Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - First administration to humans
Status
Authorised, recruiting
Participants planned
144
Countries
3
Sites
23
Acute Leukemia
Phase 1: Part 1 (Dose Escalation): Determine the (RP2Ds of bleximenib Phase 1: Part 2 (Dose Expansion): Determine the safety and tolerability at the RP2D(s) Phase 2: To evaluate the efficacy of bleximenib at the RP2D
Key facts
- Sponsor
- Janssen - Cilag International
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Neoplasms [C04]
- Trial duration
- 15 Jun 2021 → ongoing
- Decision date (initial)
- 2025-07-01
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2023-506581-31-00
- EudraCT number
- 2020-005967-30
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Pharmacodynamic, Pharmacokinetic, Dose response, Others, Efficacy, Safety
Phase 1: Part 1 (Dose Escalation): Determine the (RP2Ds of bleximenib
Phase 1: Part 2 (Dose Expansion): Determine the safety and tolerability at the RP2D(s)
Phase 2: To evaluate the efficacy of bleximenib at the RP2D
Conditions and MedDRA coding
Acute Leukemia
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.1 | LLT | 10024330 | Leukemia acute | 10029104 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 6
- 1. Phase 1 and Phase 2 adult cohorts: ≥ 18 years of age and Phase 1 adolescent cohort 12 to <18 years of age
- 2. Phase 1: R/R acute leukemia and has exhausted, or is ineligible for, available therapeutic options.
- 3. Phase 1: Acute leukemia harboring KMT2A, NPM1 or nucleoporin alterations. Phase 2: AML harboring KMT2A-r or NPM1 mutations only.
- 4. Pretreatment clinical laboratory values meeting the criteria
- 5. ECOG performance status grade of 0, 1 or 2.
- 6. A female of childbearing potential must have a negative highly sensitive serum β-human chorionic gonadotropin at screening and within 48 hours prior to the first dose of study treatment
Exclusion criteria 9
- 1. Diagnosed with acute promyelocytic leukemia, Down syndrome associated leukemia or juvenile myelomonocytic leukemia.
- 2. Active CNS involvement
- 3. Prior solid organ transplantation
- 4. Cardiovascular disease that increases the risk for torsades de pointes or that was diagnosed within 6 months prior to the first dose of study treatment.
- 5. QTc ≥450 milliseconds for males or ≥470 milliseconds for females
- 6. Any toxicity from previous anticancer therapy that has not resolved to baseline or to Grade 1 or less
- 7. Pulmonary compromise that requires the need for supplemental oxygen use to maintain adequate oxygenation
- 8. Reported temperature >100.5ºF/38ºC within 48 hours prior to the first dose of study treatment
- 9. Known allergies, hypersensitivity, or intolerance to bleximenib or its excipients.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 3
- Phase 1: Part 1 (Dose Escalation): Incidence and severity of adverse events, including dose-limiting toxicity
- Phase 1: Part 2 (Dose Expansion): Incidence and severity of adverse events
- Phase 2: Rate of CR/CRh assessed by the IRC.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 16
PRD10622891 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10622888 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- POWDER FOR ORAL SOLUTION
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10622887 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10622892 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10622890 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD11370367 · Product
- Active substance
- Bleximenib
- Substance synonyms
- N-ethyl-5-fluoro-N-isopropyl-2-[[5-(2-[(3R)-6-[(2-methoxyethyl)(methyl)amino]-2-methylhexan-3-yl]-2,6-diazaspiro[3.4]octan-6-yl)-1,2,4-triazin-6-yl]oxy]benzamide, JNJ-75276617
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD11825465 · Product
- Active substance
- Bleximenib
- Substance synonyms
- N-ethyl-5-fluoro-N-isopropyl-2-[[5-(2-[(3R)-6-[(2-methoxyethyl)(methyl)amino]-2-methylhexan-3-yl]-2,6-diazaspiro[3.4]octan-6-yl)-1,2,4-triazin-6-yl]oxy]benzamide, JNJ-75276617
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10820462 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD11370369 · Product
- Active substance
- Bleximenib
- Substance synonyms
- N-ethyl-5-fluoro-N-isopropyl-2-[[5-(2-[(3R)-6-[(2-methoxyethyl)(methyl)amino]-2-methylhexan-3-yl]-2,6-diazaspiro[3.4]octan-6-yl)-1,2,4-triazin-6-yl]oxy]benzamide, JNJ-75276617
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD8898628 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD11370368 · Product
- Active substance
- Bleximenib
- Substance synonyms
- N-ethyl-5-fluoro-N-isopropyl-2-[[5-(2-[(3R)-6-[(2-methoxyethyl)(methyl)amino]-2-methylhexan-3-yl]-2,6-diazaspiro[3.4]octan-6-yl)-1,2,4-triazin-6-yl]oxy]benzamide, JNJ-75276617
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10622886 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10622893 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD8898622 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD8898624 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10622889 · Product
- Active substance
- JNJ-75276617
- Pharmaceutical form
- POWDER FOR ORAL SOLUTION
- Route of administration
- ORAL USE
- Authorisation status
- Not Authorised
- MA holder
- JANSSEN-CILAG INTERNATIONAL N.V.
