IDH1 (AG 120) Inhibitor in Patients With IDH1 Mutated Myelodysplastic Syndrome (NCT03503409) | Clinical Trial Compass
Active — Not RecruitingPhase 2
IDH1 (AG 120) Inhibitor in Patients With IDH1 Mutated Myelodysplastic Syndrome
France68 participantsStarted 2019-05-14
Plain-language summary
patients with MDS (Myelodysplastic Syndrome) and mutated IDH1 patients will be treated with AG120 (IDH1 inhibitor)
Who can participate
Age range18 Years
SexALL
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Inclusion Criteria:
* Patients must meet all of the following criteria to participate in the study:
* Age ≥ 18 years
* Myelodysplastic syndrome according to WHO classification including non-proliferative AML up to 29% of BM blast
* Belonging to one of the following categories :
* higher risk (IPSS high or int 2 ) MDS without response to azacitidine (CR,PR, stable disease with HI) after at least 6 cycles , or relapsing after a response but without overt progression (defined by at least doubling of marrow blasts, compared to pre azacitidine bone marrow, or AML progression beyond 30% blasts)
* Untreated higher risk MDS (IPSS int-2, high) without life threatening cytopenia including ANC \<500/mm3 or any recent severe infections and /or platelets below 30,000/mm3 or any bleeding symptom
* lower risk MDS with resistance or loss of response to a previous treatment with epoetin alpha/ beta (≥60000 U/w) or Darbopoetin (≥250 ug/w) given for at least 12 weeks and RBC transfusion requirement at least 2 U/8 weeks in the previous 16 weeks
* Presence of IDH1 mutation in either blood or marrow prior to start of therapy;
* Normal renal function, defined by creatinine less than 1.5 times the upper limit of normal, creatinine clearance (Modification of diet in renal disease) creatinine clearance ≥ 50 mL/min;
* Normal liver function, defined by total bilirubin and transaminases less than 1.5 times the upper limit of normal;
* Adequate cardiac ejection fraction (\>40%);
* Patient is not known to…