Treatment Option for Relapsed or Refractory Multiple Myeloma

The purpose of this study is to provide patients access to their nonconforming idecabtagene vicleucel (ide-cel) as a treatment option for relapsed or refractory multiple myeloma. 

Idecabtagene vicleucel was previously prescribed for you on the basis of its effectiveness when it conforms to Food and Drug Administration (FDA) requirements. You had the blood collection (leukapheresis) procedure and your T cells were collected and genetically modified in a laboratory in order to produce the ide-cel T cells for treatment of your disease. The ide-cel T cells that were produced do not meet all of the criteria to be used as a routine prescription drug, as required by the FDA. 

The study will evaluate the safety and effectiveness of this therapy through the collection of information. The use of nonconforming ide-cel in this research study is investigational, which means it is not commercially approved by the FDA and it must be tested to see if it is a safe and effective treatment for the disease or condition being studied.

In order to participate you must meet the following criteria:

• Subject and/or Legally Authorized Representative (LAR) must understand and voluntarily sign an informed consent form (ICF) prior to any study-related assessments/procedures being conducted.
• Subject has multiple myeloma who was, per the treating physician assessment, eligible for treatment with ide-cel per the approved prescribing information.
• Subject is > 18 years of age at the time of signing the ICF.
• 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.
• Remanufacturing (eg, repeat leukapheresis and manufacturing) is deemed not feasible or clinically inappropriate per assessment of the treating physician in discussion with the subject. The reason for not choosing to remanufacture or to undergo a second leukapheresis must be documented in source documentation and captured on the case report form (CRF).
• 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. The treating physician is advised to contact the Medical Monitor in the event there is any concern regarding administration of LD chemotherapy.
• Females of childbearing potential (FCBP*) must:
  • 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. This applies even if the subject practices true abstinence** from heterosexual contact.
  • Either commit to true abstinence from heterosexual contact or agree to use, and be able to comply with, effective contraception without interruption. Contraception methods must include 1 highly effective method*** from screening until at least 12 months after the LD chemotherapy.
  • Agree to abstain from breastfeeding during study participation and for at least 12 months following LD chemotherapy.
  • There are insufficient exposure data to provide any recommendation concerning the duration of contraception and the abstaining from breastfeeding following treatment with nonconforming ide-cel. Any decision regarding contraception and breastfeeding after infusion should be discussed with the treating physician.
    • * A female subject of childbearing potential (FCBP) is a female who: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 12 consecutive months (ie, has had menses at any time in the preceding 12 consecutive months).
    • ** True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. In contrast, periodic abstinence (eg, calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
    • *** Highly effective methods are defined as those that result in a low failure rate (ie, less than 1% per year) when used consistently and correctly. The following are examples of highly effective methods of contraception: Intrauterine device (IUD), Hormonal (birth control pill, injections, implants), Tubal ligation, Partner's vasectomy.
• Male subjects must:
  • 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.
  • There are insufficient exposure data to provide any recommendation concerning the duration of contraception following treatment with nonconforming ide-cel. Any decision regarding contraception after infusion should be discussed with the treating physician.
    • **True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. In contrast, periodic abstinence (eg, calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
• Subjects 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. There are insufficient exposure data to provide any recommendation concerning the duration of refraining from tissue donation following treatment with nonconforming ide-cel. Therefore, subjects treated with nonconforming ide-cel should not donate blood, organs, tissues, and cells for transplantation.

You will be excluded from the study if any of the following criteria apply to you:

• Subject has a hypersensitivity to the active substance or to any of the excipients.
• 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.
• 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.
• 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.
• Pregnant or nursing women or has intention of becoming pregnant during the study.

This is a partial list of eligibility requirements. To inquire about your eligibility, please call the contact number provided. If you wish to inquire via email, please include the title of the study in your message.