NL-OMON55024尚未招募2 期
Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease - Study to investigate SPK-3006 in adults with late-onset Pompe disease
Spark Therapeutics0 个研究点目标入组 2 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 2
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. Be able to understand the purpose and risks of the study and provide signed
- •and dated informed consent and authorization to use protected health
- •information (PHI) in accordance with national and local privacy regulations;
- •2. Are male or female >=18 years of age with a confirmed diagnosis (e.g., GAA
- •genetic testing) of LOPD, or based on a documented deficiency of GAA enzyme
- •3. Have received a marketed ERT for at least the previous 24 months and
- •maintained a stable dose, frequency and compliance for the past 6 months with
- •no dose variation and no longer improving on ERT;
- •4. Have clinically moderate LOPD characteristics
- •a. Be able to walk >=75 meters on the 6MWT (assistive devices permitted) but
- •less than 500 meters assessed at two timepoints prior to initiating
- •immunosuppression (Day -6) with <10% variance between the assessments. If the
- •variance is >=10%, a third timepoint will be collected;
- •b. Have a percentage of the predicted FVC >=30% and <=80% in the upright position;
- •5. Agree to use reliable contraception for a minimum of 6 months after
- •administration of SPK-3006 or 12 weeks after the final dose of sirolimus,
- •whichever occurs later. Female candidates of child-bearing potential must have
- •a negative pregnancy test prior to initiating immunosuppression (Day -6) and on
- •Day 0 prior to administration of SPK-3006. See Section 13 (Appendix 1) for
- •guidance on reliable contraception and the definition of women of child-bearing
- •potential (WOCBP);
- •6. Agree to refrain from blood, plasma, platelets, egg or sperm, and organ
- •donation after receiving SPK-3006.
排除标准
- •1. Have active hepatitis B and/or C;
- •All candidates must be screened for both active hepatitis B and C, regardless
- •of prior known history.
- •a. Screening for hepatitis B.
- •All candidates must have a single sample at Screening for each of the following
- •tests: hepatitis B surface antigen (HBsAg), total hepatitis B core antibody
- •(anti-HBc), and a nucleic acid test for hepatitis B virus DNA (HBV-DNA viral
- •assay).A candidate is not eligible if either HbsAg is positive or HBV-DNA is
- •positive/detectable.
- •i. A candidate is not eligible if either HBsAg is positive or HBV-DNA is
- •positive/detectable.
- •ii. A candidate is eligible if the anti-HBc is positive and both HBsAg and
- •HBV-DNA are negative, as this would be consistent with a prior infection of
- •hepatitis B. Anti-HBc must be obtained in all candidates to discriminate
- •between acute infection and possible reactivation of hepatitis B during the
- •trial in candidates with no prior history of hepatitis B.
- •iii. A candidate who is currently undergoing antiviral therapy for chronic
- •hepatitis B is not eligible.
- •b. Screening for hepatitis C:
- •iv. A candidate who is currently undergoing antiviral therapy for chronic
- •hepatitis C is not eligible.
- •v. All other candidates, including those who have never been treated or who
- •have completed antiviral therapy for chronic hepatitis C must have a nucleic
- •acid test for hepatitis C viral RNA (HCV-RNA single load assay) at Screening.
- •A candidate is not eligible if the HCV-RNA load assay is positive/detectable.
- •Candidates treated with anti-viral therapy for chronic hepatitis C, must have
- •completed anti-viral therapy at least 6 months prior to Screening and have a
- •negative HCV-RNA at Screening.
- •Candidates with a documented or self-reported history of hepatitis C must
- •have a single negative HCV-RNA at Screening.
- •2. Have significant underlying liver disease. A candidate is not eligible if
- •any of the following pre-existing diagnoses, which are indicative of
- •significant underlying liver disease, are present in the medical record:
- •Liver cirrhosis;
- •Portal hypertension; or
- •Hepatic encephalopathy
- •Gamma-glutamyl transferase (GGT) >1.2X upper limit of normal (ULN) adjusted
- •for age and gender; or
- •Bilirubin >1.2X ULN adjusted for age and gender. Candidates with asymptomatic
- •elevated bilirubin (e.g., Gilbert syndrome) can be considered after discussion
- •with the Sponsor Medical Monitor.
- •All candidates who do not have the pre-existing diagnoses listed above must
- •have the following assessments performed at screening:
- •Measurement of serum albumin. A candidate is not eligible if the serum
- •albumin level is below the testing laboratory*s lower limit of normal;
- •Liver fibrosis >= stage 3. The following results are indicative of fibrosis >=
- •stage 3 and exclude the candidate from participation:
- •- FibroScan, with a score > 8.3 kPa units
- •- FibroTest/ FibroSURE with a result > 0.48
- •- AST-Platelet Ratio Index (APRI) >1;
- 另有 6 项未显示
研究者
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