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Combination Bevacizumab and PRGN-2012 in Adults With Recurrent Respiratory Papillomatosis (RRP)

3. listopadu 2026 aktualizováno: National Cancer Institute (NCI)

A Phase II Study of Combination Bevacizumab and PRGN-2012 in Adults With Recurrent Respiratory Papillomatosis (RRP)

Background:

Recurrent respiratory papillomatosis (RRP) is a rare disease that causes wart-like growths called papillomas to grow in the airway, most often in the voice box, windpipe, or lungs. These growths can make it hard to speak or breathe. Surgery can remove the papillomas, but they often come back. In some cases, they can become cancerous.

Objective:

This study istesting whether two treatments used together (PRGN-2012, a vaccine-based treatment and bevacizumab, a drug that affects blood vessel growth) can help control RRP and reduce the chance that papillomas will grow back.

Eligibility:

Adults aged 18 years and older may be able to join the study if they have RRP and meet certain treatment history requirements. This may include people who have previously received PRGN-2012 or bevacizumab, or people who have needed more than 2 surgeries to remove papillomas.

Design:

Before starting treatment, participants will have screening tests to make sure the study is safe for them. These tests may include a physical exam with blood and urine tests, heart function testing, imaging scans, and an endoscopy. During an endoscopy, a thin, flexible tube with a small camera will look at the inside of the nose, throat, voice box, and upper windpipe.

Participants will receive study treatment during 7 clinic visits over about 6 months. Bevacizumab is given through a vein amd PRGEN-2012 is given as an injection under the skin of the arm or leg. Participants may receive 1 or both drugs at each visit.

After completing treatmen, participants will return for 4 follow-up visits over 1 year. These visits may include repeat imaging, blood and urine tests, and other exams. After that, the study team will contact participantsby phone or email every 3 months for 2 years.

If their RRP gets worse during the follow-up period, they may be able to receive a second course of treatment using the same schedule....

Přehled studie

Detailní popis

Background:

  • Recurrent respiratory papillomatosis (RRP) is a rare papillomatous disease of the respiratory tract caused by Human Papilloma Virus (HPV) types 6 or 11.
  • RRP can progress to cause severe voice disturbance, airway compromise, fatal pulmonary lesions, and, rarely, invasive cancers.
  • Our group conducted the pivotal phase 1/2 clinical study of PRGN-2012 (NCT04724980), a gorilla adenovirus vector-based gene therapy capable of eliciting HPV6/11-specific T cell immunity.
  • The study was found to be positive, and a Biologics License Application (BLA) submission for this agent was accepted by the US Food and Drug Administration (FDA). On August 15th, 2025, the FDA granted full approval of PRGN-2012, making it the first FDA-approved medical therapy for adult patients with RRP. Unfortunately, 50% of study participants did not achieve a complete response in the pivotal study, indicating that additional research into therapeutic strategies is still needed.
  • We are currently conducting a phase 2 clinical trial of bevacizumab (NCT00803062) to assess the safety and clinical benefit of this agent in adults with RRP.
  • Bevacizumab has demonstrated an acceptable safety profile with consistent and rapid clinical benefit by significantly reducing papillomatous disease burden and eliminating the need for clinical interventions in 20 consecutively treated patients. Unfortunately, disease regrowth occurs following cessation of bevacizumab therapy, and prolonged treatment can lead to systemic toxicity.
  • Community physicians will soon be using both PRGN-2012 and systemic bevacizumab in clinical practice, but the combined safety and appropriate sequencing of these agents has yet to be studied.

