A Randomized, Comparative, Double-blind Trial of Pentaisomaltose and Dimethyl Sulphoxide for Cryoprotection of Hematopoietic Stem Cells in Subjects With Multiple Myeloma or Malignant Lymphoma With a Need for Autologous Transplantation

Study Overview
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Brief Summary
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A randomized, comparative, double-blind trial of pentaisomaltose and dimethyl sulphoxide for cryoprotection of hematopoietic stem cells in subjects with multiple myeloma or malignant lymphoma with a need for autologous transplantation

Detailed Description
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The trial is a randomized, comparative, double-blind trial comparing the efficacy and safety of pentaisomaltose (PentaHibe®, Pharmacosmos A/S, Holbæk; termed PIM in the following) and dimethyl sulphoxide (DMSO), when used for preservation of harvested hematopoietic stem cells. Approximately 150 subjects with a diagnosis of multiple myeloma or malignant lymphoma (a minimum of 30 % of each diagnosis and an equal distribution in each preservation group) and undergoing autologous stem cell transplantation (ASCT) will be included. The subjects will be randomized 1:1 to receive ASCT preserved in the following cryo-protective agent (CPA): A: PIM 16 % B: DMSO 10 %

Official Title
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A Randomized, Comparative, Double-blind Trial of Pentaisomaltose and Dimethyl Sulphoxide for Cryoprotection of Hematopoietic Stem Cells in Subjects With Multiple Myeloma or Malignant Lymphoma With a Need for Autologous Transplantation

Conditions
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Malignant Lymphoma, Multiple Myeloma

Intervention / Treatment
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* Other: Pentaisomaltose


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Men or women ≥ 18 years Multiple myeloma or malignant lymphoma diagnosis in which ASCT is indicated according to the standard procedures at the hospital Willing to be hospitalized for minimum 24 hours after the ASCT Willingness to participate and signing the ICF Exclusion Criteria: Multiple myeloma or lymphoma invasion of the central nervous system Previous treatment with ASCT Severe infection Unsuitable for apheresis Unable to provide ≥3 bags with a cell concentration ≥2.0×106 CD34+ cells/kg bodyweight per bag during apheresis Pregnant or nursing women. To avoid pregnancy, women of childbearing potential have to use adequate contraception (e.g., intrauterine devices, hormonal contraceptives, or double barrier method) during the whole trial period and up to 1 year after the last ASCT Men, even if surgically sterilized, (i.e., status post vasectomy), who do not agree to practice effective barrier contraception during the entire trial period and through 6 months after the last ASCT, or agrees to completely abstain from heterosexual intercourse Any other medical condition which, in the opinion of the Investigator, will put the subject's disease management at risk or may result in the subject being unable to comply with the trial requirements

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: A: Pentaisomaltose 16 %<br>Pentaisomaltose 40 % sterile solution; 40 mL will be transferred to a total volume of 100 mL, so the final concentration is 16 %. | Other: Pentaisomaltose<br>* Each subject will receive 1 or 2 bags with cryopreserved cells according to randomization group.<br>|
| Active Comparator: B: DMSO 10 %<br>Comparator: DMSO 99.9 % sterile solution; 10 mL will be transferred to a total volume of 100 mL, so the final concentration is 10 %. | Other: Pentaisomaltose<br>* Each subject will receive 1 or 2 bags with cryopreserved cells according to randomization group.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Engraftment of hematopoietic stem cells | Incidence of subjects with engraftment of hematopoietic stem cells, defined as 2 consecutive measurements with a neutrophil concentration >0.5×109/L within the first 19 days after ASCT | 19 days | 
| Incidence of subjects with AEs of special interest (limited) | Incidence of subjects with AEs of special interest (limited) occurring within 24 hours after ASCT: headache, nausea, vomiting, bradycardia, or unpleasant odor | 24 hours after ASCT | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of subjects with AEs of special interest (limited) | Incidence of subjects with AEs of special interest (limited): headache, nausea, vomiting, bradycardia, or unpleasant odor | 19 days | 
| Time to engraftment of neutrophil cells | • Time to engraftment of neutrophil cells, defined as 2 consecutive measurements with a neutrophil concentration >0.5×109/L within the first 19 days after ASCT | 19 days |