A Study of Ponatinib With Chemotherapy in Children, Teenagers, and Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia

IDENTIFIERS
Trial Protocol: Ponatinib-1501
Clinicaltrials.gov: NCT04501614
PURPOSE

This study is about an anticancer drug called ponatinib which is a tyrasine kinase inhibitor given with chemotherapy to children, teenagers, and young adults up to 21 years of age with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia who have relapsed or are resistant to other treatment. The main aims of this study are to confirm the highest dose of ponatinib tablets and minitablet capsules that can be given to participants with acceptable side effects, and to evaluate if participant's leukemia achieves remission. Participants will take ponatinib tablets with chemotherapy. For participants who cannot swallow tablets, a minitablet form of the ponatinib will be provided. Participants will take ponatinib for 10 weeks in combination with chemotherapy (reinduction and consolidation blocks) and will be followed up for at least 3 years.

Recruiting
US
AR
AU
12+
BR
CN
CZ
FR
IT
KR
MX
NL
PL
PT
ES
GB

Trial at a glance

What medical condition is being studied?   Leukemia
What is the trial testing?   Ponatinib, Ponatinib AAF, Chemotherapy Agents
Are placebos part of the trial?   No
How many participants are being enrolled?   68
When is the trial being conducted?    - 
How long is participation in the trial?   Each participant will be in this study for about 3 years or possibly longer.

Key requirements

Sex   
All
Age   
1 - 21 Years
Accepts Healthy Volunteers?   
No

Trial Details / Participation Requirements

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Trial Phase:   Phase 1/Phase 2
Trial Type:   Interventional
Trial Arms/Groups or Cohorts:   
Ponatinib: Ponatinib tablet or age appropriate formulation (AAF) in combination with chemotherapy backbone, orally, once daily in both reinduction block and consolidation block (35 days each including 29 days of treatment followed by rest period from chemotherapy for a minimum of 6 days consisting of daily ponatinib only) in Phase 1 to determine RP2D. In Phase 2 participants will receive ponatinib at RP2D in combination with chemotherapy backbone at RP2D determined in Phase 1.
Intervention:   Ponatinib, Ponatinib AAF, Chemotherapy Agents
Primary Outcome Measure(s):   
Phase 1: Recommended Phase 2 Dose (RP2D) of Ponatinib in Combination With Chemotherapy [Time Frame: Up to Week 4]
Phase 2: Complete Remission (CR) Rate at the end of Reinduction Block [Time Frame: Up to Week 4]
Secondary Outcome Measure(s):   
Phase 1: CR Rate at the end of Reinduction Block [Time Frame: Up to Week 4]
Phase 1: Presence the Breakpoint Cluster Region- Abelson (BCR-ABL1) Domain Mutation Pre and Post Ponatinib Treatment [Time Frame: Up to Week 8]
Phase 2: Percentage of Participants with Continued CR or who Achieved CR at the End of Consolidation Block [Time Frame: Up to Week 8]
Phase 2: Percentage of Participants with Negative Minimal Residual Disease (MRD) [Time Frame: Up to Weeks 4 and 8]
Phase 2: Percentage of Participants who Relapsed or Progressed Following Reinduction and Consolidation [Time Frame: Up to Weeks 4 and 8]
Phase 2: Event-free Survival (EFS) [Time Frame: Up to approximately 36 months]
Phase 2: Progression-free Survival (PFS) [Time Frame: Up to approximately 36 months]
Phase 2: Overall Survival (OS) [Time Frame: Up to approximately 36 months]
Phase 2: Duration of Response (DOR) [Time Frame: Up to approximately 36 months]
Phase 2: Percentage of Participants who Underwent Hematopoietic Stem Cell Transplantation (HSCT) [Time Frame: Up to approximately 36 months]
Phase 2: Presence of BCR-ABL Domain Mutation Pre and Post Ponatinib Treatment [Time Frame: Up to Week 8]
Phase 1: Cmax: Maximum Observed Plasma Concentration for Ponatinib [Time Frame: Predose and at multiple timepoints (Up to 24 hours) postdose on Days 1 and 22]
Phase 1: Tmax: Time of First Occurrence of Cmax for Ponatinib [Time Frame: Predose and at multiple timepoints (Up to 24 hours) postdose on Days 1 and 22]
Phase 1: AUClast: Area Under the Plasma Concentration-Time Curve From Time 0 to the Time of the Last Quantifiable Concentration for Ponatinib [Time Frame: Predose and at multiple timepoints (Up to 24 hours) postdose on Days 1 and 22]
Phase 1 and 2: Number of Participants with Adverse Events (AEs), Serious Adverse Events (SAEs), Venous Thrombotic/Embolic Events (VTEs), and Adverse Events of Special Interest (AESIs) [Time Frame: From the first dose of study drug up to 30 days after last dose of study drug (Up to 36 months)]
Entry Criteria:
- Men, women, boys, and girls can take part. - Must be from 1 year to 21 years old. - Must have Philadelphia Chromosome-Positive (Ph+) Acute Lymphoblastic Leukemia (ALL), Ph+Mixed Phenotype Acute Leukemia (ALL), and for the US sites only Philadlephia Chromosome-Like ALL. - Cannot have a history or current diagnosis of Burkitt leukemia/lymphoma or mature B-cell leukemia. Additional entry criteria will be discussed with the study doctor.