Objective:

-To determine the complete response rate, defined as the percentage of participants who do not require an intervention (either medical or surgical) in the 12 months following completion of treatment with combination systemic bevacizumab and PRGN-2012

Eligibility:

  • Histologically or cytologically confirmed diagnosis of RRP
  • Age >= 18 years old
  • A history of 2 or more surgeries or use of IV bevacizumab in the last 12 months to control laryngeal and/or tracheal RRP
  • Previous treatment with PRGN-2012 (zopapogene imadenovec [Papzimeos])

Design:

  • This is phase II, single-arm clinical trial evaluating the combination of systemic bevacizumab and PRGN-2012.
  • All participants will receive monotherapy with bevacizumab alone for every 3 weeks for 3 doses followed by bevacizumab in combination with PRGN-2012 (2 doses) and PRGN- 2012 alone (2 doses).
  • Participants will be assessed for papilloma recurrence at 6, 12, 24, 52 weeks, and every 3 months for an additional 2 years following completion of treatment (total follow-up of 3 years).
  • A total of 21 evaluable participants will be treated in the study.

Typ studie

Intervenční

Zápis (Odhadovaný)

50

Fáze

  • Fáze 2

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní kontakt

Studijní záloha kontaktů

Studijní místa

    • Maryland
      • Bethesda, Maryland, Spojené státy, 20892
        • National Institutes of Health Clinical Center
        • Kontakt:
          • NIH Clinical Center Office of Patient Recruitment (OPR)
          • Telefonní číslo: TTY dial 711 800-411-1222
          • E-mail: ccopr@nih.gov

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

18 let až 120 let (Dospělý, Starší dospělý)

Přijímá zdravé dobrovolníky

Ne

Pohlaví způsobilá ke studiu

Všechno

Popis

  • INCLUSION CRITERIA:

  • Histological or cytological diagnosis of RRP confirmed by pathology report. Note: If there is no documentation or archival sample, a biopsy will be done to confirm the diagnosis.

  • Age >= 18 years old.

  • A history of 2 or more surgeries or use of IV bevacizumab in order to control laryngeal and/or tracheal RRP within 12 months prior to the study treatment initiation.

  • Previous treatment with PRGN-2012 (zopapogene imadenovec [Papzimeos]).

  • Eastern Cooperative Oncology Group (ECOG) performance status of 0-1.

  • Participants must have an adequate organ and marrow function as defined below:

    • White blood cells (WBC) >2,000/mcL
    • Absolute neutrophil count (ANC) >= 1,000/mcL
    • Hemoglobin > 9.0 g/dL
    • Platelets >= 100,000/mcL
    • Total bilirubin <= 1.5 mg/dL. Note: participants with Gilbert s Syndrome must have a total bilirubin < 3.0 mg/dL
    • Aspartate aminotransferase (AST) <= 2.5 X institutional upper limit of normal (ULN)
    • Alanine aminotransferase (ALT) <=2.5 X institutional ULN
    • Creatinine within normal institutional limits OR Creatinine Clearance (CrCl) >= 60 mL/min/1.73 m^2 for participants with creatinine levels above institutional normal (calculated using the Cockcroft-Gault formula).
    • Prothrombin time (PT) / International normalized ratio (INR) and Partial thromboplastin time (PTT) <= 1 X institutional ULN. In participants on anticoagulation, coagulation tests should be within a therapeutic range.
    • Urinalysis Urine dipstick < 2+ proteinuria. Participants with >= 2+ proteinuria on dipstick urinalysis should undergo a 24- hour urine collection and must demonstrate <= 1g of protein in 24 hours to be eligible
  • Women of child-bearing potential (WOCBP) must agree to use a highly effective method of contraception (hormonal, intrauterine device (IUD), surgical sterilization, abstinence) for the duration of treatment and up to 6 months after completion of the study treatment.

Men must agree to use an effective method of contraception (barrier, surgical sterilization, abstinence) for the duration of treatment and up to 4 months after the last dose of study drugs. We also recommend men with partners of childbearing potential ask their partners to be on highly effective birth control (hormonal, IUD, surgical sterilization). Men must not freeze or donate sperm within the same period.

  • Women who are breastfeeding or plan to breastfeed must agree to discontinue breastfeeding from study treatment initiation.
  • Ability of participant to understand and sign a written informed consent document.

