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    Summary
    EudraCT Number:2020-004104-34
    Sponsor's Protocol Code Number:SNDX-5613-0700
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2022-12-20
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2020-004104-34
    A.3Full title of the trial
    A Phase 1/2, Open-label, Dose-Escalation and Dose-Expansion Cohort Study of SNDX-5613 in Patients with Relapsed/Refractory Leukemias, Including Those Harboring an MLL/KMT2A Gene Rearrangement or Nucleophosmin 1 (NPM1) Mutation
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the short- and long-term safety and tolerability of the drug SNDX-5613 in Patients with Relapsed/Refractory Leukemias. Various doses of SNDX-5613 will be investigated.
    A.4.1Sponsor's protocol code numberSNDX-5613-0700
    A.5.4Other Identifiers
    Name:INDNumber:142693
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSyndax Pharmaceuticals, Inc
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSyndax Pharmaceuticals, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSyndax Pharmaceuticals, Inc
    B.5.2Functional name of contact pointMain Telephone Number
    B.5.3 Address:
    B.5.3.1Street Address35 Gatehouse Drive, Building D, Floor 3
    B.5.3.2Town/ cityWaltham
    B.5.3.3Post code02451
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1781419-1400
    B.5.5Fax number+1781419-1420
    B.5.6E-mailclinicaltrials@syndax.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSNDX-5613 25 mg capsule
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRevumenib sesquifumarate
    D.3.9.1CAS number 2169919-22-4
    D.3.9.2Current sponsor codeSNDX-5613
    D.3.9.3Other descriptive nametrans N-ethyl-2-((4-(7-((4-(ethylsulfonamido)cyclohexyl)methyl)-2,7-diazaspiro[3.5]nonan-2-yl)pyrimidin-5-yl)oxy)-5-fluoro-N-isopropylbenzamide sesquifumarate
    D.3.9.4EV Substance CodeSUB207245
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSNDX-5613 113 mg Blend-in- Capsule (BIC)
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRevumenib sesquifumarate
    D.3.9.1CAS number 2169919-22-4
    D.3.9.2Current sponsor codeSNDX-5613
    D.3.9.3Other descriptive nametrans N-ethyl-2-((4-(7-((4-(ethylsulfonamido)cyclohexyl)methyl)-2,7-diazaspiro[3.5]nonan-2-yl)pyrimidin-5-yl)oxy)-5-fluoro-N-isopropylbenzamide sesquifumarate
    D.3.9.4EV Substance CodeSUB207245
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number113
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 3
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSNDX-5613 50 mg/mL F2 Oral Solution
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRevumenib sesquifumarate
    D.3.9.1CAS number 2169919-22-4
    D.3.9.2Current sponsor codeSNDX-5613
    D.3.9.3Other descriptive nametrans N-ethyl-2-((4-(7-((4-(ethylsulfonamido)cyclohexyl)methyl)-2,7-diazaspiro[3.5]nonan-2-yl)pyrimidin-5-yl)oxy)-5-fluoro-N-isopropylbenzamide sesquifumarate
    D.3.9.4EV Substance CodeSUB207245
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 4
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSNDX-5613 40 mg/mL F3 Oral Solution
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRevumenib monocitrate monohydrate
    D.3.9.1CAS number 2761046-45-9
    D.3.9.2Current sponsor codeSNDX-5613
    D.3.9.3Other descriptive nametrans N-ethyl-2-((4-(7-((4-(ethylsulfonamido)cyclohexyl)methyl)-2,7- diazaspiro[3.5]nonan-2-yl)pyrimidin-5-yl)oxy)-5-fluoro-N-isopropylbenzamide monocitrate monohydrate salt
    D.3.9.4EV Substance CodeSUB327448
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 5
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSNDX-5613 25 mg tablet
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRevumenib monocitrate monohydrate
    D.3.9.1CAS number 2761046-45-9
    D.3.9.2Current sponsor codeSNDX-5613
    D.3.9.3Other descriptive nametrans N-ethyl-2-((4-(7-((4-(ethylsulfonamido)cyclohexyl)methyl)-2,7- diazaspiro[3.5]nonan-2-yl)pyrimidin-5-yl)oxy)-5-fluoro-N-isopropylbenzamide monocitrate monohydrate salt
    D.3.9.4EV Substance CodeSUB327448
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 6
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSNDX-5613 110 mg tablet
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRevumenib monocitrate monohydrate
    D.3.9.1CAS number 2761046-45-9
    D.3.9.2Current sponsor codeSNDX-5613
    D.3.9.3Other descriptive nametrans N-ethyl-2-((4-(7-((4-(ethylsulfonamido)cyclohexyl)methyl)-2,7- diazaspiro[3.5]nonan-2-yl)pyrimidin-5-yl)oxy)-5-fluoro-N-isopropylbenzamide sesquifumarate
    D.3.9.4EV Substance CodeSUB327448
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number110
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 7
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSNDX-5613 160 mg tablet
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRevumenib monocitrate monohydrate
    D.3.9.1CAS number 2761046-45-9
    D.3.9.2Current sponsor codeSNDX-5613
    D.3.9.3Other descriptive nametrans N-ethyl-2-((4-(7-((4-(ethylsulfonamido)cyclohexyl)methyl)-2,7- diazaspiro[3.5]nonan-2-yl)pyrimidin-5-yl)oxy)-5-fluoro-N-isopropylbenzamide monocitrate monohydrate salt
    D.3.9.4EV Substance CodeSUB327448
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number160
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Relapsed or Refractory Acute Leukemias
    E.1.1.1Medical condition in easily understood language
    Relapsed or Refractory Acute Leukemias
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10024330
    E.1.2Term Leukemia acute
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Phase 1
    The objectives for Phase 1 of this study apply to all arms unless
    otherwise stated in the objective.