Trial Locations

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RESULTS
Phoenix Childrens Hospital

1919 E Thomas Rd, Phoenix, AZ 85016, USA

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Arkansas Children's Hospital

1 Children's Way, Little Rock, AR 72202, USA

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Children's Hospital Los Angeles

4650 Sunset Blvd, Los Angeles, CA 90027, USA

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Rady Childrens Hospital San Diego - PIN

3020 Children's Way, San Diego, CA 92123, USA

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Alfred I Dupont Hospital For Children

1600 Rockland Rd, Wilmington, DE 19803, USA

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Ann and Robert H Lurie Childrens Hospital of Chicago

225 E Chicago Ave, Chicago, IL 60611, USA

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Riley Hospital For Children

705 Riley Hospital Dr, Indianapolis, IN 46202, USA

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Cincinnati Children's Hospital Medical Center - PIN

3333 Burnet Ave, Cincinnati, OH 45229, USA

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Childrens Medical Center Research Institute at UT Southwestern

6000 Harry Hines Blvd, Dallas, TX 75235, USA

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Hospital Universitario Austral

Av. Juan Domingo Perón 1500, Pilar Centro, Provincia de Buenos Aires, Argentina

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Hospital Italiano de Buenos Aires

Pres. Tte. Gral. Juan Domingo Perón 4190, C1199 CABA, Argentina

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Queensland Childrens Hospital

501 Stanley St, South Brisbane QLD 4101, Australia

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Royal Children's Hospital Melbourne - PIN

50 Flemington Rd, Parkville VIC 3052, Australia

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Perth Childrens Hospital

15 Hospital Ave, Nedlands WA 6009, Australia

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Fakultni nemocnice Brno

Jihlavská 340/20, 625 00 Brno-Bohunice-Brno-Starý Lískovec, Czechia

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Fakultni nemocnice v Motole

V Úvalu 84, 150 06 Praha 5, Czechia

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Hopital Sud

16 Boulevard de Bulgarie, 35200 Rennes, France

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Assistance Publique Hopitaux de Marseille

80 Rue Brochier, 13005 Marseille, France

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Hopital Robert Debre

Hôpital Robert Debré, Bd Sérurier, 75019 Paris, France

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Hopital Des Enfants

330 Avenue de Grande Bretagne, 31300 Toulouse, France

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IRCCS Ospedale Pediatrico Bambino Gesu - INCIPIT - PIN

Piazza di Sant'Onofrio, 4, 00165 Roma RM, Italy

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Fndazione MBBM (MONZA E BRIANZA PER IL BAMBINO E LA SUA MAMMA) - c/o Centro Maria Letizia Verga

Centro Maria Letizia Verga, Via Cadore, 20900 Monza MB, Italy

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Ospedale Infantile Regina Margherita - INCIPIT - PIN

Piazza Polonia, 94, 10126 Torino TO, Italy

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Seoul National University Hospital

South Korea, Seoul, Jongno-gu, Ihwa-dong, 대학로 101

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Severance Hospital Yonsei University Health System

50-1 Yonsei-ro, Sinchon-dong, Seodaemun-gu, Seoul, South Korea

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Asan Medical Center - PPDS

88 Olympic-ro 43-gil, Pungnap 2(i)-dong, Songpa-gu, Seoul, South Korea

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The Catholic University of Korea, Seoul St. Mary's Hospital

222 Banpo-daero, Banpo-dong, Seocho-gu, Seoul, South Korea

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Princess Maxima Center for Pediatric Oncology - PIN

Heidelberglaan 25, 3584 CS Utrecht, Netherlands

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Hospital Universitario Vall d'Hebron - PPDS

Passeig de la Vall d'Hebron, 119, 08035 Barcelona, Spain

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Hospital Infantil Universitario Nino Jesus - PIN

Av. de Menéndez Pelayo, 65, 28009 Madrid, Spain

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Hospital Universitario Virgen del Rocio - PPDS

Av. Manuel Siurot, S/n, 41013 Sevilla, Spain

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Royal Hospital for Children (Glasgow) - PPDS - PIN

1345 Govan Rd, Glasgow G51 4TF, UK

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UCSF Medical Comprehensive Cancer

400 Parnassus Ave Fourth Floor, San Francisco, CA 94143, USA

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Dana Farber Cancer Institute

450 Brookline Ave, Boston, MA 02215, USA

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Children's Mercy Hospital and Clinica

3101 Broadway Blvd, Kansas City, MO 64111, USA

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Nationwide Children's Hospital

700 Children's Dr, Columbus, OH 43205, USA

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Children's Hospital of Philadelphia

3401 Civic Center Blvd, Philadelphia, PA 19104, USA

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St Jude Children's Research Hospital