EXCLUSION CRITERIA:

  • History of significant cardiovascular disease or thromboembolic event: cerebral vascular accident/stroke, myocardial infarction, unstable angina, congestive heart failure (>= New York Heart Association Classification Class II) occurring within 12 months prior to the study treatment initiation
  • Serious cardiac arrhythmia requiring medication as assessed by electrocardiogram (EKG) at screening.
  • Any investigational agents within 4 weeks prior to the study treatment initiation.
  • Systemic medical RRP therapy within 4 weeks or 3 half-lives, whichever is longer prior to the study treatment initiation.
  • Participants with a condition requiring systemic treatment with either corticosteroids (> 10 mg daily prednisone equivalents) or other immunosuppressive medications within 14 days prior to the study treatment initiation. Note: Inhaled, topical intranasal or intraocular steroids, and adrenal replacement doses <10 mg daily prednisone equivalents are permitted in the absence of active autoimmune disease.
  • History of abdominal fistula or gastrointestinal perforation within 12 months prior to the study treatment initiation.
  • Major surgery within 4 weeks prior to the study treatment initiation. Note: The surgery is considered major if a mesenchymal barrier is opened (pleural cavity, peritoneum, meninges).
  • Non-healing wounds, active ulcer, or untreated bone fracture.
  • History of hemoptysis (>2.5 mL of bright red blood per episode) within 1 month prior to the study treatment initiation.
  • History of serious hemorrhage (CTCAE Grade 4) within 12 months prior to the study treatment initiation.
  • Evidence of bleeding diathesis or significant coagulopathy (with or without current therapeutic anticoagulation).
  • Significant vascular disease (e.g., aortic aneurysm requiring surgical repair or recent peripheral arterial thrombosis) within 6 months prior to the study treatment initiation.
  • Inadequately controlled hypertension (defined as systolic blood pressure (BP) >150 mmHg and/or diastolic blood pressure > 100 mmHg). Note: an average of 3 BP readings on 2 sessions will be used to measure blood pressure if the initial reading indicates inadequately controlled hypertension. Anti-hypertensive therapy to achieve blood pressures below these parameters is allowed.
  • Prior history of hypertensive crisis or hypertensive encephalopathy.
  • Persisting toxicity related to prior therapy of Grade >1 per CTCAE. Note: Alopecia, sensory neuropathy Grade <= 2 are acceptable.
  • Known, active alcohol or drug abuse.
  • History of allergy to study drug components.
  • History of >= Grade 3 per CTCAE infusion-related reaction to bevacizumab or PRGN-2012.
  • Pregnancy confirmed with beta-human chorionic gonadotropin (beta-HCG) serum or urine pregnancy test in WOCBP at screening.
  • Uncontrolled symptomatic, intercurrent illness evaluated by medical history, physical exam, and labs, or social situations that would unacceptably increase risk for the participant or impair the ability to evaluate the endpoints of the study, or that would limit compliance with study requirements.

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Primární účel: Léčba
  • Přidělení: N/A
  • Intervenční model: Přiřazení jedné skupiny
  • Maskování: Žádné (otevřený štítek)

Zbraně a zásahy

Skupina účastníků / Arm
Intervence / Léčba
Experimentální: Arm 1

Participants with RRP will be given Bevacizumab and PRGN-2012

PRGN-2012 (5x10^11 PU) will be administered on Days 85, 100, 128, and 170.

Administration of IV bevacizumab will be done on D1, D22, D43, D85, and D170 at a dose of 10 mg/kg during Course 1 (and Course 2 if applicable).

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
To determine the complete response rate, defined as the percentage of participants who do not require an intervention (either medical or surgical) in the 12 months following completion of treatment with combination systemic bevacizumab and PRGN-...
Časové okno: From baseline to 12 months after treatment

Determined by measuring the number of participants (evaluable for clinical response) who do not require an intervention (either medical or surgical) in the 12 months following completion of treatment. This fraction of participants who are classified as having a complete response at 12 months will be reported along with 80% and 95% two-sided confidence intervals. For participants receiving re-treatment, any increase in their treatment-free interval following re-treatment will not be used for evaluation of the primary endpoint.

