A study investigating the safety and efficacy of academically-produced CAR T cells compared with standard treatment for patients with multiple myeloma whose disease has returned or not responded to previous treatments.

2025-524876-43-00 Therapeutic confirmatory (Phase III) Authorised, recruitment pending

Status Authorised, recruitment pending · 1 EU/EEA countries · 1 sites

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)
Status Authorised, recruitment pending
Participants planned 126
Countries 1
Sites 1

relapsed/refractory multiple myeloma

To compare progression free survival (PFS) of patients randomized to PoC ARI0002h CAR T-cells versus current SoC in patients previously treated with 2-4 lines of therapy

Key facts

Sponsor
Universitair Medisch Centrum Groningen
Participant type
Patients
Age range
18-64 years, 65+ years
Gender
Male and Female
Therapeutic area
Diseases [C] - Neoplasms [C04]
Decision date (initial)
2026-04-29
Transition trial
No
Low-intervention
No
Rare-disease indication
Yes
Vulnerable population
No
Funding sources
Zorginstituut Nederland (ZIN)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To compare progression free survival (PFS) of patients randomized to PoC ARI0002h CAR T-cells versus current SoC in patients previously treated with 2-4 lines of therapy

Secondary objectives 12

  1. To evaluate response rates, i.e. overall response rate (ORR), stringent complete response (sCR), complete remission (CR), very good partial response (VGPR), partial response (PR), minimal residual disease (MRD-) negativity, and sustained MRD-negativity
  2. To correlate MRD -negativity with different efficacy end points
  3. To evaluate duration of response (DOR)
  4. To assess overall survival
  5. To evaluate tolerability, with an emphasis on cytokine release syndrome (CRS), immune effector cell-associated encephalopathy (ICANS), and delayed neurotoxicity;
  6. To evaluate development of secondary malignancies
  7. To evaluate quality of life
  8. To evaluate costs and cost effectiveness of CAR T-cell therapy compared to SoC in the proposed study population.
  9. To evaluate CAR T-cell expansion, persistence, and T-cell characteristics in ARI0002h
  10. To evaluate PoC CAR T-cell production characteristics
  11. To evaluate the association of the functional characteristics of the CAR T-cell products with CAR T-cell expansion and persistence and relate all the previously mentioned items to adverse events, response rates and PFS
  12. To assess the proportion of successful batches.

Conditions and MedDRA coding

relapsed/refractory multiple myeloma

VersionLevelCodeTermSystem organ class
25.0 LLT 10086466 Relapsed/refractory multiple myeloma 100000004848

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

  1. Documented historical diagnosis of Multiple Myeloma
  2. Received 2 to 4 prior lines of antimyeloma therapy, including an IMiD, a PI and an anti-CD38 mAb.
  3. Refractory to the last line of treatment by IMWG criteria
  4. Measurable disease at screening per IMWG criteria
  5. Candidates to receive at least 1 of the 5 SoC regimens (PVd, PCd, EPd, DKd or Kd)
  6. Aged 18 years or older
  7. Capable of giving informed consent
  8. ECOG/WHO performance status of 0-2
  9. Adequate hematological, renal, hepatic, pulmonary, and cardiac function

Exclusion criteria 9

  1. Received one of the following prior therapies: BCMA-targeted therapy, T-cell engager therapy, CAR T-cell therapy, or other genetically modified T-cell therapy
  2. Active or prior history of central nervous system (CNS) or meningeal involvement of MM
  3. Cardiac atrial or cardiac ventricular MM involvement.
  4. History of or active plasma cell leukemia, Waldenstrom’s macroglobulinemia, polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes (POEMS) syndrome, or amyloidosis.
  5. Presence or suspicion of fungal, bacterial, viral, or other infection that is uncontrolled or requiring IV antimicrobials for management.
  6. Females who are pregnant or breastfeeding
  7. Participants who are not willing to practice highly effective birth control
  8. Life expectancy < 12 weeks
  9. Current participation in another clinical trial

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Progression free survival

Secondary endpoints 14

  1. Safety and toxicity of ARI0002h CAR T-cells and SoC
  2. Overall response rate
  3. Overall and sustained MRD-negativity
  4. MRD-negative CR/sCR and MRD-negative VGPR+
  5. Correlation of MRD-negativity with efficacy endpoints
  6. Best overall response
  7. Duration of response
  8. Overall survival from date of randomization
  9. Patient Reported Outcome/Quality of Life
  10. CAR T-cell expansion, persistence, and T-cell characteristics in ARI0002h-treated patients
  11. PoC CAR T-cell production characteristics
  12. The association of the functional characteristics of the CAR T-cell products with CAR T-cell expansion, persistence, AEs, response rates and PFS
  13. Proportion of successful batches
  14. Production and additional health care costs