262 Danny Thomas Pl, Memphis, TN 38105, USA

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Rua Ramiro Barcelos, 2350

Jardim das Américas, Curitiba - State of Paraná, 81520-060, Brazil

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Irmandade Da Santa Casa de Misericordia de Porto Alegre

Rua Professor Annes Dias, 295 - Centro Histórico, Porto Alegre - RS, 90020-090, Brazil

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Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

Rua Ramiro Barcelos, 2350 - Av. Protásio Alves, 211 - Santa Cecilia, Porto Alegre - RS, 90035-903, Brazil

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Clinica SUPRA

R. Assis Brasil, 404-E - Maria Goretti, Chapecó - SC, 89801-222, Brazil

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Fundacao Pio XII Hospital de Cancer de Barretos

R. Antenor Duarte Viléla, 1331 - Dr. Paulo Prata, Barretos - SP, 14784-400, Brazil

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Hospital Das Clinicas da Faculdade de Medicina de Ribeirao Preto - USP

Av. Bandeirantes, 3900 - Vila Monte Alegre, Ribeirão Preto - SP, 14049-900, Brazil

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Graacc Grupo de Apoio Ao Adolescente E A Crianca Com Cancer

Rua Pedro de Toledo, 572 - Vila Clementino, São Paulo - SP, 04039-001, Brazil

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Hospital Santa Marcelina

R. Santa Marcelina, 177 - Vila Carmosina, São Paulo - SP, 08270-070, Brazil

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Children's Hospital of Soochow University

1166 Pingchuan Rd, Gusu Qu, Suzhou Shi, Jiangsu Sheng, China

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West China Second University Hospital, Sichuan Univesity

Chenglong Ave West Section, Jinjiang Qu, Chengdu Shi, Sichuan Sheng, China

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Children's Hospital of Chongqing Medical University

136 Zhongshan 2nd Rd, Liang Lu Kou, Yuzhong Qu, Chongqing Shi, China

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The Affiliated Hospital of Guizhou Medical University

China, Guiyang Shi, Yunyan Qu, CN 北京路 16 16号 邮政编码: 550004

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The Second Hospital of Anhui Medical University

678 Furong Rd, Shushan Qu, Hefei Shi, Anhui Sheng, China, 230601

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Qilu Hospital of Shandong University

107 Wenhua W Rd, Lixia Qu, Jinan Shi, Shandong Sheng, China

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The Affiliated Hospital of Qingdao University

16 Jiangsu Rd, Shinan Qu, Qingdao Shi, Shandong Sheng, China

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Children's Hospital of Shanghai

101 Beiyuan Rd, Tang Qiao, Pudong Xinqu, Shanghai Shi, China

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Shanghai Childrens Medical Center

324B Pujian Rd, Tang Qiao, Pudong Xinqu, Shanghai Shi, China, 200127

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Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences

Tianjin, China

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Union Hospital Tongji Medical College Huazhong University of Science and Technology

China, Hubei Sheng, Wuhan Shi, Jiangan Qu, 三阳路

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Tongji Hospital Tongji Medical College Huazhong University of Science and Technology

1037 Luoyu Rd, Hongshan Qu, Wuhan Shi, Hubei Sheng, China, 430074

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Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN

Via Gerolamo Gaslini, 5, 16147 Genova GE, Italy

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Hospital Universitario Dr. Jose Eleuterio Gonzalez

Dr. José Eleuterio González (Gonzalitos) S/N, Mitras Centro, 64460 Monterrey, N.L., Mexico

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Instituto Nacional de Pediatria

Insurgentes Sur 3700 Letra C, Insurgentes Cuicuilco, Coyoacán, 04530 Ciudad de México, CDMX, Mexico

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Nuevo Hospital Civil de Guadalajara Dr. Juan I. Menchaca

Salvador Quevedo y Zubieta 750, Independencia Oriente, 44340 Guadalajara, Jal., Mexico

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Uniwersytecki Szpital Dzieciecy

Wielicka 265, 30-663 Kraków, Poland

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SPZOZ Centralny Szpital Kliniczny UM w Lodzi

Pomorska 251, 92-213 Łódź, Poland

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Wojewodzki Szpital Specjalistyczny

Żołnierska 18, 10-561 Olsztyn, Poland

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SPSK Nr 1 im. Prof.Stanislawa Szyszko Slaskiego Uniwersytetu Medycznego

Księdza Karola Koziołka 1, 41-800 Zabrze, Poland

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Instituto Portugues de Oncologia de Lisboa Francisco Gentil, E.P.E.

R. Prof. Lima Basto, 1500 Lisboa, Portugal

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Instituto Portugues de Oncologia Do Porto Francisco Gentil Epe - PPDS

4200 Porto, Portugal

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Birmingham Childrens Hospital

Steelhouse Ln, Birmingham B4 6NH, UK

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Cambridge University Hospitals NHS Foundation Trust

Hills Rd, Cambridge CB2 0QQ, UK

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Royal Marsden Hospital - Surrey

Downs Rd, Sutton SM2 5PT, UK

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Trial Protocol ID: Ponatinib-1501
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