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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

Primary Purpose

Malignant Lymphoma, Multiple Myeloma

Status
Not yet recruiting
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
Pentaisomaltose
Sponsored by
Pharmacosmos A/S
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Malignant Lymphoma

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm Type

    Other

    Other

    Active Comparator

    Arm Label

    A: Pentaisomaltose 16 %

    B: A combination of Pentaisomaltose 10 % and DMSO 2 %

    C: DMSO 10 %

    Arm Description

    Pentaisomaltose 40 % sterile solution; 40 mL will be transferred to a total volume of 100 mL, so the final concentration is 16 %.

    Combination of IMP and comparator: Pentaisomaltose 40 % sterile solution; 25 mL will be transferred to a total volume of 100 mL, so the final concentration is 10 %. DMSO 99.9 % sterile solution; 2 mL will be transferred to a total volume of 100 mL, so the final concentration is 2 %.

    Comparator: DMSO 99.9 % sterile solution; 10 mL will be transferred to a total volume of 100 mL, so the final concentration is 10 %.

    Outcomes

    Primary Outcome Measures

    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
    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

    Secondary Outcome Measures

    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
    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

    Full Information

    First Posted
    September 14, 2022
    Last Updated
    March 28, 2023
    Sponsor
    Pharmacosmos A/S
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05545202
    Brief Title
    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
    Official Title
    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 Type
    Interventional

    2. Study Status

    Record Verification Date
    March 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    May 15, 2023 (Anticipated)
    Primary Completion Date
    October 15, 2023 (Anticipated)
    Study Completion Date
    October 15, 2023 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Pharmacosmos A/S

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    Yes

    5. Study Description

    Brief Summary
    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
    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), dimethyl sulphoxide (DMSO), and a combination of both, 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 1:1:1 to receive ASCT preserved in the following cryo-protective agent (CPA): A: PIM 16 % B: A combination of PIM 10 % and DMSO 2 % C: DMSO 10 %

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Malignant Lymphoma, Multiple Myeloma

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 4
    Interventional Study Model
    Parallel Assignment
    Masking
    ParticipantCare ProviderInvestigatorOutcomes Assessor
    Masking Description
    The subject, trial personnel, and Pharmacosmos personnel will be blinded to the CPA. Blinding will be obtained by shielding the blinded trial personnel from seeing the preparation of the CPA and having unblinded personnel not involved in any trial assessments (efficacy or safety) responsible for randomization, preparation, and accountability of the CPA. The CPA will not be labelled in a way that would reveal the content of the CPA for the blinded personnel or the subject. CPA accountability will be monitored by an unblinded CRA.
    Allocation
    Randomized
    Enrollment
    150 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    A: Pentaisomaltose 16 %
    Arm Type
    Other
    Arm Description
    Pentaisomaltose 40 % sterile solution; 40 mL will be transferred to a total volume of 100 mL, so the final concentration is 16 %.
    Arm Title
    B: A combination of Pentaisomaltose 10 % and DMSO 2 %
    Arm Type
    Other
    Arm Description
    Combination of IMP and comparator: Pentaisomaltose 40 % sterile solution; 25 mL will be transferred to a total volume of 100 mL, so the final concentration is 10 %. DMSO 99.9 % sterile solution; 2 mL will be transferred to a total volume of 100 mL, so the final concentration is 2 %.
    Arm Title
    C: DMSO 10 %
    Arm Type
    Active Comparator
    Arm Description
    Comparator: DMSO 99.9 % sterile solution; 10 mL will be transferred to a total volume of 100 mL, so the final concentration is 10 %.
    Intervention Type
    Other
    Intervention Name(s)
    Pentaisomaltose
    Intervention Description
    Each subject will receive 1 or 2 bags with cryopreserved cells according to randomization group.
    Primary Outcome Measure Information:
    Title
    Engraftment of hematopoietic stem cells
    Description
    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
    Time Frame
    19 days
    Title
    Incidence of subjects with AEs of special interest (limited)
    Description
    Incidence of subjects with AEs of special interest (limited) occurring within 24 hours after ASCT: headache, nausea, vomiting, bradycardia, or unpleasant odor
    Time Frame
    24 hours after ASCT
    Secondary Outcome Measure Information:
    Title
    Incidence of subjects with AEs of special interest (limited)
    Description
    Incidence of subjects with AEs of special interest (limited): headache, nausea, vomiting, bradycardia, or unpleasant odor
    Time Frame
    19 days
    Title
    Time to engraftment of neutrophil cells
    Description
    • 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
    Time Frame
    19 days

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Pharmacosmos A/S Clinical and Non-clinical Research
    Phone
    +4559485959
    Email
    info@pharmacosmos.com

    12. IPD Sharing Statement

    Learn more about this trial

    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

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