A clinical gene therapy study with hematopoietic stem cells for the treatment of patients suffering from a plasma cell tumor.

Phase 1

• Conditions: Multiple myeloma in early relapse after intensive front line therapy; MedDRA version: 20.0Level: LLTClassification code 10028228Term: Multiple myelomaSystem Organ Class: 100000004864; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2018-001741-14-IT
• Lead Sponsor: OSPEDALE SAN RAFFAELE

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-06-12
• Study Completion: 2021-11-11
• Last Update Posted: 9 January 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

1. Multiple myeloma patients with early relapse after intensive front-line treatment and disease measurable by serum biomarkers that have obtained at least a VGPR after second-line salvage treatment;
2. Able and willing to provide written informed consent;
3. Able to comply with study protocol and procedures;
4. Male or Female;
5. Age 18-70 years;
6. Fit patients: Performance status scores: ECOG <2 and Karnofsky > 70%; Life expectancy of = 6 months;
7. Adequate cardiac, renal, hepatic and pulmonary functions;
8. Women of child-bearing potential enrolled in the study must have a negative pregnancy test at screening and agree to use acceptable methods of contraception during the trial;
9. Men enrolled in the study with partners who are women of child bearing potential, must be willing to use an acceptable barrier contraceptive method during the trial or have undergone successful vasectomy at least 6 months prior to entry into the study.
Are the trial subjects under 18? no
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 8
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

1. Use of other investigational agents within 4 weeks prior to experimental treatment (within 6 weeks if use of long-acting agents);
2. Severe active viral, bacterial, or fungal infection at eligibility evaluation;
3. Active autoimmune disease or a history of clinically relevant autoimmune manifestations requiring immunosuppressive treatment, i.e. psoriasis, systemic lupus erythematosus, rheumatoid arthritis, vasculitis, immune-mediated peripheral neuropathies;
4. Sarcoidosis requiring active steroid or immunosuppressive treatment;
5. Primary amyloidosis;
6. History of neuropsychiatric illness including severe depression, schizophrenia, bipolar disorders, impaired cognitive function, dementia or suicidal tendency;
7. Neuropathy > grade 2;
8. History of severe cardiovascular disease such as prior stroke, coronary artery disease requiring intervention, unresolved arrhythmias;
9. Malignant neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or family history of familial cancer syndromes;
10. Myelodysplasia, cytogenetic or molecular alterations specifically associated with clonal hematopoiesis of the myeloid lineage, or other serious hematological disorder other than the plasma cell dyscrasia;
11.Other clinical conditions judged by the Investigator non-compatible with the study procedures;
12. Positivity for HIV-1, HIV-2 (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA (or negative HCV RNA but on antiviral treatment) and/or Treponema Pallidum or Mycoplasma active infection;
13. Active alcohol or substance abuse within 6 months of the study;
14. Pregnancy or lactation;
15. Previous allogeneic bone marrow transplantation, kidney or liver transplant, or gene therapy;
16. Prior to conditioning: inability to meet the target mobilization cell number after at least 2 attempts of HSPC collection.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified