JPRN-jRCT2063210019招募中1 期
Phase I / IIa clinical study (FIH study) to investigate the safety and efficacy of high-purity mesenchymal stem cell (REC-01) transplantation in pediatric patients with hypophosphatasia - REC-01-001
Taketani Takeshi0 个研究点目标入组 3 人开始时间: 2021年6月23日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 3age old 至 < 12age old（—）
- 性别
- All
入选标准
- •[Registration (1)] Immediately after obtaining consent, confirm that the following criteria (1) to (5) are met.
- •[Registration (2)] When the bone marrow fluid and cord blood can be arranged from a suitable donor (selection criteria (6) and (7)) and the REC-01 production schedule from the bone marrow fluid is in sight, cord blood transplantation and cord blood are available. Schedule REC-01 for the first dose and confirm that it conforms to (1) to (5) again within 1 month before the scheduled date and during the screening period before starting the pretreatment for cord blood transplantation.
- •1) Patients who have voluntarily obtained consent from the subject's substitute (guardian) based on the explanation / consent document approved by the clinical trial review committee.
- •(2) Patients diagnosed with hypophosphatasia according to the diagnostic criteria for pediatric specific chronic diseases Note 1
- •Note 1: Diagnostic criteria established by the Ministry of Health, Labor and Welfare Grant-in-Aid for Scientific Research to Overcome Intractable Diseases Basic and Clinical Study for Individually Optimized Treatment of Hypophosphatasia Disease group (Research Leader: Keiichi Ozono) ..
- •[Main symptoms] 1. Bone calcification disorder: Simple X-ray findings of bone include low calcification of bone, deformation of long canal bone, and rickets-like metaphyseal irregularity.
- •2. Early loss of deciduous teeth (dropout under 4 years old)
- •[Main laboratory findings] 1. Low serum alkaline phosphatase (ALP) level (Note the normal value by age: Judging by the normal value by age at each institution, the serum ALP value of growing children is 100 IU / L. If less than, you need to doubt your true nature)
- •(3) Patients with severe HPP defined below who have been administered ashotase alpha at the time of obtaining consent and whose symptoms have been stable after a sufficient administration period (6 months to several years).
- •Definition of severe illness: Those who meet all of the following: Having developed before 6 months of age, having respiratory disorders (Note 2), and ALPL gene mutation showing ALP activity of 50% or less compared to healthy subjects Have (Note 3)
- •Note 2: Respiratory disorders are those who have received or have received oxygen administration or mechanical ventilation management (including non-invasive positive pressure ventilation therapy).
- •Note 3: The specific activity per amount of ALP enzyme protein is 50% or less compared to healthy subjects.
- •(4) Patients whose consent date is 3 to 12 years old
- •(5) Patients who do not have the following obstacles that interfere with transplantation
- •ECOG performance status score at enrollment is 3 or less, total bilirubin level is 2.0 mg / dL or less, serum creatine is below the upper limit of normal age, or creatine clearance (measured value) is 60 mL / min or more. No ECG abnormalities requiring treatment, BNP 100 pg / mL or less, and left ventricular ejection fraction 50% or more.
- •(6) As a bone marrow fluid donor for the production of REC-01, a donor that matches 3 or more of the HLA 6 loci (-A, -B, -DRB1) between the bone marrow fluid donor and the subject can be found.
- •(7) According to the usual cord blood transplantation, a donor with 4 or more of the 6 HLA loci (-A, -B, -DRB1) matching can be found between the cord blood donor and the subject.
- •(8) Pregnant female patients should be admitted to this clinical trial only if the pregnancy test confirms that they are not pregnant and that the pat
排除标准
- •[Registration (1)] Immediately after obtaining consent, confirm that it does not violate any of the following criteria (1) to (10).
- •(1) History of heart failure requiring treatment.
- •(2) History of renal failure.
- •(3) Uncontrollable infectious diseases: Infectious diseases in which signs and symptoms related to infection continue and no improvement is observed even with appropriate treatment.
- •(4) Uncontrollable diabetes: Coexisting uncontrolled diabetes with HbA1c of 8% or more despite appropriate treatment.
- •(5) Coexisting cirrhosis.
- •(6) Coexistence of severe mental disorders that interfere with treatment performance.
- •(7) The brain wave is a flat brain wave.
- •(8) Patients who have had a cord blood transplant before obtaining consent.
- •(9) Patients who have been treated for mesenchymal stem cell transplantation in the past.
- •(10) Patients with hypersensitivity to bovine and human serum-derived proteins and antibiotics.
- •(11) In addition, patients who are judged by the investigator or investigator to be ineligible as subjects.
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