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Clinical Trials/NL-OMON54689
NL-OMON54689RecruitingPhase 2

Evaluation of safety and efficacy of mitapivat sulfate in adult patients with sickle cell disease - ESTIMATE

Julius Clinical0 sites10 target enrollmentStarted: TBDLast updated:
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Conditions

Trial Snapshot

Phase
Phase 2
Status
Recruiting
Sponsor
Enrollment
10
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional

Eligibility Criteria

Ages
16 to 64 (—)

Inclusion Criteria

  • •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.

Exclusion Criteria

  • •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

Investigators

Sponsor
Julius Clinical

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