Molecular Characterization of Inflammatory Tumors Facilitates Initiation of Effective Therapy.
Franziska Wachter, Alyaa Al-Ibraheemi, Maria C Trissal...
https://pubmed.ncbi.nlm.nih.gov/34814185Actively Recruiting
Phase 1
Phase 2
Age: 0 - 25Years
All Genders
ID04094610
A Phase 1/2, Open-Label, Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity Study of Repotrectinib in Pediatric and Young Adult Subjects With Advanced or Metastatic Malignancies Harboring ALK, ROS1, NTRK1-3 Alterations
Led by Turning Point Therapeutics, Inc. · Updated on 2025-11-19
75
Participants Needed
68
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating repotrectinib, an oral drug, in pediatric and young adult patients with advanced or metastatic cancers that have specific genetic changes in ALK, ROS1, or NTRK1-3 genes. The study includes two phases: Phase 1 focuses on finding safe dose levels and the recommended dose for children under 12, while Phase 2 assesses the drug's anti-tumor activity in patients aged 12 to 25 with certain gene fusions or alterations. This study aims to address cancers that have progressed despite other treatments or have no standard cure options. In Phase 1, about 12 children under 12 years old with advanced solid tumors or lymphoma will receive different doses of repotrectinib to determine safety and the best dosing. Phase 2 includes three groups of patients aged 12 to 25, with varying genetic profiles and prior treatment histories, all receiving oral repotrectinib. The drug will be given continuously in cycles, with careful monitoring. Enrollment for Phase 1 and Phase 2 occurs simultaneously, allowing younger children to join Phase 2 once their dose is established. Participants will undergo regular assessments including scans and tests to measure tumor response, drug levels in the blood, and side effects. The study tracks several outcomes such as response rate, duration of response, survival, and brain tumor progression over approximately three years. Safety and tolerability are closely monitored during the first 28 days of treatment, with ongoing follow-up visits throughout the study period. This long-term observation helps researchers understand both the drug's effects and patient well-being.
CONDITIONS
Brief Title
A Study of Repotrectinib in Pediatric and Young Adult Subjects Harboring ALK, ROS1, OR NTRK1-3 Alterations
Who Can Participate
Age: 0 - 25Years
All Genders
Eligibility Criteria
You may qualify if you...
- Documented genetic ROS1 mutation, fusion, amplification, or NTRK1-3 fusion identified by a qualified lab
- Age under 12 years for Phase 1; age 12 to 25 years for Phase 2
- Prior chemotherapy is allowed
- Prior immunotherapy is allowed
- All prior treatment side effects (except hair loss) resolved to mild level or better
- Measurable disease by RECIST or RANO at enrollment
- Neurologically stable with stable or decreasing steroids for at least 7 days if CNS tumor or metastases
- Lansky score (under 16 years) or Karnofsky score (16 years or older) of at least 50
- Life expectancy of at least 12 weeks
- Adequate blood, kidney, and liver function
- For Phase 2 cohorts: specific genetic and treatment history criteria with confirmed measurable disease for some cohorts
You will not qualify if you...
- Neuroblastoma with only bone marrow disease evaluable by aspiration only
- Major surgery within 14 days before starting repotrectinib
- Active infections requiring treatment, including bacterial, fungal, viral, or HIV
- Gastrointestinal diseases or malabsorption syndromes affecting drug absorption
- Certain heart problems including long QT interval, heart block, or conditions increasing risk of arrhythmia
- Peripheral neuropathy grade 2 or higher
- Use or expected need for strong CYP3A4 inhibitors or inducers
- Allergies to repotrectinib or its ingredients
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants receive oral repotrectinib (TPX-0005) to assess safety, tolerability, and anti-tumor activity.
Visits occur on Day 1 and Day 15 of Cycle 1 for pharmacokinetic assessments; additional visits scheduled per protocol
Follow-up
Duration - Up to approximately three years
Participants are monitored for response and long-term outcomes after treatment ends.
Periodic visits during follow-up based on clinical needs
Trial Site Locations
Total: 68 locations
1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027-6062
Actively Recruiting
2
University of California at Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
3
Children's Hospital Colorado - Anschutz Medical Campus
Aurora, Colorado, United States, 80045
Actively Recruiting
4
Local Institution - 2105
Orlando, Florida, United States, 32806
Completed
5
Local Institution - 2120
Orlando, Florida, United States, 32827
Completed
6
Children's Healthcare of Atlanta - Egleston Hospital
Atlanta, Georgia, United States, 30329
Actively Recruiting
7
Maine Medical Center
Scarborough, Maine, United States, 04074
Actively Recruiting
8
Dana Farber Cancer Institute.
