Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
65
Countries
1
Sites
8
Newly Diagnosed Multiple Myeloma
To characterize the efficacy of cilta-cel infusion following a fludarabine free LD regimen (Cohort A) or a low-dose cilta-cel infusion following a standard cyclophosphamide and fludarabine LD regimen (Cohort B)
Key facts
- Sponsor
- Janssen Cilag International
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Neoplasms [C04]
- Trial duration
- 3 Oct 2025 → ongoing
- Decision date (initial)
- 2025-08-20
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
- Funding sources
- Janssen Research & Development, LLC
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Pharmacokinetic, Safety, Dose response
To characterize the efficacy of cilta-cel infusion following a fludarabine free LD regimen
(Cohort A) or a low-dose cilta-cel infusion following a standard cyclophosphamide and fludarabine LD regimen (Cohort B)
Conditions and MedDRA coding
Newly Diagnosed Multiple Myeloma
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.0 | LLT | 10028228 | Multiple myeloma | 10029104 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 6
- 1. At the time of informed consent, be ≥18 years of age (or at least the legal age of consent in the jurisdiction in which the study is taking place) but ≤80 years of age.
- 2. Documented diagnosis of NDMM according to the most recent IMWG diagnostic criteria and measurable disease at diagnosis (prior to start of any anti-myeloma therapy)
- 3. Not considered a candidate for high-dose chemotherapy with stem cell transplantation due to: -Advanced age; or -Presence of comorbid condition(s) likely to have a negative impact on tolerability of high-dose chemotherapy with stem cell transplantation; or -Participant refusal of high-dose chemotherapy with stem cell transplantation as initial treatment.
- 4. Participant must have received induction therapy. Initially, only participants receiving triplet induction therapy with DRd or VRd will be enrolled. Only after sponsor notification, participants receiving quadruplet DVRd induction therapy may be enrolled.Participants must have achieved ≥PR to be enrolled on the most recent disease assessment.
- 5. ECOG Performance Status score of 0 or 1
- 6. Clinical laboratory values as defined in the protocol
Exclusion criteria 6
- 1. Prior systemic therapy for multiple myeloma or smoldering myeloma other than VRd, DRd or DVRd as per inclusion 4a and short course of corticosteroids . For participants who require emergency treatment, refer to protocol Section 4.4.
- 2. Radiation therapy for treatment of plasmacytoma within 14 days before enrollment. Palliative radiation for pain control secondary to lytic lesion is allowed within 14 days of enrollment, if the radiation portal covered ≤5% of the bone marrow reserve
- 3. Frailty index of ≥ 2 according to Myeloma Geriatric Assessment score
- 4. The following cardiac conditions: - New York Heart Association stage III or IV congestive heart failure. - Myocardial infarction or coronary artery bypass graft ≤6 months prior to enrollment. - History of clinically significant ventricular arrhythmia or unexplained syncope, not believed to be vasovagal in nature or due to dehydration. - History of severe non-ischemic cardiomyopathy. - Impaired cardiac function (LVEF <45%) as assessed by echocardiogram or multiple-gated acquisition scan (performed ≤60 days prior to apheresis).
- 5. Grade 2 or higher ongoing non-hematologic toxicity due to induction therapy, with the exception of grade 2 peripheral neuropathy due to bortezomib.
- 6. Participants who require continuous supplemental oxygen.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- MRD-negative CR at 12 months after cilta-cel infusion with a sensitivity of 10^-5
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 2
CARVYKTI 3.2 × 10^6 – 1 × 10^8 cells dispersion for infusion
PRD9718535 · Product
- Active substance
- Ciltacabtagene Autoleucel
- Substance synonyms
- LCAR-B38M CAR-T cells, AUTOLOGOUS BI-EPITOPE BCMA-TARGETED CAR T-CELLS JNJ-68284528, LCAR-B38M-TRANSDUCED CAR-T CELLS JNJ-68284528, AUTOLOGOUS HUMAN T CELLS GENETICALLY MODIFIED EX-VIVO WITH A LENTIVIRAL VECTOR ENCODING A CHIMERIC ANTIGEN RECEPTOR FOR B-CELL MATURATION ANTIGEN, Autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a CAR for BCMA, JNJ-68284528, LCAR-B38M
- Pharmaceutical form
- DISPERSION FOR INFUSION
- Route of administration
- INTRAVENOUS USE
- Max daily dose
- 0 Kg kilogram(s)
- Max total dose
- 0 Kg kilogram(s)
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Authorised
- ATC code
- L01XL05 — -
- Marketing authorisation
- EU/1/22/1648/001
- MA holder
- JANSSEN-CILAG INTERNATIONAL NV
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/20/2252
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- The clinical trial will be conducted with "clinical" material. The lentiviral vector (LV) and drug product (DP) are produced at the commercial sites using the same manufacturing process and the same compendial and non-compendial raw materials compared to the authorized product.