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Cesnicabtagene autoleucel

PRD10699108 · Product

Active substance
Autologous Genetically Modified T Lymphocytes Transduced with Lentivirus Expressing Car Protein Directed Against Bcma
Pharmaceutical form
DISPERSION FOR INFUSION
Route of administration
INTRAVENOUS
Max daily dose
3000000 Other
Max total dose
6000000 Other
Max treatment duration
100 Week(s)
Authorisation status
Not Authorised
MA holder
FUNDACIÓ DE RECERCA CLINIC BARCELONA-INSTITUT D´INVESTIGACIONS BIOMÈDIQUES AUGUST PI I SUNYER
Paediatric formulation
No
Orphan designation
No

Comparator 11

Bortezomib Accord 3.5 mg powder for solution for injection

PRD3046904 · Product

Active substance
Bortezomib
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS INJECTION
Max daily dose
1.3 mg/m2 milligram(s)/square meter
Max total dose
00 mg/m2 milligram(s)/square meter
Max treatment duration
999 Week(s)
Authorisation status
Authorised
ATC code
L01XX32 — -
Marketing authorisation
EU/1/15/1019/001
MA holder
ACCORD HEALTHCARE S.L.U.
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Kyprolis 30 mg powder for solution for infusion

PRD4301210 · Product

Active substance
Carfilzomib
Pharmaceutical form
SOLUTION FOR INFUSION
Route of administration
INTRAVENOUS ADMINISTRATION
Max daily dose
70 mg/m2 milligram(s)/square meter
Max total dose
0 mg/m2 milligram(s)/square meter
Max treatment duration
999 Week(s)
Authorisation status
Authorised
ATC code
L01XG02 — -
Marketing authorisation
EU/1/15/1060/003
MA holder
AMGEN EUROPE B.V.
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Kyprolis 10 mg powder for solution for infusion

PRD4301209 · Product

Active substance
Carfilzomib
Pharmaceutical form
SOLUTION FOR INFUSION
Route of administration
INTRAVENOUS ADMINISTRATION
Max daily dose
70 mg/m2 milligram(s)/square meter
Max total dose
00 mg/m2 milligram(s)/square meter
Max treatment duration
999 Week(s)
Authorisation status
Authorised
ATC code
L01XG02 — -
Marketing authorisation
EU/1/15/1060/002
MA holder
AMGEN EUROPE B.V.
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Kyprolis 60 mg powder for solution for infusion

PRD3374183 · Product

Active substance
Carfilzomib
Pharmaceutical form
SOLUTION FOR INFUSION
Route of administration
INTRAVENOUS ADMINISTRATION
Max daily dose
70 mg/m2 milligram(s)/square meter
Max total dose
00 mg/m2 milligram(s)/square meter
Max treatment duration
999 Week(s)
Authorisation status
Authorised
ATC code
L01XG02 — -
Marketing authorisation
EU/1/15/1060/001
MA holder
AMGEN EUROPE B.V.
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

ENDOXAN omhulde tablet, omhulde tabletten, 50 mg

PRD352573 · Product

Active substance
Cyclophosphamide Monohydrate
Pharmaceutical form
FILM-COATED TABLET
Route of administration
ORAL
Max daily dose
50 mg milligram(s)
Max total dose
00 mg milligram(s)
Max treatment duration
999 Week(s)
Authorisation status
Authorised
ATC code
L01AA01 — CYCLOPHOSPHAMIDE
Marketing authorisation
RVG 01155
MA holder
BAXTER B.V.
MA country
Netherlands
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Pomalidomide Accord 4 mg hard capsules

PRD11509290 · Product

Active substance
Pomalidomide
Pharmaceutical form
CAPSULE, HARD
Route of administration
ORAL
Max daily dose
4 mg milligram(s)
Max total dose
00 mg milligram(s)
Max treatment duration
999 Week(s)
Authorisation status
Authorised
ATC code
L04AX06 — -
Marketing authorisation
EU/1/24/1831/024
MA holder
ACCORD HEALTHCARE S.L.U.
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Dexamethason Activase 2 mg tabletten

PRD9775508 · Product

Active substance
Dexamethasone
Pharmaceutical form
TABLET
Route of administration
ORAL
Max daily dose
40 mg milligram(s)
Max total dose
00 mg milligram(s)
Max treatment duration
999 Week(s)
Authorisation status
Authorised
ATC code
H02AB02 — DEXAMETHASONE
Marketing authorisation
RVG 127835
MA holder
ACTIVASE PHARMACEUTICALS LIMITED
MA country
Netherlands
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Dexamethason Teva 4 mg, tabletten

PRD626962 · Product

Active substance
Dexamethasone
Substance synonyms
DEXAMETASONE, DEXAMETHASONUM
Pharmaceutical form
TABLET
Route of administration
ORAL
Max daily dose
40 mg milligram(s)
Max total dose
00 mg milligram(s)
Max treatment duration
999 Week(s)
Authorisation status
Authorised
ATC code
H02AB02 — DEXAMETHASONE
Marketing authorisation
RVG 29754
MA holder
TEVA NEDERLAND B.V.
MA country
Netherlands
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Empliciti 300 mg powder for concentrate for solution for infusion.