- Paediatric formulation
- No
- Orphan designation
- No
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 10
| Organisation | City, country | Duties |
|---|---|---|
| Hematogenix Laboratory Services LLC ORG-100040020
|
Tinley Park, United States | Laboratory analysis |
| Almac Clinical Technologies LLC ORG-100043036
|
Souderton, United States | Interactive response technologies (IRT) |
| Parexel China Co. Ltd. ORG-100043174
|
Shanghai, China | Data management |
| Ancillare LP ORG-100044089
|
Horsham, United States | Other |
| Hematogenix Laboratory Services Limited ORG-100047188
|
Cheadle, United Kingdom | Laboratory analysis |
| Hematogenix ORG-100047219
|
Cyberjaya, Malaysia | Laboratory analysis |
| SGS Belgium ORG-100007917
|
Mechelen, Belgium | Other |
| Labcorp Central Laboratory Services LP ORG-100032236
|
Indianapolis, United States | Other |
| Eresearchtechnology Inc. ORG-100013039
|
Philadelphia, United States | Code 13, Other |
| Janssen Pharmaceutica ORG-100000625
|
Beerse, Belgium | Laboratory analysis |
Locations
3 EU/EEA countries · 23 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Belgium | Authorised, recruiting | 2 | 1 |
| France | Ongoing, recruiting | 38 | 13 |
| Spain | Ongoing, recruiting | 46 | 9 |
| Rest of world
Japan, Canada, United Kingdom, Brazil, United States, Israel, Taiwan, China, Korea, Republic of, Australia
|
— | 58 | — |
Investigational sites
Universitair Ziekenhuis Gent
Pediatric hemato - Oncology, Corneel Heymanslaan 10, 9000, Gent
Centre Hospitalier Regional Universitaire De Tours
Service d'Hematologie et Therapie Cellulaire et Centre d’Investigation Clinique, 2 Boulevard Tonnelle, 37000, Tours
Assistance Publique Hopitaux De Paris
Centre D’Investigations Cliniques, 1 Avenue Claude Vellefaux, 75010, Paris
Institut Paoli-Calmettes
Unite de Traitement des Leucemies, 232 Boulevard De Sainte Marguerite, Bp 156, Marseille
Institut Universitaire Du Cancer Toulouse-Oncopole
Hematologie, 1 Avenue Irene Joliot Curie, 31100, Toulouse
CHRU De Nancy
Service Hematologie, Rue Du Morvan, 54500, Vandoeuvre Les Nancy
Les Hopitaux Universitaires De Strasbourg
Service Hematologie, 1 Avenue Moliere, Bp 49, Strasbourg Cedex 2
CHRU De Nancy
Service Onco-Hematologie pédiatrique, Rue Du Morvan, 54500, Vandoeuvre Les Nancy
Centre Hospitalier Universitaire De Nantes
Service d'Hematologie, 1 Place Alexis Ricordeau, 44000, Nantes
Institut De Cancerologie Strasbourg Europe
Service Hematologie, 17 Rue Albert Calmette, 67200, Strasbourg
Assistance Publique Hopitaux De Paris
Hopital Trousseau, departement oncologie et hématologie pédiatrique, 26 Avenue Du Docteur Arnold Netter, 75012, Paris
CHU Besancon
Service Hematologie, 3 Boulevard Alexandre Fleming, 25000, Besancon
Hospices Civils De Lyon
Service Hematologie, 165 Chemin Du Grand Revoyet, 69310, Pierre Benite
Institut Gustave Roussy
Departement D'Innovation Therapeutique Et Essais Precoces, 114 Rue Edouard Vaillant, 94800, Villejuif
Hospital Universitario Fundacion Jimenez Diaz
Hematology, Avenida De Los Reyes Catolicos 2, 28040, Madrid
Hospital Clinico Universitario De Valencia
Hematology, Avenida Blasco Ibanez 17, 46010, Valencia
University Hospital Virgen Del Rocio S.L.
Hematology, Avenida De Manuel Siurot S/n, 41013, Sevilla
Hospital Universitario De Salamanca
Hematology, Paseo De San Vicente 58-182, 37007, Salamanca
Clinica Universidad De Navarra
Hematology, Avenue Pio XII 36, 31008, Pamplona
Hospital Clinic De Barcelona
Hematology department, Calle Villarroel 170, 08036, Barcelona
Hospital General Universitario Gregorio Maranon
Hematology, Calle Del Doctor Esquerdo 46, 28007, Madrid
Hospital Universitari Vall D Hebron
Hematology, Edificio Materno-Infantil, Passeig De La Vall D'hebron 119-129, Barcelona
Hospital Infantil Universitario Nino Jesus
Hematology - Pediatric, Avenida De Menendez Pelayo 65, 28009, Madrid
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 |
|---|---|---|---|---|---|
| Belgium | 2026-01-14 | ||||
| France | 2021-06-15 | 2021-06-15 | |||
| Spain | 2021-08-02 | 2021-11-15 |
Application history
10 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-09-22 | Spain | Acceptable 2023-11-02
|
2023-11-02 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-02-22 | Spain | Acceptable 2024-05-20
|
2024-05-20 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-06-14 | Spain | Acceptable 2024-09-06
|
2024-09-06 |
| 4 | SUBSTANTIAL MODIFICATION | SM-3 | 2024-12-20 | Spain | Acceptable 2025-04-10
|
2025-04-10 |
| 5 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-04-15 | Acceptable | 2025-05-21 | |
| 6 | SUBSEQUENT ADDITION OF MSC | APP-6 | 2025-04-23 | Acceptable 2025-04-10
|
2025-07-01 | |
| 7 | SUBSEQUENT ADDITION OF MSC | APP-7 | 2025-04-23 | Acceptable 2025-04-10
|
2025-07-03 | |
| 8 | SUBSTANTIAL MODIFICATION | SM-5 | 2025-08-13 | Spain | Acceptable with conditions 2025-11-24
|
2025-11-24 |
| 9 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-12-19 | Acceptable with conditions 2025-11-24
|
2025-12-19 | |
| 10 | SUBSTANTIAL MODIFICATION | SM-6 | 2025-12-19 | Spain | Acceptable with conditions | 2026-01-20 |