NL-OMON54689招募中2 期
Evaluation of safety and efficacy of mitapivat sulfate in adult patients with sickle cell disease - ESTIMATE
Julius Clinical0 个研究点目标入组 10 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 10
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
- 16 至 64（—）
入选标准
- •1. Male or female with homozygous sickle cell anemia (HbSS) or HbS/beta(0 or
- •+)-thallassemia). 2. Documented history of VOCs, and number of days admitted in
- •hospital for acute sickle cell related complications during 24 months before
- •inclusion. 3. SCD with at least one of the following conditions: I. Had at
- •least 1 (but no more than 10) VOC in the past 12 months prior to the first day
- •of study treatment. II. any sickle cell related hospital admission in the past
- •12 months prior to the first day of study treatment; III. any history of sickle
- •cell related complications (such as osteonecrosis, osteoporosis, nephropathy,
- •retinopathy, leg ulcer, acute chest syndrome, acute hemolytic crisis); IV.
- •presence of any clinical biomarkers associated with increased mortality in SCD
- •prior to the first day of study treatment (NT-proBNP >160 pg/mL, LDH/HbCO ratio
- •>1,200, tricuspid regurgitant jet velocity =2.5 m/s). 4. Age 16 years and
- •older, inclusive; subjects age 16 or 17 years must be documented Tanner Stage
- •5. 5. Hemoglobin <=6.9 mmol/L (approx 11.1 g/dL) and >2.5 mmol/L (approx 4.0
- •g/dL). 6. For subjects on hydroxyurea: the dose must have been stable for at
- •least 3 months prior the 1st day of study treatment. 7. Subjects must start or
- •continue taking at least the equivalent of daily 0.7 mg oral folic acid for the
- •duration of the study. 8. Have adequate organ function based on ALT, AST,
- •billirubin, creatinine, neutrophil and platelet count and INR. 9. Willing and
- •able to give written informed consent and comply to all study procedures. 10.
- •Patients with increased albumin to creatinine ratio are prioritized above
- •patients with a normal albumin to creatinine ratio. Both are eligible. 11. For
- •women of reproductive potential: have a negative urine and serum pregnancy test
- •at screening. 12. For (fertile men of) women of reproductive potential: Agree
- •to use double anticonception during the study plus 90 days (for males) or 28
- •days (for females) after the last dose of the study drug.
排除标准
- •1. More than 10 VOCs within the past 12 months.
- •2. Hospitalized for sickle cell crisis or other vaso-occlusive event within 14
- •days prior to the first day of study treatment (rescreening is allowed).
- •3. Have a point of sickling (PoS) <=24.6 mmHg as quantified by the Oxygenscan
- •during screening to exclude subjects with no clinical relevant detectable
- •4. Subjects age 16 or 17 years who are documented Tanner stage 1-4 (see
- •Appendix II).
- •5. Receiving regularly scheduled (red blood cell) transfusion, defined as more
- •than 4 transfusions in the 12 months prior to the first day of study treatment,
- •and/or have received a transfusion within the past 3 months prior to the first
- •day of study treatment.
- •6. Have a significant medical condition that confers an unacceptable risk to
- •participation in the study, and/or that could confound interpretation of the
- •study data (such as poorly controlled hypertension, cardiac diseases,
- •cholelithiasis, cholecystitis, cholestatis hepatitis, iron overload that could
- •result in cardiac/hepatic/pancreatic dysfunction, have diagnosis of other
- •congenital or acquired blood disorder, active hepatitis B or C infection or
- •antibodies, HIV-1 of HIV-2 antibodies, active infections, poorly controlled
- •diabetes mellitus, history of primary malignancy (except for non-melanomatous
- •skin cancer, curatively treated cervical or breast carcinoma in situ with no
- •known active disease present and no treatment administered during the last 3
- •years, unstable extramedullary hematopoiesis that could pose a risk of imminent
- •neurologic compromise, severe hepatic fibrosis/cirrhosis or NASH, current or
- •recent history of psychiatric disorder that could compromise the ability of the
- •subject to cooperate with study visits and procedures.
- •7. Are currently enrolled in another therapeutic clinical trial involving
- •ongoing therapy with any investigational or marketed product or placebo.
- •Participation in registry studies is allowed.
- •8. Have exposure to any investigational drug, device, or procedure within 3
- •months prior to the first dose of study treatment.
- •9. Have had any prior treatment with a pyruvate kinase activator.
- •10. Have a prior bone marrow or stem cell transplant.
- •11. Are currently pregnant or breastfeeding, or planning to become pregnant
- •during the course of the study.
- •12. Have a history of major surgery within 6 months of signing informed
- •consent. Note that procedures such as laparoscopic gallbladder surgery are not
- •considered major in this context.
- •13. Are currently receiving medications that are strong inhibitors of CYP3A4 or
- •strong inducers of CYP3A4 that have not been stopped for a duration of at least
- •5 days or a timeframe equivalent to 5 half-lives (whichever is longer) prior to
- •the first dose of study treatment.
- •14. Are currently receiving hematopoietic stimulating agents (eg,
- •erythropoietins, granulocyte colony stimulating factors, thrombopoietins) that
- •have not been stopped for a duration of at least 28 days prior to the first
- •dose of study treatment.
- •15. Known allergy to mitapivat or its excipients (microcrystalline cellulose,
- •croscarmellose sodium, sodium stearyl fumarate, and mannitol) or history of
- •acute allergic reaction to drugs characterized by acute hemolytic anemia,
- •drug-induced liver injury, anaphylaxis, rash of erythema multiforme ty
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