An Open-Label, Multi-Center, Expanded Treatment Protocol of Quizartinib in Adult Subjects With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FLT3-ITD Mutations
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Acute Myeloid Leukemia With Gene Mutations
- Sponsor
- Daiichi Sankyo
- Status
- No Longer Available
- Last Updated
- 6 years ago
Overview
Brief Summary
An expanded access program (EAP):
- Allows doctors to give medicine to patients,
- Before it is approved by health authorities.
This EAP is for:
- Quizartinib
- Patients with FLT3-ITD mutated AML,
- AML that has come back, or
- Is resistant to other therapies.
A participant will receive quizartinib if:
- The doctor submits a request,
- The participant is eligible, and
- The country allows the EAP.
Detailed Description
This is an expanded access program (EAP) providing access to quizartinib for participants with Relapsed/Refractory FLT3-ITD mutated AML, prior to approval by local regulatory agencies. Availability of the EAP is dependent upon physician request, country eligibility and local/country regulations. EAP countries included Austria, Belgium, Brazil, Denmark, France, Germany, Ireland, Italy, Netherlands, Norway, South Korea, Spain, Sweden, Switzerland, Taiwan, Thailand, United Kingdom, and United States. Physicians may request access to the EAP for participants who they feel may benefit from quizartinib and meet the eligibility criteria. Participants may continue quizartinib until there is a lack of clinical benefit or the occurrence of unacceptable toxicity. Treatment should be interrupted for allogeneic hematopoietic stem cell transplantation (HSCT), but may be resumed after the transplant. Participant enrollment may continue until 18 months after regulatory approval, depending on country regulation; or until such time the marketed medication is available, whichever occurs first. Participants will be asked to follow the care as outlined by their treating physician. Participants will be followed for 30 days after their final treatment or until the patient is transitioned to commercially available product. Physicians will be required to report safety data to Daiichi-Sankyo Inc. Quizartinib is currently under development for the treatment of patients 18 years of age or older with relapsed (including after HSCT) or refractory FLT3-ITD mutated AML, and has been granted Fast Track Status in the US and an Orphan Drug Indication in the United States, Europe and Asia.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Participants must satisfy all of the following inclusion criteria prior to receive treatment:
- •Has provided written informed consent as approved per local regulations with privacy language in accordance with national regulations (eg, Health Insurance Portability and Accountability Act \[HIPAA\] authorization for US sites) prior to any protocol-related procedures
- •Is 18 years of age or the minimum legal adult age (whichever is greater) at the time of informed consent
- •Has morphologically documented primary AML or AML secondary to myelodysplastic syndrome (MDS) or other myeloproliferative neoplasms (MPN), as defined by the World Health Organization (WHO) 2008 classification with ≥5% blasts in bone marrow, with or without extramedullary disease
- •Is in relapse or refractory (R/R) to prior therapy, with or without HSCT
- •Refractory is defined as:
- •participant did not achieve complete remission (CR), CR with incomplete platelet recovery (CRp), or CR with incomplete hematological recovery (CRi) under initial intensive therapy, or
- •did not achieve CR, CRp, CRi, or partial remission (PR) under initial sufficient time course of treatment with hypomethylating agents or low-dose cytarabine (LoDAC)
- •must have received at least one complete block of induction therapy seen as the optimum choice of therapy to induce remission or at least 4 cycles of therapy with a hypomethylating agent deemed sufficient to induce a response
- •Relapse is defined as: relapse diagnosed by bone marrow assessment or by the appearance of peripheral blasts after the achievement of CR, CRp, or CRi, as defined by 2003 International Working Group criteria after AML therapy with or without consolidation or maintenance, and with or without HSCT
Exclusion Criteria
- •Participants who meet any of the following criteria will be disqualified from enrollment:
- •Is eligible to enroll in a recruiting clinical trial of quizartinib or is currently enrolled in an ongoing clinical trial of quizartinib
- •Has previously participated in a randomized clinical study of quizartinib with an endpoint of survival that is not closed for efficacy
- •Has acute promyelocytic leukemia (AML subtype M3)
- •Has persistent, clinically significant ≥Grade 3 non-hematologic toxicity from prior AML therapy
- •Has clinically significant acute graft-versus-host disease (GvHD) or GVHD requiring initiation of treatment or treatment escalation within 21 days, and/or ≥Grade 3 persistent or clinically significant non-hematologic toxicity related to HSCT
- •Has uncontrolled or significant cardiovascular disease, including:
- •QTcF interval \>450 ms
- •Bradycardia of less than 50 beats per minute (bpm) unless the patient has a pacemaker
- •Diagnosed or suspected long QT syndrome, or known family history of long QT syndrome
Outcomes
Primary Outcomes
Not specified