Health TrialsbyHealthPanel

Personalized Autologous Transplant for Multiple Myeloma

ConditionMultiple Myeloma
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorEmory University

About this trial

This phase I trial studies the best dose and side effects of mephalan in treating patients with multiple myeloma who are undergoing stem cell transplant. Chemotherapy drugs, such as mephalan, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This trial uses a new method of dosing that is based on analysis of each individual's blood levels of melphalan after receiving a part of the dose, termed pharmacokinetic analysis. This may help to learn more about how to dose melphalan correctly and which patients are likely to benefit from a personalized dose.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Patient must have the clinical diagnosis of a plasma cell neoplasm requiring treatment per the treating physician using the International Myeloma Working Group (IMWG) and World Health Organization (WHO) criteria as guidelines. This can include extraosseous plasmacytoma, monoclonal immunoglobulin deposition disease, and heavy-chain diseases as these diagnoses, while rare, can be treated in part with autologous transplant

If enrolling in phase A of this protocol, the patient

must have received 2+ lines of therapy as defined by the IMWG; and

Must have estimated glomerular filtration rate (eGFR) by Cockcroft-Gault > 40 mL/min; and

Disqualifiers

Patients known to meet criteria for progressive disease or clinical relapse between screening and planned melphalan infusion day -3

Subject has any of the following cardiac abnormalities

History of clinically significant cardiovascular disease with New York Heart Association class III or IV congestive heart failure or

Severe non-ischemic cardiomyopathy or

Trial design

Design model

Single group

Treatments tested in this trial

  • Autologous Hematopoietic Stem Cell Transplantation

    Procedure/Surgery

    Undergo ASCT

  • Melphalan

    Drug

    Given IV

  • Questionnaire Administration

    Other intervention

    Ancillary studies

Treatment groups

90 Participants
are divided into 1 treatment group
Group A: Treatment (Melphalan-based autologous transplant)Experimental treatment 3 interventions

Trial outcomes

Primary outcomes

1

Predicted versus observed total melphalan area under the curve (AUC)

Will be measured using pharmacokinetic data from the day -3 and day -1 dose.

Time frame
At Days -3 and -1
2

Incidence of exposure limiting toxicity

Will measure proportion of patients with clinically significant mucositis, tachyarrhythmia, and delayed engraftment. Toxicity will be evaluated according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) guidelines.

Time frame
During the first 30-days of treatment
3

Minimal residual disease (MRD) negativity post-transplant

MRD status will be determined per International Myeloma Working Group (IMWG) criteria using next generation sequencing in patients with suspected complete response (CR).

Time frame
Up to 90 days post-transplant

Secondary outcomes

1

Overall response rate

The number and percentage of subjects experiencing objective response will be descriptively summarized overall and by cohort. Responders will be defined as those that achieve best response of partial response (PR) or better defined according to the IMWG Uniform response criterion. CR rate will be calculated with an exact 95% confidence interval, both within cohorts but not between cohorts.

Time frame
Up to 90 days post transplantation
2

Incidence of selected grade 3/4 toxicities at the recommended AUC range

The maximum grade for each type of toxicity will be recorded for each patient, and frequency tables will be reviewed to determine toxicity patterns. Adverse events will be summarized and described within each cohort. They will initially be reviewed regardless of attribution, but also whether they are possibly, probably, or definitely related to treatment. In addition, will review all adverse event data that are graded as 3, 4, or 5 and classified as either "unrelated" or "unlikely to be related" to study treatment in the event of an actual relationship developing. The incidence of severe adverse events or toxicities will be described. Will assess the proportion of patients who experience grade 3 or higher non-hematologic toxicity. To assess tolerability, will also capture the proportion of patients who go off treatment due to adverse events.

Time frame
Up to 60 days post transplantation

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Emory University

Lead sponsor

National Cancer Institute (NCI)

Collaborator

Gateway for Cancer Research

Collaborator

Source ClinicalTrials.gov
Apply now