PRD4073295 · Product

Active substance
Elotuzumab
Substance synonyms
HULUC63, BMS901608
Pharmaceutical form
SOLUTION FOR INFUSION
Route of administration
INTRAVENOUS ADMINISTRATION
Max daily dose
20 mg/kg milligram(s)/kilogram
Max total dose
00 mg/kg milligram(s)/kilogram
Max treatment duration
999 Week(s)
Authorisation status
Authorised
ATC code
L01XC23 — -
Marketing authorisation
EU/1/16/1088/001
MA holder
BRISTOL-MYERS SQUIBB PHARMA EEIG
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Empliciti 400 mg powder for concentrate for solution for infusion.

PRD4073310 · Product

Active substance
Elotuzumab
Substance synonyms
HULUC63, BMS901608
Pharmaceutical form
SOLUTION FOR INFUSION
Route of administration
INTRAVENOUS ADMINISTRATION
Max daily dose
20 mg/kg milligram(s)/kilogram
Max total dose
00 mg/kg milligram(s)/kilogram
Max treatment duration
999 Week(s)
Authorisation status
Authorised
ATC code
L01XC23 — -
Marketing authorisation
EU/1/16/1088/002
MA holder
BRISTOL-MYERS SQUIBB PHARMA EEIG
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

DARZALEX 1800 mg solution for injection

PRD8157849 · Product

Active substance
Daratumumab
Substance synonyms
HuMax-CD38
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS INJECTION
Max daily dose
1800 mg milligram(s)
Max total dose
00 mg milligram(s)
Max treatment duration
999 Week(s)
Authorisation status
Authorised
ATC code
L01FC01 — -
Marketing authorisation
EU/1/16/1101/004
MA holder
JANSSEN-CILAG INTERNATIONAL NV
MA country
Norway
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Universitair Medisch Centrum Groningen

70 Total trials 36 Recruiting 17 Ended
Academic / Non-commercial
Sponsor organisation
Universitair Medisch Centrum Groningen
Address
Hanzeplein 1
City
Groningen
Postcode
9713 GZ
Country
Netherlands

Scientific contact point

Organisation
Universitair Medisch Centrum Groningen
Contact name
Wilfried Roeloffzen

Public contact point

Organisation
Universitair Medisch Centrum Groningen
Contact name
Marleen Breems

Third parties 3

OrganisationCity, countryDuties
Hospital Clinic De Barcelona
ORG-100009329
 Barcelona, Spain Other
Universitair Medisch Centrum Groningen
ORG-100022118
 Groningen, Netherlands Laboratory analysis
Hemato-Oncologie voor Volwassenen Nederland (Hovon) Stichting
ORG-100010258
 Rotterdam, Netherlands On site monitoring, Code 10, Code 12, Code 5, Data management, E-data capture, Code 8

Locations

1 EU/EEA country · 1 investigational sites

By country

CountryMS statusPlanned subjectsSites
Netherlands Authorised, recruitment pending 126 1
Rest of world — 0 —

Investigational sites

Netherlands

1 site · Authorised, recruitment pending
Universitair Medisch Centrum Groningen
Hematology, Hanzeplein 1, 9713 GZ, Groningen

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 20 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) D1 HO183 Protocol 2025-524876-43 Redacted 2.0
Protocol (for publication) D4 HO183 EQ-5D-5L Dutch 1
Protocol (for publication) D4 HO183 EQ-5D-5L ENG 1.3
Protocol (for publication) D4 HO183 QLQ-C30 ENG 3.0
Protocol (for publication) D4 HO183 QLQ-C30 NLD 1
Protocol (for publication) D4 HO183 QLQ-MY20 ENG 0
Protocol (for publication) D4 HO183 QLQ-MY20 NLD 1
Recruitment arrangements (for publication) K1 HO183 Recruitment arrangements NL 1
Subject information and informed consent form (for publication) L1 HO183 SIS and ICF main Redacted 2.0
Subject information and informed consent form (for publication) L1 HO183 SIS and ICF pregnancy Redacted 2.0
Summary of Product Characteristics (SmPC) (for publication) E2 SmPC bortezomib 0
Summary of Product Characteristics (SmPC) (for publication) E2 SmPC darzalex_daratumumab 0
Summary of Product Characteristics (SmPC) (for publication) E2 SmPC dexamethason 2mg 0
Summary of Product Characteristics (SmPC) (for publication) E2 SmPC empliciti_elotuzumab 0
Summary of Product Characteristics (SmPC) (for publication) E2 SmPC endoxan_cyclophosphamide 0
Summary of Product Characteristics (SmPC) (for publication) E2 SmPC kyprolis_carfilzomib 0
Summary of Product Characteristics (SmPC) (for publication) E2 SmPC pomalidomide 0
Summary of Product Characteristics (SmPC) (for publication) SmPC dexamethason 4mg 0
Synopsis of the protocol (for publication) D1 HO183 Protocol synopsis ENG 2025-524876-43-00 1
Synopsis of the protocol (for publication) D1 HO183 Protocol synopsis NLD 2025-524876-43-00 1

Application history

1 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2025-12-19 Netherlands Acceptable with conditions
2026-04-28
 2026-04-29

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