From baseline to 12 months after treatment

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
To determine the recurrence-free interval of laryngotracheal papillomatous disease after combination treatment
Časové okno: Baseline through 3 years after treatment

The time to recurrence of papillomatous disease after completion of treatment will be recorded and reported descriptively.

Baseline through 3 years after treatment
To determine the safety of the combination of bevacizumab and PRGN-2012
Časové okno: Between the first dose of drug on D1 through 6 weeks, as well as on the 6-week safety follow-up visit. After 6-week safety follow-up visit, only adverse events that are serious and related to the study interventions

AEs will be reported by type and grade.

Between the first dose of drug on D1 through 6 weeks, as well as on the 6-week safety follow-up visit. After 6-week safety follow-up visit, only adverse events that are serious and related to the study interventions
To determine the treatment-free interval (either medical or surgical) after completion of treatment with combination systemic bevacizumab and PRGN-2012
Časové okno: Up to 3 years after treatment

Reported descriptively by measuring the duration between completion of combination treatment and the time to the requirement of the first clinical intervention (either medical or surgical) following completion of protocol therapy.

Up to 3 years after treatment
To determine the overall response rate (ORR) of pulmonary RRP defined as complete response (CR) and partial response (PR) by RECIST 1.1 in participants with measurable pulmonary disease after combination treatment
Časové okno: Baseline and 6 weeks after completion of treatment

The fraction of participants with a pulmonary RRP partial response and a pulmonary RRP complete response will be reported in all treated pulmonary participants, along with 95% confidence intervals for each.

Baseline and 6 weeks after completion of treatment
To determine the treatment-free interval (either medical or surgical) after completion of retreatment with combination systemic bevacizumab and PRGN-2012
Časové okno: Baseline through 3 years after treatment

Reported descriptively following completion of protocol retreatment.

Baseline through 3 years after treatment

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Vyšetřovatelé

  • Vrchní vyšetřovatel: Scott M Norberg, D.O., National Cancer Institute (NCI)

Publikace a užitečné odkazy

Osoba odpovědná za zadávání informací o studiu tyto publikace poskytuje dobrovolně. Mohou se týkat čehokoli, co souvisí se studiem.

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia (Odhadovaný)

9. listopadu 2026

Primární dokončení (Odhadovaný)

1. července 2029

Dokončení studie (Odhadovaný)

1. července 2030

Termíny zápisu do studia

První předloženo

8. srpna 2026

První předloženo, které splnilo kritéria kontroly kvality

10. srpna 2026

První zveřejněno (Aktuální)

11. srpna 2026

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

4. listopadu 2026

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

3. listopadu 2026

Naposledy ověřeno

5. srpna 2026

Více informací

Termíny související s touto studií

Plán pro data jednotlivých účastníků (IPD)

Plánujete sdílet data jednotlivých účastníků (IPD)?

ANO

Popis plánu IPD

This study will comply with the NIH Data Management and Sharing (DMS) Policy, which applies to all new and ongoing NIH-funded research in the IRP, as of January 25, 2023, that is associated with a ZIA, with a clinical protocol that undergoes scientific review.

Časový rámec sdílení IPD

Data will be made available as soon as possible or at the time of associated publication. Data not published in a manuscript will be shared via public source once the data set completes QC.

Kritéria přístupu pro sdílení IPD

Clinical data will be made available upon request and with the permission of the study PI.

Typ podpůrných informací pro sdílení IPD

  • PROTOKOL STUDY
  • MÍZA
  • ICF

Informace o lécích a zařízeních, studijní dokumenty

Studuje lékový produkt regulovaný americkým FDA

Ano

Studuje produkt zařízení regulovaný americkým úřadem FDA

Ne

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