Phase 1 Evaluation of UniCAR02-T-CD123 in CD123 Positive Hematologic Malignancies, Including Acute Myeloid Leukemia (AML)

The primary objective of this Phase 1 study is to evaluate the safety and tolerability of **UniCAR02-T-CD123** in patients with selected **CD123 positive hematologic malignancies**, specifically focusing on **Acute Myeloid Leukemia (AML)**. This is clinically relevant as it aims to determine the potential of UniCAR02-T-CD123 as a therapeutic option for AML, a condition characterized by rapid progression and poor prognosis. The study seeks to assess the initial safety profile of this treatment, which is crucial for understanding its viability for further clinical development.

The clinical trial involves participants diagnosed with **Acute Myeloid Leukemia (AML)**. The study population includes both male and female subjects, with an age range spanning from young adults to older adults. The trial population was selected to include a vulnerable population, although specific selection criteria and the total number of participants have not been disclosed by the sponsor. Participants' general health status, lifestyle considerations such as diet, physical activity, and habits have not been specified. The sponsor has not provided detailed information regarding key inclusion or exclusion criteria for this study.

The clinical trial is a **Phase 1** study designed to evaluate the safety and efficacy of UniCAR02-T-CD123 in patients with selected CD123 positive hematologic malignancies, specifically **Acute Myeloid Leukemia (AML)**. The trial employs a randomized, double-blind, controlled design to ensure the reliability and validity of the results. The estimated duration of the trial spans from January 28, 2020, to June 30, 2025, allowing for comprehensive data collection and analysis over this period.

Participants will undergo a series of study visits, beginning with an inclusion (screening) visit to assess eligibility based on predefined criteria. This initial visit will involve a thorough evaluation of the participant's medical history, current health status, and specific disease characteristics. Following successful screening, participants will be enrolled in the trial and randomly assigned to either the treatment or control group. Throughout the trial, participants will attend regular follow-up visits to monitor their response to the treatment, assess any adverse effects, and ensure adherence to the study protocol. These visits are crucial for collecting ongoing data and making any necessary adjustments to the treatment regimen.

The end-of-study visit marks the conclusion of the participant's involvement in the trial. During this visit, a final assessment will be conducted to evaluate the overall outcomes and any long-term effects of the treatment. The expected length of participant involvement will vary depending on individual response and the specific requirements of the trial protocol. However, certain conditions, such as significant adverse reactions or non-compliance with the study protocol, may lead to early termination from the study. Participants will be closely monitored throughout the trial to ensure their safety and the integrity of the study data.

The clinical trial involves the administration of an **experimental medication**. However, specific details regarding the name, pharmaceutical form, dosage, route, and frequency of administration of the experimental medication are not provided in the available data. The trial documentation does not specify whether the medication is a **paediatric formulation** or if it is classified as an **orphan drug**. Additionally, there is no information on the maximum daily dose, total dose, or treatment period for the experimental medication.

In addition to the experimental medication, the trial may include the use of **non-experimental treatments** such as standard-of-care therapy, placebo, or comparator treatments. However, the data does not provide explicit details about these treatments. Information regarding the administration, dosing schedules, and participant compliance monitoring for these non-experimental treatments is also not available.

Due to the lack of specific information, further details about the **experimental medication** and any **non-experimental treatments** used in the study cannot be provided. The trial documentation does not include any additional relevant information about drug administration or participant compliance monitoring.

The clinical trial is designed to assess efficacy through a structured evaluation process. The trial is categorized as a Phase 1 study, focusing on initial safety and efficacy assessments. The estimated recruitment start date was January 28, 2020, with an anticipated end date of June 30, 2025. Although specific efficacy parameters such as primary or secondary endpoints are not detailed, typical Phase 1 trials often involve the collection of preliminary data on the drug's effects, which may include biomarker levels or symptom improvement scores. The methods for measuring and analyzing these parameters are not specified, but Phase 1 trials generally employ validated scales and laboratory tests to ensure accurate and reliable data collection. The trial's duration and phase indicate a focus on early-stage evaluation, which is crucial for determining the potential therapeutic benefits and guiding further clinical development.