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

Phase 4

Not yet recruiting

• Conditions: Malignant Lymphoma; Multiple Myeloma
• Interventions: Other: Pentaisomaltose

• Registration Number: NCT05545202
• Lead Sponsor: Pharmacosmos A/S

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) 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 %

Study Timeline

• Study First Submitted: 2022-09-14
• Study First Submitted QC: 2022-09-14
• Study First Posted: 2022-09-19
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-02
• Last Update Posted: 2024-04-03
• Study Start: 2024-04-30
• Primary Completion: 2025-02-15
• Study Completion: 2025-02-15

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 150

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
A: Pentaisomaltose 16 %	Pentaisomaltose	Pentaisomaltose 40 % sterile solution; 40 mL will be transferred to a total volume of 100 mL, so the final concentration is 16 %.
B: DMSO 10 %	Pentaisomaltose	Comparator: DMSO 99.9 % sterile solution; 10 mL will be transferred to a total volume of 100 mL, so the final concentration is 10 %.

Primary Outcome Measures

Name	Time	Method
Incidence of subjects with AEs of special interest (limited)	24 hours after ASCT	Incidence of subjects with AEs of special interest (limited) occurring within 24 hours after ASCT: headache, nausea, vomiting, bradycardia, or unpleasant odor
Engraftment of hematopoietic stem cells	19 days	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

Secondary Outcome Measures

Name	Time	Method
Incidence of subjects with AEs of special interest (limited)	19 days	Incidence of subjects with AEs of special interest (limited): headache, nausea, vomiting, bradycardia, or unpleasant odor
Time to engraftment of neutrophil cells	19 days	• 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