Boston, Massachusetts, United States, 02215
Actively Recruiting
9
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
10
Local Institution - 2110
New Brunswick, New Jersey, United States, 08901
Completed
11
Local Institution - 2102
New York, New York, United States, 10065
Completed
12
Levine Children's Hospital- Pediatric Neuro-Oncology
Charlotte, North Carolina, United States, 28203
Actively Recruiting
13
Local Institution - 2112
Cleveland, Ohio, United States, 44195
Completed
14
Local Institution - 2114
Hershey, Pennsylvania, United States, 17033
Completed
15
Children's Hospital of Philadelphia-Center for Childhood Cancer Research
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
16
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38015
Actively Recruiting
17
The University of Texas Southwestern Medical Center - Harold C Simmons Comprehensive Cancer Center
Dallas, Texas, United States, 75390
Actively Recruiting
18
Baylor College of Medicine
Houston, Texas, United States, 77030
Actively Recruiting
19
Local Institution - 2104
Houston, Texas, United States, 77030
Completed
20
Children's Hospital of Richmond at VCU
Richmond, Virginia, United States, 23219
Withdrawn
21
Local Institution - 6104
Randwick, New South Wales, Australia, 2031
Actively Recruiting
22
Local Institution - 6103
Westmead, New South Wales, Australia, 0
Completed
23
Children's Health Queensland Hospital and Health Service
South Brisbane, Queensland, Australia, 4101
Actively Recruiting
24
Perth Childrens Hospital
Nedlands, Western Australia, Australia, 6009
Actively Recruiting
25
University Of Calgary
Calgary, Alberta, Canada, T3B 6A8
Actively Recruiting
26
Stollery Children'S Hospital
Edmonton, Alberta, Canada, T6G 2B7
Actively Recruiting
27
Children'S Hospital Of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Actively Recruiting
28
St Justine Hospital
Montreal, Quebec, Canada, H3T 1C5
Actively Recruiting
29
Rigshospitalet - Glostrup
Copenhagen, Denmark, 2100
Actively Recruiting
30
Local Institution - 6111
Lyon, Rhone, France, 69008
Not Yet Recruiting
31
Centre Hospitalier Universitaire D'Angers
Angers, France, 49933
Actively Recruiting
32
Centre Hospitalier Universitaire de Bordeaux - Groupe Hospitalier Pellegrin
Bordeaux, France, 33076
Actively Recruiting
33
Institut d Hematologie et d Oncologie Pediatriques
Lyon, France, 69373
Actively Recruiting
34
Hôpitaux Universitaires de Marseille Timone
Marseille, France, 13385
Actively Recruiting
35
Local Institution - 6110
Marseille, France, 13385
Not Yet Recruiting
36
Local Institution - 6112
Nantes, France, 44093
Not Yet Recruiting
37
Local Institution - 6109
Paris, France, 75005
Not Yet Recruiting
38
Institut Gustave-Roussy
Villejuif, France, 94805
Actively Recruiting
39
Local Institution - 6108
Villejuif, France, 94805
Not Yet Recruiting
40
Fondazione IRCCS - Istituto Nazionale dei Tumori
Milan, Italy, 20133
Actively Recruiting
41
Local Institution - 6113
Padova, Italy, 35128
Withdrawn
42
Local Institution - 4302
Rome, Italy, 00165
Not Yet Recruiting
43
Local Institution - 6114
Torino, Italy, 10126
Withdrawn
44
National University Hospital
Singapore, Singapore, 119228
Actively Recruiting
45
KK Women's and Children's Hospital
Singapore, Singapore, 229899
Actively Recruiting
46
Local Institution - 6303
Seoul, Seodaemun-gu, South Korea, 03722
Completed
47
Seoul National University Hospital
Seoul, South Korea, 03080
Actively Recruiting
48
Asan Medical Center
Seoul, South Korea, 05505
Actively Recruiting
49
Local Institution - 6304
Seoul, South Korea, 06351
Completed
50
Hospital Sant Joan De Deu
Esplugues de Llobregat, Barcelona, Spain, 08950
Actively Recruiting
51
Clínica Universidad de navarra
Pamplona, Navarre, Spain, 31008
Actively Recruiting
52
Local Institution - 6105
Barcelona, Spain, 08014
Not Yet Recruiting
53
Hospital Universitari Vall d'Hebron
Barcelona, Spain, 8035
Actively Recruiting
54
Hospital Infantil Universitario Nino Jesus
Madrid, Spain, 28009
Actively Recruiting
55
Local Institution - 6106
Madrid, Spain, 28009
Not Yet Recruiting
56
Clinica Universidad de Navarra
Madrid, Spain, 28022
Actively Recruiting
57
HM Sanchinarro University Hospital
Madrid, Spain, 28050
Actively Recruiting
58
Hospital Universitario Virgen del Rocio
Seville, Spain, 41013
Actively Recruiting
59
Hospital Universitario Y Politecnico La Fe
Valencia, Spain, 46026
Actively Recruiting
60
Local Institution - 6107
Valencia, Spain, 46026
Not Yet Recruiting
61
National Taiwan University Hospital
Taipei, Taiwan, 100225
Actively Recruiting
62
Taipei Medical University Hospital
Taipei, Taiwan, 11031
Actively Recruiting
63
Alder Hey Children's NHS Foundation Trust
Liverpool, England, United Kingdom, L12 2AP
Actively Recruiting
64
Local Institution - 4403
Birmingham, United Kingdom, B4 6DH
Completed
65
University Hospital of Wales
Cardiff, United Kingdom, CF14 4XW
Actively Recruiting
66
Royal Hosp. for Children
Glasgow, United Kingdom, G51 4TF
Actively Recruiting
67
The Royal Marsden NHS Foundation Trust
London, United Kingdom, SW3 6JJ
Actively Recruiting
68
Great Ormond Street Hospital For Children NHS Foundation Trust
London, United Kingdom, WC1N 3JH
Actively Recruiting
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Research Team
B
BMS Study Connect Contact Center www.BMSStudyConnect.com
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First line of the email MUST contain the NCT# and Site #.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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