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Melphalan Hydrochloride

Clinical trials are studying Melphalan Hydrochloride in different cancers, including adult Philadelphia chromosome-positive acute lymphoblastic leukemia and retinoblastoma. These studies look at how well treatment works, how safe and effective it is, and what outcomes matter most for patients, such as survival, eye preservation, and visual function.

Table of Contents

Trial overview

The trial data shows two interventional studies that include Melphalan Hydrochloride as part of the treatment plan.[1][2] One study is in adult Philadelphia chromosome-positive acute lymphoblastic leukemia, and the other is in ocular conservative treatment for retinoblastoma.[1][2] Both studies are authorised.[1][2]

Adult Ph+ ALL study

NCT04722848 is a Phase 3 study in adults with Philadelphia chromosome-positive acute lymphoblastic leukemia, also written as Ph+ ALL.[1] The study title says it is testing whether a sequence based on Ponatinib plus Blinatumomab works better than chemotherapy with Imatinib.[1] Melphalan Hydrochloride is listed among the drugs used in the chemotherapy part of the trial.[1]

The study includes adults aged 18 years and older, with no upper age limit.[1] The brief summary says the trial is looking at a front-line chemo-free strategy with Ponatinib plus steroids for induction, followed by Blinatumomab for consolidation, and comparing it with a chemotherapy scheme combined with Imatinib.[1] In simple terms, the researchers want to know which treatment path gives better results for newly treated adults with this leukemia.[1]

Retinoblastoma study

NCT04681417 is a Phase 2 study of conservative treatment for retinoblastoma, which is an eye cancer.[2] The study looks at the effectiveness of new management strategies and visual outcome.[2] Melphalan Hydrochloride is listed in the investigational arm and in the reference arm as part of eye-directed treatment.[2]

The brief summary says Study 1 evaluates local control of the disease in patients eligible for intra-arterial chemotherapy, often shortened to IAC.[2] Study 2 looks at visual function in patients eligible for other conservative treatments, including intravenous chemotherapy with local eye treatments or local treatment without intravenous chemotherapy.[2] This means the trial is focused on saving the eye and keeping vision as good as possible.[2]

Main outcomes and endpoints

The leukemia trial uses event-free survival, or EFS, as the primary endpoint.[1] The trial defines events as not reaching MRD negativity, death from any cause, toxicity, and resistance, including resistance linked to ABL1 mutation development.[1] MRD negativity means no measurable minimal residual disease, which is the very small amount of cancer that may remain after treatment.[1]

The retinoblastoma trial has two main outcomes.[2] The first is eye preservation 24 months after randomization in the investigational and reference arms.[2] The second is the percentage of patients with major, mild, or no visual impairment according to WHO criteria when the child reaches 6 years of age and has at least 24 months of follow-up.[2]

Who can participate

The leukemia study is for adults with Ph+ ALL, and the source states there is no upper age limit.[1] The retinoblastoma study is for patients eligible for conservative eye treatment, including those who can receive intra-arterial chemotherapy or other eye-sparing approaches.[2] These are different patient groups, so the trial goals are also different.[1][2]

Trial phases and study design

NCT04722848 is an interventional Phase 3 trial with 236 planned participants.[1] Phase 3 studies usually compare treatment strategies in larger groups to see which works better.[1] NCT04681417 is an interventional Phase 2 trial with 225 planned participants.[2] Phase 2 studies usually focus more closely on how well a treatment works and on key patient outcomes.[2]

In the leukemia study, the design is a comparison between a Ponatinib plus Blinatumomab strategy and a chemotherapy plus Imatinib strategy.[1] In the retinoblastoma study, the design includes an investigational arm and a reference arm, with one part of the study being randomized.[2] These designs help researchers compare outcomes in a fair way.[1][2]

Trial ID Phase Condition studied Status Enrollment
NCT04722848 Phase 3 Adult Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia Authorised 236
NCT04681417 Phase 2 Ocular conservative treatment for retinoblastoma Authorised 225

FAQ

  • What conditions are being studied with Melphalan Hydrochloride? The trial data includes adult Philadelphia chromosome-positive acute lymphoblastic leukemia and ocular conservative treatment for retinoblastoma. These are very different cancers, so the studies are testing Melphalan Hydrochloride in different patient groups.
  • Who can take part in these trials? One study includes adults with Ph+ acute lymphoblastic leukemia, with no upper age limit. Another study includes patients eligible for conservative eye treatment for retinoblastoma.
  • What phases are these trials? One trial is Phase 3, which usually compares treatments in a larger group of patients. The other is Phase 2, which often looks more closely at how well a treatment works and how it affects patients.
  • What is being measured in the trials? The leukemia trial measures event-free survival, which means how long patients stay free from key problems such as not reaching deep remission, death, toxicity, or resistance. The retinoblastoma trial measures eye preservation and visual function.
  • Are these trials testing Melphalan Hydrochloride alone? No. In the leukemia trial, Melphalan Hydrochloride is listed among several drugs in a chemotherapy plan. In the retinoblastoma trial, it is compared as part of eye-directed treatment strategies.

Ongoing Clinical Trials on Melphalan Hydrochloride

  • Study on Retinoblastoma Treatment: Evaluating the Effectiveness of Topotecan, Vincristine Sulfate, and Etoposide in Patients Eligible for Conservative Therapy

    Recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    France

  • Study on Ponatinib and Blinatumomab for Adults with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

Glossary

  • Acute Lymphoblastic Leukemia (ALL): A fast-growing cancer of the blood and bone marrow. The trial focuses on the adult form with a specific chromosome change.
  • Philadelphia chromosome-positive (Ph+): A genetic change found in some leukemia cells. It helps doctors identify a specific type of disease for study.
  • Retinoblastoma: A cancer of the eye that usually affects children. The trial studies eye-saving treatment approaches.
  • Interventional study: A clinical trial where researchers assign a treatment or strategy and then measure the results.
  • Phase 2: An earlier trial phase that looks at how well a treatment works and gathers more information about its effects.
  • Phase 3: A later trial phase that compares treatments in larger groups of patients.
  • Event-free survival (EFS): The length of time a patient remains free from important treatment problems or disease events.
  • Minimal residual disease (MRD): Very small amounts of cancer that may remain after treatment and are hard to detect.
  • MRD negativity: A test result showing no detectable minimal residual disease.
  • Eye preservation: Keeping the eye from being removed or lost because of disease.
  • Visual function: How well a person can see and use vision in daily life.
  • Randomization: A method of assigning patients to different treatment groups by chance.

References