    • To determine the safety, tolerability, maximum tolerated dose (MTD),
    and recommended Phase 2 dose (RP2D) of SNDX-5613 in patients with
    relapsed/refractory (R/R) acute leukemia in each of the arms studied.

    • To characterize the pharmacokinetics (PK) parameters of SNDX-5613
    and relevant metabolites in each of the arms studied.

    Phase 2
    • To evaluate short- and long-term safety and tolerability of SNDX-5613.
    • To assess the complete remission (CR)+ complete remission with
    partial hematologic recovery (CRh) rate.

    E.2.2Secondary objectives of the trial
    Phase 1
    To compare the PK parameters of SNDX-613 and relevant metabolites
    when administered in a tablet formulation compared with a capsule
    formulation.

    Phase 2
    • To assess postbaseline transfusion independence.
    • To assess the composite definition of complete remission (CRc) rate
    [CR + CRh + complete remission with incomplete hematologic recovery
    (CRi) + complete remission with incomplete platelet recovery (CRp)].
    • To assess the overall response rate (ORR) (CRc + morphological
    leukemia-free state [MLFS] + partial remission [PR]).
    • To assess the time to response (TTR), duration of response (DOR), and
    event free survival (EFS).
    • To assess overall survival (OS).
    • To characterize PK parameters of SNDX-5613 and relevant
    metabolites.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Diagnosis:
    1. Patients in Phase 1 Arm A and B must have active acute leukemia harboring Lysine (K) methytransferase 2A (KMT2A) rearrangement or Nucleophosmin (NPM1) mutation as defined by the National Comprehensive Cancer Network (NCCN) Guidelines stated in the protocol.
    Patients in Phase 1 Arm C, Arm D, Arm E and Arm F must meet one of the following 2 criteria:
    •active acute leukemia (bone marrow blasts ≥5% or reappearance of blasts in
    peripheral blood) as defined by the NCCN guidelines stated in the protocol.
    •acute leukemia harboring an KMT2A rearrangement, Nucleporin 98 (NUP98) rearrangement, or NPM1 mutation that have detectable disease in the bone marrow not meeting criterion for active leukemia as described above.
    2. Phase 1:
    • Arm A: Patients must not be receiving any strong CYP3A4 inhibitor/inducers or fluconazole. Patients who were receiving a strong CYP3A4 inhibitor/inducer or fluconazole must have discontinued the medication at least 7days prior to enrollment.
    • Arm B: Patients must be receiving itraconazole, ketoconazole, posaconazole, or voriconazole for antifungal prophylaxis for at least 7days prior to enrollment and while on SNDX-5613 treatment. Patients must not be receiving any other strong CYP3A4 inhibitors/inducers.
    • Arm C: Patients must weigh ≥35 kg and be willing to receive daily cobicistat from C1D2 for at least 28 days. Patients must not be receiving any other strong or moderate CYP3A4 inhibitors/inducers. Patients who were receiving a moderate/strong CYP3A4 inhibitor/inducer must have discontinued the medication at least 7days prior to enrollment.
