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No longer recruiting (closed or complete)Last updated: 7 February 2024

Oral LGH447: This phase I trial is evaluating an oral drug (LGH447) alone and in combination with midostaurin for the treatment of Acute Myeloid Leukaemia or Myelodysplastic SyndromeA Phase I, Multicenter, Open-label Study of Oral LGH447 in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome (Other ID's; CLGH447X2102, 2013-003756-20)

Trial purpose

Medical clipboardCancer treatment

Tumor type

Blood Cancers Haematological

Age

People18 - 75

Trial acronym

Oral LGH447

Clinical summary

Summary

This study will assess the safety and preliminary efficacy of escalating doses of LGH447 in AML and MDS

Conditions

This trial is treating people with Acute Myeloid Leukaemia, Myelodysplasia

Eligibility

Inclusion

-Male or female patients ≥18 years of age who present with one of the following:

LGH447 monotherapy arm

  • Refractory/Relapsed AML following no more than 2 prior therapies, or in previously untreated AML patients who are not candidates for standard therapy.
  • High and very high risk MDS according to the revised International Prognostic Scoring System (rIPSS) who have failed prior therapies, such as azacitidine and decitabine
  • Patients with rIPSS score of > 4.5

LGH447 and midostaurin combination arm

  • Refractory/Relapsed AML following no more than 2 prior therapies, or in previously untreated AML patients who are not candidates for standard therapy. AML patients may have either FLT3 wild type or FLT3-ITD/TKD mutant disease, and FLT3 mutation status needs to be defined at study entry.

    • For AML patients, peripheral blast counts < 50,000 blasts/mm3
    • For MDS patients;
  • Platelet count > 25,000/mm3
  • Neutrophils > 500/mm3
  • Blood transfusions are allowed to maintain clinically adequate hemoglobin and hematocrit levels

    • Patients with active central nervous system (CNS) disease are eligible to participate and may be treated concurrently with intrathecal (or intra Ommaya) chemotherapy
    • Patients who are maintained on prophylactic antibiotics are eligible to participate as long as agents comply with the list of approved concomitant medications
    • Performance status ≤ 2
    • Meet other lab criteria

Exclusion

  • Systemic antineoplastic therapy (including unconjugated therapeutic antibodies and toxin immunoconjugates) or any experimental therapy within 7 days or 5 half-lives, whichever is longer, before the first dose of LGH447 monotherapy or LGH447 in combination with midostaurin
  • Radiotherapy with a wide field of radiation within 28 days or radiotherapy with a limited field of radiation for palliation within 7 days of the first dose of LGH447 monotherapy or LGH447 in combination with midostaurin
  • Patients who received CNS irradiation for meningeal leukemia, except if radiotherapy occurred > 3 months previously
  • Major surgery within 4 weeks before the first dose of LGH447 monotherapy or LGH447 in combination with midostaurin
  • Ongoing therapy with corticosteroids greater than 10 mg of prednisone or its equivalent per day. Inhaled and topical steroids are permitted
  • Patients who are currently receiving hydroxyurea to control peripheral blood leukemic blasts and cannot be discontinued for at least 48 hours prior to obtaining PD biomarkers at screening/baseline and during the study
  • Patients who are currently receiving treatment with prohibited medication and that cannot be discontinued at least one week prior to the start of treatment with LGH447 monotherapy or LGH447 in combination with midostaurin
  • Active infection requiring systemic therapy or other severe infection, including pneumonia, within 2 weeks before the first dose of LGH447 monotherapy or LGH447 in combination with midostaurin
  • Known human immunodeficiency virus (HIV) positive
  • Corrected QT interval (QTc) of > 450 milliseconds (ms) in males and > 470 milliseconds (ms) in females on baseline electrocardiogram (ECG) (using corrected QT interval using Fridericia [QTcF] or local standards).
  • Uncontrolled cardiovascular condition, including ongoing cardiac arrhythmias, congestive heart failure, angina, or myocardial infarction within the past 6 months
  • Pregnant or nursing

Inclusion

  • You have had treatment but your cancer has gotten worse or has not responded to the treatment you have been given.
  • You have had treatment, but your cancer has come back (relapsed or recurrent).

Exclusion

  • You have certain types of non-cancer medical conditions.
  • You have had certain treatments, surgical procedures or drugs.
  • You are currently being treated on a clinical trial.
Message

Clinical trials have complex eligibility criteria, and other criteria may apply for this trial. Ask your doctor about whether this trial could be right for you.

Participating hospitals

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More information

Trial Identifiers

Information on this page is partially produced from ClinicalTrials.gov *. View further details about this trial on the registry via the links below:

Trial sponsor

Novartis Pharmaceuticals

Scientific Title

A Phase I, Multicenter, Open-label Study of Oral LGH447 in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome (Other ID's; CLGH447X2102, 2013-003756-20)

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