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

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

• Status: Not yet recruiting
• Conditions: Multiple Myeloma; Malignant Lymphoma
• Intervention / Treatment: Other: Pentaisomaltose

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 Type: Interventional
• Enrollment (Estimated): 150
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Pharmacosmos A/S Clinical and non-clinical research; Phone Number: +4559485959; Email: info@pharmacosmos.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Pharmacosmos A/S

Study record dates

Study Major Dates

• Study Start (Estimated): April 30, 2024
• Primary Completion (Estimated): February 15, 2025
• Study Completion (Estimated): February 15, 2025

Study Registration Dates

• First Submitted: September 14, 2022
• First Submitted That Met QC Criteria: September 14, 2022
• First Posted (Actual): September 19, 2022

Study Record Updates

• Last Update Posted (Actual): April 3, 2024
• Last Update Submitted That Met QC Criteria: April 2, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Hematologic Diseases; Hemorrhagic Disorders; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Lymphoma; Multiple Myeloma; Neoplasms, Plasma Cell
• Other Study ID Numbers: P-Pentaisomaltose-CRYO-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No