    Arm D: Patients must be receiving fluconazole for at least 7 days prior to enrollment and while on SNDX-5613 treatment. Patients must not be receiving any other strong or moderate CYP3A4 inhibitors/inducers.
    •Arms E: Patients must not be receiving any weak, moderate, or strong
    CYP3A4 inhibitors/inducers for at least 7 days prior to enrollment and
    while on SNDX5613 treatment.
    • Arm F: Patients must be receiving isavuconazole for at least 7 days prior to enrollment and while on SNDX-5613
    treatment. Patients must not be receiving any other weak, moderate, or
    strong CYP3A4 inhibitors/inducers.
    3. Phase 2: Documented R/R acute leukemia:
    Cohort 2A: Documented R/R ALL/MPAL with a KMT2A rearrangement.
    Cohort 2B: Documented R/R AML with a KMT2A rearrangement.
    Cohort 2C: Documented R/R AML with NPM1m.
    Mutational status is to be reviewed locally to determine patient eligibility in Phase 2 and confirmed centrally.
    Disease Status:
    4. Recurrent or refractory AML/ALL or MPAL, as defined by standardized criteria after standard of care therapy. Patients with persistent leukemia after initial therapy or with recurrence of leukemia at any time after achieving a response during or after the course of treatment are eligible. Refractory or relapsed leukemia is defined by presence of >=5% blasts in the bone marrow and/or persistence or reappearance of peripheral blasts. Patients who have <5% blasts in the bone marrow at baseline may be replaced to ensure a sufficient number of patients for the efficacy analyses.
    Age/Weight:
    5. Male or female patients aged >=6 months. Patients intend to receive SNDX-5613 in combination with cobicistat must weigh ≥35 kg. See Protocol Section 10.11.3.1 for age of inclusion criteria in Germany.
    Performance Level:
    6. Eastern Cooperative Oncology Group performance status score 0-2 (if aged ≥18yrs); Karnofsky Performance Scale of ≥50 (if aged ≥16yrs and <18yrs); Lansky Performance Score of ≥50 (if aged >=12 years and <16 years). See Protocol Section 10.11.3.1 for ECOG performance status criteria in Germany.
    Prior Therapy:
    7. Any prior treatment-related toxicities resolved to ≤Grade 1 prior to enrollment, with the exception of ≤Grade 2 neuropathy or alopecia.
    8. Radiation Therapy: At least 60 days from prior total body irradiation (TBI), craniospinal radiation and/or ≥50% radiation of the pelvis, or at least 14 days from local palliative radiation therapy (small port).
    9. Stem Cell Infusion: At least 60 days must have elapsed from hematopoietic stem cell transplant (HSCT) and at least 4 weeks (from first dose) must have elapsed from donor lymphocyte infusion (DLI).
    10. Immunotherapy: At least 42days since prior immunotherapy, including tumor vaccines, and at least 21days since receipt of chimeric antigen receptor therapy or other modified T or Natural Killer (NK) cell therapy.
    11. Antileukemia Therapy: At least 14days or 5 half-lives, whichever is
    shorter since the completion of antileukemic therapy with the exceptions as defined in the protocol.

