ISRCTN17160440已完成3 期
Autologous Stem cell Transplantation In refractory Crohn's disease - Low Intensity Therapy Evaluation
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 45
研究概览
简要总结
2019 Protocol article in https://pubmed.ncbi.nlm.nih.gov/31151436/ (added 11/08/2022) 2024 Results article in https://doi.org/10.1016/s2468-1253(23)00460-0 (added 12/02/2024)
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Participant of any gender, aged between 18 – 60
- •2. Participants must be willing and able to provide full informed consent
- •3. Participants should be well nourished and of healthy weight in the opinion of the PI (typically BMI >18.5)
- •4. Diagnosis of CD using colonoscopy, histology and/or radiology
- •5. Disease duration of at least six months
- •6. Disease distribution accessible to endoscopic assessment (ileal, ileo-caecal, or colonic)
- •7. Active clinical CD activity with impaired quality of life at any time within 3 months prior to randomisation into the trial, as assessed by a gastroenterology clinician
- •8. Participants will be refractory or intolerant to azathioprine, mercaptopurine or methotrexate
- •9. Participants will be refractory or intolerant to at least two classes of biologic therapy (currently anti-TNF therapy, Vedolizumab or Ustekinumab) despite dose optimisation
- •10. Participants where surgery is considered not appropriate or has been declined
- •11. Endoscopic evidence of active disease in screening (SES CD ulceration sub-score of 2 or more in at least one segment)
- •12. Satisfactory EBMT Autoimmune Disease Working Party (ADWP) recommended screening assessment prior to HSCT
- •13. Willingness to discontinue all immunosuppressant medication after randomisation if allocated to HSCT arm
- •14. Participants, who, in the opinion of the TMG, are fit enough to undergo treatment
排除标准
- •1. Diagnosis of ulcerative colitis or indeterminate colitis.
- •2. No evidence of active CD on screening ileocolonoscopy.
- •3. Inability to assess for active disease at ileocolonoscopy due to strictures.
- •4. Undrained perianal fistulae (patients with previous perianal disease or perianal disease adequately drained with a seton in situ are eligible)
- •5. Presence of undrained perianal sepsis on screening pelvic MRI.
- •6. Evidence of intra-abdominal sepsis on abdominal MRI.
- •7. Active or latent mycobacterial infection.
- •8. Prior exposure to Hepatitis B, Hepatitis C or HIV.
- •9. Evidence of an enteric or systemic infection.
- •10. Participant is currently pregnant or breastfeeding, or planning pregnancy within the study duration. Current pregnancy will be confirmed with a pregnancy test at screening assessment.
- •11. Unwilling to use adequate contraception (if appropriate) until at least 12 months after the last dose of study drug.
- •12. Contraindication to the use of cyclophosphamide, fludarabine, filgrastim or rabbit ATG.
- •13. Participants with significant medical co-morbidity that precludes HSCT adjudicated by the TMG.
- •14. Participants with significant psychiatric co-morbidity.
- •15. Significant language barriers, which are likely to affect the participant’s understanding of the study, or ability to complete outcome questionnaires.
- •16. Concurrent participation in another interventional clinical trial.
- •17. Participants who are not considered medically fit for HSCT defined by any of the following:
- •17.1. Renal: creatinine clearance <40 ml/min (measured or estimated)
- •17.2. Cardiac: clinical evidence of refractory congestive heart failure, left ventricular ejection fraction <45% by multigated radionuclide angiography (MUGA) or cardiac echo; uncontrolled ventricular arrhythmia; pericardial effusion with haemodynamic consequences as evaluated by an experienced echo cardiographer
- •17.3. Hepatic: AST > two times the upper limit of normal
- •17.4. Concurrent neoplasms or myelodysplasia
- •17.5. Bone marrow insufficiency defined as neutropenia with an absolute neutrophil count <1x10(9)/l, or thrombocytopenia with a platelet count <50x10(9)/l, or anaemia with a haemoglobin <80 g/l
- •17.6. Uncontrolled hypertension, defined as resting systolic blood pressure >= 140 and/or resting diastolic pressure >= 90 mmHg despite at least 2 anti-hypertensive agents (subject to discussion at TMG).
- •17.7. Uncontrolled acute or chronic infection with HIV, HTLV – 1 or 2, hepatitis viruses or any other infection the investigator or TMG consider a contraindication to participation.
- •17.8. Other chronic disease causing significant organ failure, including established cirrhosis with evidence of impaired synthetic function on biochemical testing and known respiratory disease causing resting arterial oxygen tension <8 kPa or carbon dioxide tension >6.7 kPa. FEV1/FVC <50%. Patients not known to have respiratory disease need not have blood gas measurements.
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