PRD12658204 · Product
- Active substance
- Ciltacabtagene Autoleucel
- Substance synonyms
- LCAR-B38M CAR-T cells, AUTOLOGOUS BI-EPITOPE BCMA-TARGETED CAR T-CELLS JNJ-68284528, LCAR-B38M-TRANSDUCED CAR-T CELLS JNJ-68284528, AUTOLOGOUS HUMAN T CELLS GENETICALLY MODIFIED EX-VIVO WITH A LENTIVIRAL VECTOR ENCODING A CHIMERIC ANTIGEN RECEPTOR FOR B-CELL MATURATION ANTIGEN, Autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a CAR for BCMA, JNJ-68284528, LCAR-B38M
- Pharmaceutical form
- DISPERSION FOR INFUSION
- Route of administration
- INTRAVENOUS USE
- Max daily dose
- 0 kg kilogram(s)
- Max total dose
- 0 kg kilogram(s)
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/20/2252
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Janssen Cilag International
- Sponsor organisation
- Janssen Cilag International
- Address
- Turnhoutseweg 30
- City
- Beerse
- Postcode
- 2340
- Country
- Belgium
Scientific contact point
- Organisation
- Janssen Cilag International
- Contact name
- CTIS Point of Contact
Public contact point
- Organisation
- Janssen Cilag International
- Contact name
- CTIS Point of Contact
Third parties 7
| Organisation | City, country | Duties |
|---|---|---|
| Labcorp Central Laboratory Services SARL ORG-100011524
|
Meyrin, Switzerland | Laboratory analysis |
| Ancillare LP ORG-100044089
|
Horsham, United States | Other |
| Cerba Research ORG-100042694
|
Gent, Belgium | Laboratory analysis |
| CMT Cellex Manufacturing Transports and Logistics GmbH ORG-100026399
|
Cologne, Germany | Laboratory analysis |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | Data management |
| 4g Clinical LLC ORG-100042775
|
Wellesley, United States | Interactive response technologies (IRT) |
| CellCarta ORG-100039881
|
Antwerp, Belgium | Laboratory analysis |
Locations
1 EU/EEA country · 8 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Ongoing, recruiting | 33 | 8 |
| Rest of world
Australia, United States
|
— | 32 | — |
Investigational sites
Hospital Germans Trias I Pujol
Hematologia, Carretera Canyet 1a Planta, 08916, Badalona
University Hospital Virgen Del Rocio S.L.
Hematologia, Avenida De Manuel Siurot S/n, 41013, Sevilla
Hospital Universitario 12 De Octubre
Hematologia, Avenida De Cordoba Sn, 28041, Madrid
Clinica Universidad De Navarra
Hematologia, Pio XII Etorbidea 36, 31008, Pamplona
Hospital Universitario Marques De Valdecilla
Hematologia, Avenida Valdecilla Sn, 39008, Santander
Hospital Clinic De Barcelona
Hematologia, Calle Villarroel 170, 08036, Barcelona
Hospital Universitario De Salamanca
Hematologia, Paseo De San Vicente 58-182, 37007, Salamanca
Hospital Universitari Vall D Hebron
Hematologia, Passeig De La Vall D'Hebron 119-129, 08035, Barcelona
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Spain | 2025-10-03 | 2025-10-03 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 12 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | REDACTED_D1_Protocol_2025-521975-30 | Am1 |
| Protocol (for publication) | REDACTED_D4_PF Temp Diary_ES_ENG_2025-521975-30 | 1 |
| Protocol (for publication) | REDACTED_D4_PF Temp diary_ES_SPA_2025-521975-30 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K1_Recruitment Arrangements_ES_ENG_2025-521975-30 | 1 |
| Recruitment arrangements (for publication) | REDACTED_K2_Recruitment material_Recruitment Brochure_ES_SPA_2025-521975-30 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Addendum 1_Conditioning Therapy_ES_SPA_2025-521975-30 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Addendum 1_OOS_ES_SPA_2025-521975-30 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Clinical Cohorte A_ES_SPA_2025-521975-30 | 2 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Clinical Cohorte B_ES_SPA_2025-521975-30 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L1_SIS and ICF Pregnancy_ES_SPA_2025-521975-30 | 1 |
| Subject information and informed consent form (for publication) | REDACTED_L2_Subject Wallet Card_ES_SPA_2025-521975-30 | 2 |
| Synopsis of the protocol (for publication) | REDACTED_D1_Protocol Synopsis_ES_SPA_2025-521975-30 | Am1 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-06-02 | Spain | Acceptable 2025-08-18
|
2025-08-20 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-09-15 | Spain | Acceptable 2025-10-28
|
2025-10-29 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2026-03-27 | Spain | Acceptable 2026-05-14
|
2026-05-19 |