    See protocol for additional inclusion criteria.
    E.4Principal exclusion criteria
    Diagnosis:
    1. Diagnosis of active acute promyelocytic leukemia.
    2. Isolated extramedullary relapse (Phase 2 only).
    3. Active CNS disease. Refer to the protocol for further details.
    Infection:
    4. Detectable human immunodeficiency virus (HIV) viral load within the previous 6 months. Patients with a known history of HIV 1/2 antibodies
    must have viral load testing prior to study enrollment.
    5. Hepatitis B (defined as hepatitis B virus [HBV] surface antigen positive and HBV core antibody positive, or positive HBV
    deoxyribonucleic acid [DNA].
    6. Hepatitis C (defined as positive hepatitis C [HCV] antibody with reflex to positive HCV ribonucleic acid [RNA]).
    Pregnancy and Breast-Feeding:
    7. Pregnant or nursing women. Negative serum pregnancy tests are required during Screening and a negative serum or urine pregnancy test is required within 72 hours prior to receiving the first study drug administration, in females of childbearing potential. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required.
    Concurrent Conditions:
    8. Cardiac Disease:
    • Any of the following within the 6 months prior to study entry: myocardial infarction, uncontrolled/unstable angina, congestive heart failure, life-threatening, uncontrolled arrhythmia, cerebrovascular accident, or transient ischemic attack.
    • QTc using Fridericia's correction >450 msec.
    9. Gastrointestinal (GI) Disease:
    • Any GI issue of the upper GI tract likely to affect oral drug absorption or ingestion.
    • Cirrhosis with a Child-Pugh score of B or C.
    10. Graft-Versus-Host Disease (GVHD): Signs or symptoms of acute or chronic GVHD >Grade 0 within 4 weeks of enrollment. All transplant
    patients must have been off all systemic immunosuppressive therapy and calcineurin inhibitors for at least 4 weeks prior to enrollment. Patients may be on physiological doses of steroids.
    11. Concurrent malignancy in the previous 2 years, with the exception of basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or
    carcinoma in situ treated with potentially curative therapy, or concurrent low-grade lymphoma, that is asymptomatic and lacks bulky disease and shows no evidence of progression, and for which the patient is not receiving any systemic therapy or radiation.
    12. Concurrent malignancy must be in complete remission or no evidence of disease during this timeframe.
    13. History of or any concurrent condition, therapy, laboratory abnormality, or allergy to excipients that in the Investigator's opinion
    might confound the results of the study, interfere with the patient's participation for the full duration of the study, or is not in the best interest of the patient to participate.
    Concomitant Medications and Interventions:
    14. Any commercially available or investigational antileukemic therapy other than SNDX-5613, with exceptions as defined in the protocol.
    15. Please refer to the protocol for the list of exclusion that apply to related to concomitant use of CYP3A4 inhibitors or inducers (Phase 1 and Phase 2).
    16. Phase 1 and Phase 2: Patients requiring the concurrent use of medications known or suspected to prolong the QT/QTc interval, with the exception of drugs with low risk of QT/QTc prolongation that are used as standard supportive therapies and the azoles permitted in the relevant arms of Phase 1 and in Phase 2. Please see Appendix 10.7 of the protocol for examples of medications that may be appropriate substitutes for such medications.
    Please refer to the protocol for additional exclusion criteria.
    E.5 End points
    E.5.1Primary end point(s)
    Phase 1:
    • Occurrence of dose-limiting toxicities (DLTs).
    • Frequency, duration, and severity of treatment-emergent adverse events (TEAEs), treatment-related TEAEs (TRAEs), and serious adverse events (SAEs).
    • Incidence and shifts of clinically significant clinical laboratory abnormalities.
    • Change from baseline in other observations related to safety, including electrocardiograms (ECGs), vital signs, ophthalmologic examination findings, and performance status.
    • Pharmacokinetic (PK) parameters: maximum plasma concentration (Cmax), time to maximum plasma concentration (Tmax), area under the plasma concentration versus time curve (AUC) from time 0 to t (AUC0–t), AUC from time 0 to 24 hours (AUC0–24), apparent oral clearance (CL/F), apparent volume of distribution (Vz/F), and half-life (t1/2).

