Study to Evaluate the Safety and Efficacy of ARI0002h, for the Initial Treatment of Patients With Primary Plasma Cell Leukaemia

NCT06830733 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: 2024-515053-21-00
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 7

Brief Summary

Phase II, pilot, open-label, prospective, multicenter, non-randomized study to evaluate the safety and efficacy of ARI0002h (cesnicabtagene autoleucel) in 20 patients with newly diagnosed primary plasma cell leukemia (PCL). The study population is patients between 18 and 75 years of age with newly diagnosed primary plasma cell leukemia (pPCL), with a life expectancy of more than 3 months. The primary objective is to assess the safety and efficacy of CARTBCMA ARI0002h (cesnicabtagene autoleucel) after initial treatment to induce response in patients with newly diagnosed primary plasma cell leukaemia.

Conditions

Leukemia, Plasma Cell

Outcome Measures

Primary Outcomes (2)

• Overall response rate (ORR)

  Overall response rate (ORR) during the initial 3 months after the first infusion (at least presenting a partial response according to the International Myeloma Working Group criteria).

  3 months after the first infusion

• Rate of patients who develop cytokine release syndrome and/or neurological toxicity

  Rate of patients who develop cytokine release syndrome and/or neurological toxicity in the first 30 days after CARTBCMA administration, according to the criteria and grading defined in the international consensus document

  30 days after CARTBCMA administration

Secondary Outcomes (17)

• Duration of response

  From day 28 after infusion to study completion, an average of 24 months

• Response rates

  During the first year after administration

• Complete response rate

  at 3, 6, and 12 months after the first infusion

• Overall response rate

  at 6, and 12 months after the first infusion

• Time to complete response

  through study completion, an average of 24 months

Study Arms (1)

ARI0002h

EXPERIMENTAL

ARI0002 will be administered intravenously in two doses. First dose will b e a fractionated infusion of three doses of the investigational medicinal product (day 0: 10% dose, day 3: 30% dose, day 7: 60% dose), and a second adminsitration as a single dose at 2 months after the first in those patients who have achieved at least a minimal response, have not presented PCL progression after the first infusion or serious complications after the first infusion.

Genetic: ARI0002h

Interventions

ARI0002h GENETIC

* Treatment with ARI0002h cells * Other names: CARTBCMA\_J22.9-h:CD8TM:4-1BB:CD3. Adult differentiated autologous T cells from peripheral blood, expanded and transduced with a lentivirus to express a chimeric antigen receptor with anti-BCMA (TNFRSF17) specificity conjugated to the 4-1BB co-stimulatory domain and the CD3z signalling domain that has been humanized.

ARI0002h

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients between 18 and 75 years old diagnosed with newly diagnosed primary plasma cell leukemia (the presence of 5% or more circulating plasma cells in peripheral blood smears in patients otherwise diagnosed with symptomatic multiple myeloma), according to International Myeloma Working Group (IMWG).
• Disease measurable at diagnosis by monoclonal component in serum or urine, or by free light chains in serum according to the eligibility criteria for clinical trials of the "International Myeloma Working Group".
• ECOG Performance Status from 0 to 2
• Life expectancy greater than 3 months.
• Adequate venous access and absence of contraindications for lymphoapheresis.
• Patients who, after being informed, give their consent by signing the Informed Consent Document.

You may not qualify if:

• No previous treatments, except for induction therapy for primary plasma cell leukemia.
• Administration of any anti-BCMA therapy as part of induction
• Not having achieved at least a minimal response with induction treatment (IMWG criteria)
• Absolute lymphocyte count \<0.1x109/L
• Active immunosuppressive therapy except for prednisone 10 mg/day (or equivalent).
• Any other concomitant neoplasia, unless it has been in complete remission for 3 years or longer, except for non-melanoma skin cancer or completely resected in situ carcinoma.
• Active infection requiring treatment.
• Active HIV, HBV, or HCV infection.
• Uncontrolled medical illness
• Severe organ impairment that meets any of the following criteria: EF\<40%, DLCO \<40%, GFR \<30 ml/min, bilirubin \>3 times the upper limit of normality (unless due to Gilbert syndrome)
• Previous diagnosis of symptomatic AL amyloidosis,
• Pregnant or lactating women. Women of childbearing potential must have a negative pregnancy test at the screening phase.
• Women of childbearing potential, including those whose last menstrual cycle was in the year prior to screening, who are unable or unwilling to use highly effective contraceptive methods\* from the beginning of the study to completion of the study.
• Men who are unable or unwilling to use highly effective contraceptive methods\* from the beginning of the study to completion of the study.
• Contraindication to receive lymphodepletive chemotherapy.

Sponsors & Collaborators

• Fundacion Clinic per a la Recerca Biomédica: lead

Study Sites (7)

Hospital Marqués de Valdecilla

Santander, Cantabria, 39008, Spain

Location

Clínica Universitaria de Navarra

Madrid, Madrid, 28027, Spain

Location

Hospital 12 de Octubre

Madrid, Madrid, 28041, Spain

Location

Hospital Universitario Virgen de la Arrixaca

Murcia, Murcia, 30120, Spain

Location

Clínica Universitaria de Navarra

Pamplona, Navarre, 31008, Spain

Location

Complejo Asistencial Universitario de Salamanca

Salamanca, Salamanca, 37007, Spain

Location

Hospital Clinic Barcelona

Barcelona, 08036, Spain

Location

MeSH Terms

Conditions

Leukemia, Plasma Cell

Condition Hierarchy (Ancestors)

Leukemia; Neoplasms by Histologic Type; Neoplasms; Multiple Myeloma; Neoplasms, Plasma Cell; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases

Central Study Contacts

Carlos Fernandez de Larrea, MD, PhD

CONTACT

+34932775400; cfernan1@clinic.cat

Maria Joyera

CONTACT

+34932775400; joyera@recerca.clinic.cat

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 20, 2024
• First Posted: February 17, 2025
• Study Start: April 1, 2025
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2027
• Last Updated: April 25, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

ES (7)