EXPANDED ACCESS PROTOCOL (EAP) FOR SUBJECTS RECEIVING IDECABTAGENE VICLEUCEL THAT IS NONCONFORMING FOR COMMERCIAL RELEASE

JRCT ID: jRCT2053220006

Registered date:22/04/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Adult subjects with relapsed or refractory multiple myeloma eligible for treatment with ide-cel per
• Date of first enrollment: 01/05/2022
• Target sample size: 16
• Countries of recruitment: United States of America,Japan,Canada,Japan
• Study type: Interventional

Intervention(s)

In accordance with the ide-cel prescribing information, local routine care practices and as per the treating physician's discretion, administer the lymphodepleting chemotherapy regimen: cyclophosphamide 300 mg/m2 intravenously (IV) and fludarabine 30 mg/m2 IV for 3 days.
Nonconforming ide-cel is to be administered 2 days after completion of LD chemotherapy.

Outcome(s)

Primary Outcome

[Safety]Type, frequency, and severity of adverse
events (AEs) and laboratory abnormalities; Up to 3 months post date of infusion of nonconforming ide-cel

Secondary Outcome

[Efficacy, Overall Response Rate (ORR)]The percentage of subjects with a best overall response (BOR) of either stringent complete response (sCR), complete response (CR), very good partial response (VGPR) or partial response (PR) from the time of the final nonconforming ide-cel infusion until disease progression, end of study, the start of another anticancer therapy or hematopoietic stem cell transplantation (HSCT); Up to 3 months post date of infusion of nonconforming ide-cel.
[Efficacy, Complete Response (CR) Rate]The percentage of subjects with at least a CR following nonconforming ide-cel
infusion until disease progression, end of study, the start of another anticancer therapy or HSCT; Up to 3 months post
date of infusion of nonconforming ide-cel

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.Subject and/or LAR must understand and voluntarily sign an ICF prior to any study related assessments/procedures being conducted.
2. Subject has multiple myeloma who was, per the treating physician assessment, eligible for treatment with ide-cel per the approved prescribing information.
3. Subject is >= 18 years of age at the time of signing the ICF.
4. Subject had a subject-specific batch of ide-cel manufactured intended for commercial treatment; however, the final manufactured product was nonconforming and did not meet commercial release criteria. The Sponsor Internal Review Process has determined that the nonconforming ide-cel may be released for use under the EAP.
5. Remanufacturing (eg, repeat leukapheresis and manufacturing) is deemed not feasible or clinically inappropriate per assessment of the treating physician in discussion with the subject.
6. Subject is clinically stable, has recovered from any prior toxicities prior to receiving LD chemotherapy, and has adequate bone marrow function to receive LD chemotherapy.
7. Females of childbearing potential must:
a. Have a negative pregnancy test as verified by the treating physician within 7 days prior to the first dose of LD chemotherapy following institutional testing methodology practices.
b. Either commit to true abstinence from heterosexual contact or agree to use, and be able to comply with, effective contraception without interruption.
c. Agree to abstain from breastfeeding during study participation and for at least 12 months following LD chemotherapy.
8. Male subjects must:
a. Practice true abstinence or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential for at least 12 months after LD
chemotherapy even if the subject has undergone a successful vasectomy.
b. Subject must agree to not donate blood, organs, tissue, sperm or semen and egg cells for usage in other individuals for at least 1 year following LD chemotherapy.

Exclude criteria

1. Subject has a hypersensitivity to the active substance or to any of the excipients.
2. Subject should not experience a significant worsening in clinical status that would, in the opinion of the treating physician, either increase the risk of AEs associated with LD
chemotherapy, or exclude them from treatment with nonconforming ide-cel.
3. Subject has any significant medical condition, laboratory abnormality, or psychiatric illness, sociologic or geographic condition that would prevent the subject from participating in the EAP, complying with protocol requirements or confound the ability to interpret the data in the Investigator's judgement.
4. Subject has any condition and/or laboratory abnormality that places the subject at unacceptable risk if he/she were to participate in the EAP based on the Investigator's judgement.

Related Information

• Primary Sponsor: Hayakawa Jin
• Secondary ID(s): NCT04771078