    In Phase 1, the study endpoints will be assessed by dose cohort and overall and will apply to all arms unless otherwise stated in the
    endpoint.

    Phase 2:
    • CR+CRh rate.
    • Frequency, duration, and severity of TEAEs, TRAEs, and SAEs.
    • Incidence and shifts of clinically significant clinical laboratory abnormalities.
    • Change from baseline in other observations related to safety, including ECGs, vital signs, ophthalmologic examination findings, and performance status.

    For Phase 2, endpoints will be assessed by disease cohort (2A, 2B, and 2C separately) and pooled MLLr population.
    E.5.1.1Timepoint(s) of evaluation of this end point
    AEs: BL, C1(Day 3/4, 7/8, 10/11, 14/15, 17/18, 21/22), C2(D1,D15), ≥
    C3D1, EoT, f/up
    ECG: SCR, BL, C1(Day 1, 2, 3/4, 7/8, 14/15, 21/22), C2D15, ≥C3D1,
    f/up
    Holter Monitoring (Phase I): BL
    Vital signs: SCR, BL, C1(Day 1, 2, 3/4, 7/8, 10/11, 14/15), C2(D1,D15),
    C3D1, EoT, f/up
    Ophthalmologic exam: SCR, ≥C3D1, EoT, f/up
    Performance Status: SCR, BL, ≥C2D1, C3D1, f/up
    Hematology: SCR, BL, C1(D 3/4, 7/8, 10/11, 14/15, 17/18, 21/22), ≥
    C2D1, C3D1, EoT, f/up
    Clinical Chemistry: SCR, BL, C1(Day 2, 3/4, 7/8, 10/11, 14/15, 17/18,
    21/22); ≥C2D1, C3D1, EoT, f/up
    Urinalysis: C1D1/BL, C2D1, C3D1, f/up
    PK: BL, C1(Day 2, 3/4, 7/8, 14/15), C2 (Day 8, 15), ≥C3D1
    Coagulation panel: SCR
    C=Cycle; D=day; SCR=screening; BL=Baseline; EoT=End of Treatment;
    f/up=Safety follow-up
    E.5.2Secondary end point(s)
    Phase 1:
    Note that no secondary endpoints have been defined for Phase 1.

    Phase 2:
    • Transfusion independence, defined as any transfusion-free period
    lasting for at least 56 consecutive days, during which the patient is
    either on SNDX-5613 therapy or after cessation of SNDX-5613 therapy
    but prior to the start of new therapy.
    • CRc rate (ie, CR+CRh+CRi+CRp).
    • ORR (CRc+MLFS+PR).
    • Time to response.
    • Duration of response.
    • Event free survival.
    • Overall survival.
    • PK parameters: Cmax, Tmax, AUC0–t, AUC0–24, CL/F, Vz/F, and t1/2.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Disease assessments: throughout the duration of the study.
    PK: as above
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned9
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Canada
    Israel
    United States
    France
    Germany
    Italy
    Lithuania
    Netherlands
    Spain
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Last visit of the last subject.
    Note that after the Safety Follow-up visit all patients will be followed for long-term survival status via telephone contact or office visit until death or closure of the study by the Sponsor.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days7
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months5
    E.8.9.2In all countries concerned by the trial days20
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 49
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 29
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 184
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 100
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 65
    F.4.2.2In the whole clinical trial 333
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    No intervention following the completion of study participation is planned.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2023-05-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-06-28
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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