Description
The purpose of this guidance is to assist sponsors in the clinical development of drugs and biological products for the treatment of acute myeloid leukemia (AML). Specifically, this guidance addresses FDA’s current thinking regarding the overall development program and clinical trial designs for the development of drugs to support an indication of treatment of AML, including indications limited to an individual phase of treatment (e.g., maintenance, transplantation preparative regimen, etc.).
Scope & Applicability
Product Classes
5Requires analytical comparability per ICH Q5E
DLT criteria for chimeric antigen receptor T cells
A type of ATMP involving recombinant nucleic acids or viral vectors.
sponsors should consult specific guidances for these products
sponsors should consult specific guidances for these products
Stakeholders
3Entity responsible for submitting applications under section 524B
Possible to conduct the FIH trial in healthy volunteers
Responsible for qualifications, training, and trial conduct; Individual responsible for trial conduct and data governance at a site.; May delegate tasks but retains overall responsibility; Person responsible for the conduct of the clinical trial at a trial site; Responsible for trial conduct and participant safety; Responsible for trial conduct, data integrity, and investigational product management.; Individual responsible for trial conduct at a site and informing the institution.; maintaining
Regulatory Context
Attributes
10Status that does not exempt a drug from pediatric investigation requirements under certain conditions.
Baseline status for transfusion independence assessment.
Baseline status for transfusion independence assessment.
Marrow blasts percentage used for CR response
Lower doses of cytotoxic chemotherapy or targeted drugs with limited organ toxicities.
Regimens expected to cause high-grade organ toxicity or prolonged neutropenia.
Durable CR support for approval depends on the benefit-risk ratio.
Efficacy endpoint (EFS)
Efficacy endpoint (OS)
DLT criteria for trial safety
Identified Hazards
Hazards
1A toxicity (CRS) associated with CAR T cells.
Related CFR Sections (2)
- 21CFR312.32§ 312.32 IND safety reporting.
(a) Definitions. The following definitions of terms apply to this section:Read full regulation →
- 21CFR312.64§ 312.64 Investigator reports.
(a) Progress reports. The investigator shall furnish all reports to the sponsor of the drug who is responsible for collecting and evaluating the results obtained. The sponsor is required under § 312.33 to submit annual reports to FDA on the progress of the clinical investigations.Read full regulation →
See Also (8)
- Safety Reporting Requirements for INDs and BA/BE Studies: Guidance for Industry and Investigators (Status: Final)
- Specifications for Preparing and Submitting Electronic ICSRs and ICSR Attachments (Status: Final)
- Acute Myeloid Leukemia: Developing Drugs and Biological Products for Treatment: Draft Guidance for Industry (Status: Draft)
- Sponsor Responsibilities - Safety Reporting Requirements and Safety Assessment for IND and Bioavailability/Bioequivalence Studies: Draft Guidance for Industry (Status: Draft)
- Electronic Submission of IND Safety Reports Technical Conformance Guide : Guidance for Industry (Status: Final)
- FDA Regional Implementation Guide for E2B(R3) Electronic Transmission of Individual Case Safety Reports for Drug and Biological Products (Status: Final)
- Safety Reporting Requirements for INDs (Investigational New Drug Applications) and BA/BE (Bioavailability/Bioequivalence) Studies: Guidance for Industry and Investigators (Status: Final)
- Assessment of Abuse Potential of Drugs (Status: Final)