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

Sponsor

Pharmacosmos A/S (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05545202

Collaborator

(none)

150

Enrollment

Arms

Anticipated Duration (Months)

Study Details

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

Condition or Disease	Intervention/Treatment	Phase
Malignant Lymphoma Multiple Myeloma	Other: Pentaisomaltose	Phase 4

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 %

Study Design

Study Type:

Interventional

Anticipated Enrollment :

150 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes 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.

Primary Purpose:

Treatment

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

Anticipated Study Start Date :

Nov 7, 2022

Anticipated Primary Completion Date :

Jul 7, 2023

Anticipated Study Completion Date :

Jul 7, 2023

Arms and Interventions

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.
Other: B: A combination of Pentaisomaltose 10 % and DMSO 2 % 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 %.	Other: Pentaisomaltose Each subject will receive 1 or 2 bags with cryopreserved cells according to randomization group.
Active Comparator: C: 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.

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.

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

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 %.

Other: Pentaisomaltose

Each subject will receive 1 or 2 bags with cryopreserved cells according to randomization group.

Active Comparator: C: 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.

Outcome Measures

Primary Outcome Measures

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
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

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

Pharmacosmos A/S

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Pharmacosmos A/S

ClinicalTrials.gov Identifier:

NCT05545202

Other Study ID Numbers:

P-Pentaisomaltose-CRYO-01

First Posted:

Sep 19, 2022

Last Update Posted:

Sep 19, 2022

Last Verified:

Sep 1, 2022

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Lymphoma

Multiple Myeloma

Neoplasms, Plasma Cell

Study Results

No Results Posted as of Sep 19, 2022