NCT05407766撤回1 期
A Phase I Safety Study of Allogeneic Bone Marrow Derived MSCs for Refractory Perianal Fistulizing Crohn's Disease
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 主要终点
- OSSM-001 (300M dose) related adverse events as assessed by CTCAE v5.0
研究概览
简要总结
OSSM-001 will be evaluated in a double blind, randomized phase I safety study of a single injection of 100 and 300 million bone marrow derived MSCs in 16 patients with refractory perianal fistulas in the setting of Crohn's disease. There will be 2 cohorts (100M and 300M MSCs); 8 subjects in each cohort; 6 subjects receiving the investigational product and 2 subjects receiving placebo (normal saline) in each cohort.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Men and Women 18-75 years of age with a diagnosis of unhealing perianal fistulas in the setting of Crohn's disease and must be on maximal standard of care therapy (e.g., antibiotics, 5-ASA, and immunomodulator, anti-TNF, anti-integrin, and anti-interleukin therapies) for at least six months duration.
- •Presence of perianal fistula with maximum of two internal fistula tracts and a maximum of three external openings based on clinical assessment and MRI. Fistula must have been draining for at least 6 weeks prior to the screening visit.
- •Medically refractory perianal fistulizing Crohn's disease (i.e., standard of care such as antibiotics, immunosuppressives)
- •Have no contraindications to MR evaluations: e.g., pacemaker or magnetically active metal fragments, claustrophobia
- •Ability to comply with protocol
- •Competent and able to provide written informed consent
- •Concurrent Crohn's-related therapies with stable doses corticosteroids, 5- ASA drugs, immunomodulators, anti-TNF therapy, anti-integrin and anti-interleukin therapies are permitted.
- •A female participant and of childbearing potential must have a negative serum or urine pregnancy test at time of screening, and must not plan to become pregnant during the study.
排除标准
- •Inability to give informed consent.
- •Clinically significant medical conditions within the six months before administration of MSCs: e.g. myocardial infarction, active angina, congestive heart failure or other conditions that would, in the opinion of the investigators, compromise the safety of the patient.
- •Specific exclusions: Hepatitis B or C, HIV, Abnormal AST or ALT at screening
- •History of cancer including melanoma (with the exception of localized skin cancers) in the past five years
- •Received treatment with an investigational drug or device within 60 days of randomization or have not completed 5 half-life washout (whichever is later) for drugs or biologics. If the half-life of the product is not known, then subjects cannot be randomized within 6 months of investigation drug treatment.
- •Participated in a cell therapy-based trial within 6 months before randomization
- •Participated in Crohn's/ulcerative colitis or any fistulizing Crohn's study within 6 months before randomization
- •Pregnant or breast feeding or trying to become pregnant.
- •Presence of a rectovaginal or perineal body fistula
- •Change in Crohn's immunosuppressive regimen prior to enrollment. Subjects should be on stable, maximal medical therapy for 4 months prior to enrollment.
- •Uncontrolled intestinal Crohn's disease which will require escalation for medical therapy or surgery within 3 months of enrollment
- •Severe anal canal disease that is stenotic and requires dilation or severe proctitis making difficult to the surgery
- •A female or male participant unwilling to agree to use acceptable contraception methods during participation in study. Acceptable contraception methods include: Implants, injectables, combined oral contraceptives, an intrauterine device, a bilateral tubal ligation, a vasectomy, a vasectomized partner for female participant, double-barrier methods, and physical barrier if male participant is not vasectomized
- •Associated perianal abscess(es).
- •Laboratory exclusions: Serum creatinine levels >1.5 times the upper limit of normal (ULN). Total bilirubin >1.5 × ULN. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >5.0 × ULN. Hemoglobin <10.0 g/dL for females or <11.0 g/dL for males. Platelets <75.0 × 109/L.
- •Any contraindications to MRI or surgical or anesthetic procedure(s)
- •Any major GI surgery or major perianal local surgery within 6 months of randomization
研究组 & 干预措施
Placebo
Placebo Comparator
Single injection of normal saline
干预措施: Placebo (Other)
300M of OSSM-001
Experimental
Single injection of mesenchymal stem cells at dose of 300M
干预措施: OSSM-001 (Biological)
100M of OSSM-001
Experimental
Single injection of mesenchymal stem cells at dose of 100M
干预措施: OSSM-001 (Biological)
结局指标
主要结局
OSSM-001 (300M dose) related adverse events as assessed by CTCAE v5.0
时间窗: 12 weeks
To determine the safety of a single injection of 300M allogeneic bone marrow MSCs for the treatment of refractory perianal fistulizing Crohn's disease by assessing OSSM-001 related adverse events as assessed by CTCAE v5.0.
OSSM-001 (100M dose) related adverse events as assessed by CTCAE v5.0
时间窗: 12 weeks
To determine the safety of a single injection of 100M allogeneic bone marrow MSCs for the treatment of refractory perianal fistulizing Crohn's disease by assessing OSSM-001 related adverse events as assessed by CTCAE v5.0.
次要结局
- Fistula healing assessed by radiographic (MRI) assessments for OSSM-001 (300M dose)(12 weeks)
- Fistula healing assessed by clinical assessments for OSSM-001 (300M dose)(12 weeks)
- Fistula healing assessed by radiographic (MRI) assessments for OSSM-001 (100M dose)(12 weeks)
- Fistula healing assessed by clinical assessments for OSSM-001 (100M dose)(12 weeks)
研究者
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