CTRI/2022/03/041304已完成1 期
Gene Therapy for Hemophilia A with a high expression Factor VIII transgene inautologous Hematopoietic Stem Cells - GTHA
Centre for stem cell Research A Unit of inStem Bengaluru0 个研究点目标入组 6 人开始时间: 待定最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 6
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
入选标准
- •1.Able to provide informed consent for the protocol approved by the Institutional Review Board.
- •2.Male subjects who are = 45 years of age and have completed family with children. Upper limit of < 55 years of age
- •3.Patients who have high AAV antibody titers which makes them ineligible for participation in other AAV gene therapy clinical trials
- •4.Diagnosis of severe hemophilia A ( <1 IU/dl factor VIII activity).
- •5.Documented history of more than 100 exposures of factor VIII treatment.
- •6.Average of at least 3 bleeds requiring treatment per year over the prior three years, at least 3 bleeds per year during the 3 years preceding the initiation of prophylaxis, or evidence of joint damage (knee, elbow or ankle) on physical or radiographic examination thought to be related to hemophilia.
- •7.Performance status (Karnofsky score) of at least 70
- •8.Willingness to use barrier contraception or limit sexual intercourse to post-menopausal, surgically sterilized, or contraception-practicing partners, for 100 days after transplantation.
- •9.Willing and able to comply with the requirements of the protocol.
排除标准
- •1.History of spontaneous central nervous system bleeding within the last 5 years.
- •2.Significant functional deficits in major organs which would interfere with successful outcome following autologous stem cell transplant, the following guidelines will be utilized:
- •Cardiac: There should be no evidence of significant cardiac dysfunction (resting left ventricular ejection fraction of < 50%) and no marked cardiomegaly. There should not be uncontrollable hypertension.
- •Renal: GFR < 60 ml/min/1.73m2 as calculated using the Cockcroft-Gault equation.
- •Hepatic: There should be no evidence of hepatic dysfunction which is defined as a serum bilirubin of > 1.5 mg/dl and AST/ALT > 3X the upper limit of normal,
- •Hematologic: Absolute neutrophil counts (ANC) < 1000/mm3 and platelets counts < 150,000/µL.
- •Pulmonary function with a corrected DLCO of < 50% predicted
- •History of a FVIII inhibitor ( > 0.6 Bethesda Units/ml) including at least 2 measurements over the preceding 5 years or any single titer > 5 BU/ml.
- •5.Previous stem cell transplant.
- •6.HIV or RPR positive.
- •7.Patients who have allergic reactions to lupine ATG
- •8.Evidence of hepatitis B active infection or chronic carrier
- •9.Evidence of chronic hepatitis C infection. Absence of chronic infection will be documented with at least 2 negative viral loads at least 6 months apart.
- •10.Diagnosis of a bleeding disorder other than hemophilia A
- •11.Use of medication(s) that can affect hemostasis (e.g. aspirin and non-COX-2 selective non-steroid anti-inflammatory drugs).
- •12.History of cancer or familial cancer syndromes (e.g. leukemia, colorectal cancer).
- •13.Any condition in the opinion of the principle investigator that will negatively impact the subject’s ability to safely undergo an autologous stem cell transplant.
- •14.Any reason in the opinion of the principle investigator that will negatively impact the subject’s ability to complete the clinical trial per the trial protocol.
研究者
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