A Phase 3, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Darvadstrocel in the Treatment of Complex Perianal Fistula in Pediatric Subjects with Crohn's Disease over a Period of 24 Weeks and an Extended Follow-up Period for a Total of up to 52 Weeks. - Darvadstrocel-3004

Takeda0 个研究点目标入组 5 人开始时间: 待定最近更新: 2024年9月9日

适应症

试验速览

阶段: 3 期
状态: 尚未招募
发起方
入组人数: 5

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

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研究设计

研究类型: Interventional

入排标准

年龄范围: 2 至 17（—）

入选标准

•Subject eligibility is determined according to the following criteria prior to
•entry into the study:
•1. In the opinion of the investigator, the subject is capable of understanding
•and complying with protocol requirements.
•2. The subject, or when applicable, the subject's legally acceptable
•representative, signs and dates a written, informed consent/pediatric assent
•form and any required privacy authorization before the initiation of any study
•procedures.
•3. The subject is male or female aged 4 to <18 years at the time of informed
•consent/pediatric assent.
•4. The subject has a CD diagnosis based on accepted clinical, endoscopic,
•histological and/or radiologic criteria at least 6 months before the screening
•5. The subject has complex perianal fistula refractory to at least one of the
•following treatments: immunosuppressants or biologics (anti-TNFs,
•anti-integrin, anti-interleukin
•[IL] 12/23).
•Fistula(s) refractory to therapy are defined in this study as follows:
•Immunosuppressants: Inadequate response after 3 months, based on clinical
•assessment, or more treatment with azathioprine, 6-mercaptopurine or
•methotrexate.
•Biologics: Inadequate response after 14 weeks (16 weeks for anti-IL12/23),
•based on clinical assessment, or more standard treatment for induction and
•maintenance.
•6. A complex perianal fistula(s) that meets one or more of the following
•criteria, modified from the American Gastroenterological Association (AGA)
•technical review:
•High intersphincteric, transsphincteric, extrasphincteric, or
•suprasphincteric as assessed by MRI.
•Presence of 2 or 3 external openings (tracts) as assessed by clinical
•examination.
•Associated fluid (abscess) collections as assessed by MRI.
•This study requires that the subject has complex perianal fistulas with a
•maximum of 2 internal openings and a maximum of 3 external openings, based on
•clinical assessment. Darvadstrocel treatment is targeted for fistulas that
•connect between internal and external openings. A central reading of a locally
•performed pelvic MRI will be performed to confirm the location of the fistula
•and potential associated perianal abscess(es). Fistulas must have been draining
•for at least 6 weeks before the screening visit. Subjects with actively
•draining simple subcutaneous fistulas, at the time of the screening visit, are
•not allowed in this study.
•7. The subject has inactive or mildly active luminal CD defined by meeting all
•of the following criteria:
•a. Colonoscopy, flexible sigmoidoscopy or rectoscopy performed either at
•screening or within the 6 months before screening, demonstrating no rectal
•ulcers larger than 0.5 cm. A subject who has documented rectal ulcers larger
•than 0.5 cm within the 6 months before screening but has undergone subsequent
•treatment may be eligible if there are no rectal ulcers larger than 0.5 cm on a
•sigmoidoscopy or rectoscopy performed after treatment or at the time of
•b. The improvement of, or no worsening in stool frequency, sustained for 1 week
•or more, in the interval between the colonoscopy, flexible sigmoidoscopy or
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排除标准

•Any subject who meets any of the following criteria will not qualify for entry
•into the study:
•1. The subject has received any investigational compound within 12 weeks/84
•days before screening.
•2. The subject has received darvadstrocel/eASC in a previous clinical study or
•as a therapeutic agent.
•3. The subject is an immediate family member, study site employee, or is in a
•dependent relationship with a study site employee who is involved in conduct of
•this study (eg, spouse, parent, child, sibling), or may consent under duress.
•4. The subject weighs <10 kg at screening.
•5. The subject has, in the judgment of the investigator, clinically significant
•abnormal hematological parameters of hemoglobin, hematocrit, or erythrocytes at
•6. The subject has a history of hypersensitivity or allergies to darvadstrocel
•or any of its excipients.
•7. The subject takes or is required to take excluded medications.
•8. The subject has concomitant perianal fistula(s) with only internal or
•external opening(s).
•9. The subject has concomitant internal fistula(s) such as ileo-vesical,
•rectovaginal or ileo-colonic fistula(s).
•10. The subject has an abscess >2 cm, unless resolved in the preparation
•11. The subject has rectal and/or anal stenosis, and/or active proctitis, which
•would restrict the surgical procedure.
•12. The subject underwent surgery for the fistula other than drainage or seton
•13. The subject has diverting stomas.
•14. The subject has ongoing systemic corticosteroid treatment or has been
•treated with systemic corticosteroids within 4 weeks before screening.
•15. The subject requires new treatment with immunosuppressants/anti-TNF agents
•during the screening period.
•16. The subject has known or suspected COVID-19 by the investigator within the
•past 2 months (additional testing may be performed at the discretion of the
•investigator). Positive antibody testing for COVID without other evidence of
•current or recent active infection does not exclude participation.
•-Subjects who were in screening at the time that COVID-19-related factors
•resulted in discontinuation may also be rescreened with approval of the sponsor
•or designee.
•17. The subject requires surgery in the perianal region for reasons other than
•fistulas at the time of screening or foreseen either during the study and/or
•during the 24 weeks after treatment administration.
•18. The subject has a serum creatinine >=2 × upper limit of normal (ULN).
•19. The subject has hepatic impairment defined by both of the following
•laboratory ranges:
•a) Total bilirubin >=1.5 × ULN.
•b) Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) >=2 × ULN.
•20. The subject has known history of abuse of alcohol or other addictive
•substances in the 6 months before screening.
•21. The subject has malignant tumor or a prior history of any malignant tumor,
•including any type of fistula carcinoma.
•22. The subject has current or recent (within 3 months before the screening)
•history of abnormal, severe, progressive, uncontrolled hepatic, hematologic,
•gastrointestinal (except CD), endocrine, pulmonary, cardiac, neurological,
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研究者

发起方

Takeda

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