Intra-Articular Bevacizumab for Preventing Recurrent Hemarthrosis in Hemophilia With Chronic Synovitis

NCT ID: NCT07187661

Last Updated: 2025-09-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-09-09

Study Completion Date

2026-04-15

Brief Summary

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Hemophilia is an inherited bleeding disorder characterized by deficiency of clotting factors, leading to increased bleeding tendencies. The most common complications are joint bleeds (hemarthroses), which cause chronic changes in joints and ultimately disability. Recurrent hemarthroses often result from chronic synovitis in target joints of patients with hemophilia, a process driven by Vascular Endothelial Growth Factor (VEGF) mediated pathological angiogenesis. Intra-articular administration of Bevacizumab, a VEGF neutralizing monoclonal antibody, may block this process and reduce the frequency of recurrent joint bleeds. This study evaluates the efficacy and safety of intra-articular Bevacizumab for preventing recurrent hemarthrosis in patients with hemophilia and chronic synovitis.

Detailed Description

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Hemophilia A is the most prevalent inherited bleeding disorder in Pakistan, accounting for approximately 69% of all hemophilia cases. A major complication is recurrent joint bleeding (hemarthrosis), which leads to chronic synovitis and progressive hemophilic arthropathy. This joint deterioration significantly impairs mobility and quality of life.

The underlying pathophysiology is driven by repeated hemarthroses, which cause increased local expression of Vascular Endothelial Growth Factor (VEGF). VEGF promotes abnormal angiogenesis and synovial proliferation, perpetuating a cycle of inflammation and bleeding, even when symptoms are not overt.

Bevacizumab is a recombinant humanized monoclonal antibody that specifically inhibits VEGF. Local intra-articular administration offers a targeted therapeutic approach to disrupt this pathogenic cycle directly at the site of pathology. By neutralizing VEGF, Bevacizumab may mitigate synovitis, reduce bleeding frequency, and slow progression of hemophilic arthropathy, while minimizing systemic exposure and associated adverse effects.

To investigate this therapeutic strategy, a two-year, open-label, single-arm clinical trial will be conducted at the Institute of Pathology and Diagnostic Medicine (IPDM), Khyber Medical University (KMU), and Hayatabad Medical Complex, Peshawar, Pakistan. A total of 25 participants with Hemophilia A and chronic synovitis in one or more target joints (knee, elbow, or ankle) will be enrolled.

All participants will receive intra-articular Bevacizumab injections following prophylactic factor replacement therapy to prevent procedure-related bleeding. The first four participants will receive 20 mg/0.8 mL per injection. If well tolerated without major toxicities, the remaining participants will receive 40 mg/1.6 mL per injection. Each injection will be administered into the target joint once every 28 days for a total of four doses.

The primary efficacy outcome will be the change in the Annualized Bleeding Rate (ABR) of the target joint, calculated from 3-month pre- and post-treatment data. Secondary outcomes will include changes in joint health assessed using the Hemophilia Joint Health Score (HJHS, Version 2.1; range 0-124, where higher scores indicate worse joint health) and synovial inflammation measured by Magnetic Resonance Imaging (MRI) with the International Prophylaxis Study Group (IPSG)-compatible scoring system.

This study may provide critical insight into a novel, VEGF-targeted strategy for managing chronic synovitis in hemophilia patients and preserving long-term joint health.

Conditions

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Hemophilia Chronic Synovitis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

This is an interventional clinical trial utilizing a pre- and post-study design. It is a single-arm study, meaning all enrolled participants will receive the investigational intervention. The trial is open-label, with no masking (blinding) of participants, care providers, investigators, or outcomes assessors. As it is a single-arm study, there is no allocation or randomization process. The total enrollment for this study is 25 participants.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

This is an open-label study. No masking is used.

Study Groups

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Intra-articular Bevacizumab

All participants will receive intra-articular injections of Bevacizumab. The initial dose for the first four patients will be 20 mg/0.8 ml. If no major toxicities are observed, the dose will be escalated to 40 mg/1.6 ml for the remaining patients. Injections will be administered monthly for a duration of 4 months.

Group Type EXPERIMENTAL

Intra-articular Bevacizumab

Intervention Type DRUG

This clinical trial investigates the intra-articular injection of Bevacizumab, a recombinant humanized monoclonal antibody that inhibits Vascular Endothelial Growth Factor (VEGF). The intervention functions by binding to and neutralizing VEGF-A, thereby blocking the pathogenic angiogenesis and vascular permeability that characterizes chronic hemophilic synovitis. For administration, the first four patients will receive a dose of 20 mg in 0.8 mL per injection, and if this is well-tolerated without major toxicities, the dose for all subsequent patients will be increased to 40 mg in 1.6 mL. Each injection will be administered directly into the target joint (knee, elbow, or ankle) once every 28 days for a total of four doses. Crucially, all injections will be performed only after appropriate prophylactic factor replacement to mitigate any procedure-related bleeding risk.

