

Ondokuz Mayis Univ. Med. Fac.

Samsun, Turkey (Türkiye)

Location

CI Dnipropetrovsk CMCH #4 of Dnipropetrovsk RC

Dnipro, Ukraine

Location

CI of Healthcare Kharkiv City Clin Children's Hosp #16 Kharkiv NMU

Kharkiv, Ukraine

Location

Communal Institution of Kyiv Regional Council Kyiv Regional Clinical Hospital

Kyiv, Ukraine

Location

Kyiv CCH #9 City SPC of Diagnostics & Treatment of Patients with HP

Kyiv, Ukraine

Location

Nat.Children Specialized Hosp.OKHMATDYT of the Ministry of Health of Ukraine

Kyiv, Ukraine

Location

SI Institute of Blood Pathology and Transfusion Medicine of NAMSU

Lviv, Ukraine

Location

M.V. Sklifosovskyi Poltava RCH Dept of Pulmonology HSEIU Ukrainian Medical Stomatological Academy

Poltava, Ukraine

Location

CI of TRC Ternopil UH

Ternopil, Ukraine

Location

CI Zaporizhzhya Regional Clinical Hospital of ZRC

Zaporizhzhya, Ukraine

Location

MeSH Terms

Conditions

Hemophilia A; Pain; Arthralgia

Interventions

rofecoxib

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Joint Diseases; Musculoskeletal Diseases

Study Officials

• Judith Boice, PhD

  Tremeau Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2020
• First Posted: December 24, 2020
• Study Start: June 2, 2021
• Primary Completion: September 28, 2022
• Study Completion: September 28, 2022
• Last Updated: October 27, 2022

Record last verified: 2022-08

Locations

IT (2); PL (2); ES (1); CA (2); AU (3); US (19); UA (9); TU (8)