Interventions

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Intra-articular Bevacizumab

This clinical trial investigates the intra-articular injection of Bevacizumab, a recombinant humanized monoclonal antibody that inhibits Vascular Endothelial Growth Factor (VEGF). The intervention functions by binding to and neutralizing VEGF-A, thereby blocking the pathogenic angiogenesis and vascular permeability that characterizes chronic hemophilic synovitis. For administration, the first four patients will receive a dose of 20 mg in 0.8 mL per injection, and if this is well-tolerated without major toxicities, the dose for all subsequent patients will be increased to 40 mg in 1.6 mL. Each injection will be administered directly into the target joint (knee, elbow, or ankle) once every 28 days for a total of four doses. Crucially, all injections will be performed only after appropriate prophylactic factor replacement to mitigate any procedure-related bleeding risk.

Intervention Type DRUG

Other Intervention Names

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Avastin Genentech

Eligibility Criteria

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Inclusion Criteria

* Confirmed diagnosis of Hemophilia A.
* Presence of one or more target joints (knee, elbow, ankle) with chronic synovitis and a history of \>2 hemarthrosis episodes in the past 6 months.
* Target joint World Federation of Hemophilia (WFH) joint score of 2-3.
* Adequate hematological, renal, and liver function (as specified by protocol lab values).
* Ability and willingness to provide informed consent and comply with the study protocol.

Exclusion Criteria

* HIV positive diagnosis.
* Severely damaged joints or anatomical limitations preventing safe injection.
* Contraindications to MRI.
* Uncontrolled hypertension.
* Recent major surgery/trauma (\<28 days).
* Serious non-healing wound, active cardiovascular disease, or other significant comorbidities that could increase risk or interfere with the study.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Khyber Medical University Peshawar

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Dr Kinza Ayaz, MBBS

Role: PRINCIPAL_INVESTIGATOR

Khyber Medical University Peshawar

Prof. Dr. Yasar M Yousafzai, PhD

Role: PRINCIPAL_INVESTIGATOR

Khyber Medical University Peshawar

Dr. Muhammad Tariq Masood Khan, MBBS

Role: PRINCIPAL_INVESTIGATOR

Khyber Medical University Peshawar

Dr Khalid Khan, MBBS

Role: PRINCIPAL_INVESTIGATOR

Khyber Medical University Peshawar

Locations

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Hayatabad Medical Complex

Peshawar, KPK, Pakistan

Site Status RECRUITING

Institute of Pathology and Diagnostic Medicine (IPDM)

Peshawar, KPK, Pakistan

Site Status RECRUITING

Countries

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Pakistan

Central Contacts

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Dr Kinza Ayaz, MBBS

Role: CONTACT

+92 320 9124645

Prof. Dr. Yasar M Yousafzai, PhD

Role: CONTACT

+92 321 9054010

Facility Contacts

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Dr Khalid Khan, MBBS

Role: primary

Dr Kinza Ayaz, MBBS

Role: backup

Dr Kinza Ayaz

Role: primary

+92 320 9124645

Prof. Dr. Yasar M Yousafzai

Role: backup

+92 321 9054010

References

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Sadiq MA, Ahmed S, Afzal M, Tasfeen S. A basis for prenatal diagnosis of Haemophilia-A in Pakistani patients. Pak J Med Sci. 2022 Nov-Dec;38(8):2065-2070. doi: 10.12669/pjms.38.8.6524.

Reference Type BACKGROUND
PMID: 36415265 (View on PubMed)

Hirayama AB, Silva AKCD, Rocha JS, Roberti MDRF. Prevalence of symptoms in hemophilia carriers in comparison with the general population: a systematic review. Hematol Transfus Cell Ther. 2019 Oct-Dec;41(4):349-355. doi: 10.1016/j.htct.2019.02.006. Epub 2019 Jun 17.

Reference Type BACKGROUND
PMID: 31412987 (View on PubMed)

Gualtierotti R, Solimeno LP, Peyvandi F. Hemophilic arthropathy: Current knowledge and future perspectives. J Thromb Haemost. 2021 Sep;19(9):2112-2121. doi: 10.1111/jth.15444. Epub 2021 Jul 27.

Reference Type BACKGROUND
PMID: 34197690 (View on PubMed)

Hassan S, Monahan RC, Mauser-Bunschoten EP, van Vulpen LFD, Eikenboom J, Beckers EAM, Hooimeijer L, Ypma PF, Nieuwenhuizen L, Coppens M, Schols SEM, Leebeek FWG, Smit C, Driessens MH, le Cessie S, van Balen EC, Rosendaal FR, van der Bom JG, Gouw SC. Mortality, life expectancy, and causes of death of persons with hemophilia in the Netherlands 2001-2018. J Thromb Haemost. 2021 Mar;19(3):645-653. doi: 10.1111/jth.15182. Epub 2020 Dec 18.

Reference Type BACKGROUND
PMID: 33217158 (View on PubMed)

Iorio A, Stonebraker JS, Chambost H, Makris M, Coffin D, Herr C, Germini F; Data and Demographics Committee of the World Federation of Hemophilia. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries. Ann Intern Med. 2019 Oct 15;171(8):540-546. doi: 10.7326/M19-1208. Epub 2019 Sep 10.

Reference Type BACKGROUND
PMID: 31499529 (View on PubMed)

Other Identifiers

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KMU/DIR/CTU/2025/006

Identifier Type: -

Identifier Source: org_study_id

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