New targeted drug shows promise for Hard-to-Treat cancers
NCT ID NCT03093116
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new drug called repotrectinib in people with advanced solid tumors that have certain gene changes (ALK, ROS1, or NTRK). The trial has two parts: first, finding a safe dose, and second, seeing how well the drug shrinks tumors. About 500 adults with advanced cancers that have not responded to other treatments are being enrolled.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- repotrectinib (TPX-0005) capsules
- What this could lead to
- If successful, this could lead to a new targeted treatment option for people with advanced solid tumors that have specific genetic changes (ALK, ROS1, or NTRK).
- What could go wrong
- This is an early phase 1/2 trial, so safety and effectiveness are not yet proven. It may not work for all participants, and side effects are still being studied.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 500 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2017
- Expected to finish
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Feb 2028
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 years and older
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
PHASE 1 Key Inclusion Criteria: 1. Histologically or cytologically confirmed diagnosis of locally advanced, or metastatic solid tumor (including primary CNS tumors) (Stage IV, American Joint Committee on Cancer v.7) that harbors an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement by protocol specified tests. 2. ECOG PS 0-1. 3. Age ≥18 (or age ≥ 20 of age as required by local regulation). 4. Capability to swallow capsules intact (without chewing, crushing, or opening). 5. At least 1 measurable target lesion according to RECIST version 1.1. CNS-only measurable disease as defined by RECIST version 1.1 is allowed. 6. Prior cytotoxic chemotherapy is allowed. 7. Prior immunotherapy is allowed. 8. Resolution of all acute toxic effects (excluding alopecia) of any prior anti-cancer therapy to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 4.03 Grade less than or equal to 1. 9. Patients with asymptomatic CNS metastases (treated or untreated) and/or asymptomatic leptomeningeal carcinomatosis are eligible to enroll if they satisfy the protocol specified criteria. 10. Baseline laboratory values fulfilling the following requirements:Absolute neutrophils count (ANC) ≥1500/mm3 (1.5 × 109/L); Platelets (PLTs) ≥100,000/mm3 (100 × 109/L); Hemoglobin ≥ 9.0 g/dL transfusions are allowed; Serum creatinine or creatinine clearance Within normal limits or \> 40 mL/min; Total serum bilirubin \< 1.5 × ULN; Liver transaminases (ASTs/ALTs) \< 2.5 × ULN; \< 5 × ULN if liver metastases are present Alkaline phosphatase (ALP); \< 2.5 × ULN; \< 5 × ULN if liver and/or bone metastasis are present; Serum calcium, magnesium, and potassium Normal or CTCAE grade ≤ 1 with or without supplementation 11. Life expectancy ≥ 3 months. PHASE 2 Key Inclusion Criteria 1. Histologically or cytologically confirmed diagnosis of locally advanced, or metastatic solid tumor (including primary CNS tumors) that harbors a ROS1, or NTRK1-3 gene fusion. 2. Subject must have a documented ROS1 or NTRK1-3 gene fusion determined by tissue-based local testing using either: 1. a next-generation sequencing (NGS) or quantitative polymerase chain reaction (qPCR) test will be accepted to determine molecular eligibility. • Adequate tumor tissue needs to be sent to the Sponsor designated central diagnostic laboratory for retrospective confirmation by a central diagnostic laboratory test selected by the Sponsor. OR 2. a fluorescence in situ hybridization (FISH) test AND prospective confirmation of fusion status by a central diagnostic laboratory test selected by the Sponsor PRIOR to enrollment will be accepted to determine molecular eligibility. * Adequate tumor tissue must be sent to the Sponsor designated central diagnostic laboratory for prospective confirmation by a central diagnostic laboratory test selected by the Sponsor PRIOR to enrollment. 3. Eastern Cooperative Oncology Group (ECOG) Performance Status 0-1. 4. Age ≥12 (or age ≥ 20 as required by local regulation). 5. Willing and able to provide written institutional review board (IRB)/institutional ethics committee-approved Informed Consent or an Assent signed by a parent or legal guardian for subjects age 12 to 17. 6. At least 1 measurable target lesion according to RECIST (v1.1) prospectively confirmed by Blinded Independent Central Radiology Review (BICR), selected by Sponsor, PRIOR to enrollment. Subjects with CNS-only measurable disease ≥10 mm as defined by RECIST (v1.1) are eligible. 7. Subjects with advanced solid tumors harboring ROS1, NTRK1, NTRK2, or NTRK3 rearrangement will be assigned into 6 distinct expansion (EXP) cohorts provided all inclusion and exclusion criteria are met. i. EXP-1: ROS1 TKI-naïve ROS1+ NSCLC ii. EXP-2: 1 Prior ROS1 TKI and 1 Platinum based chemo ROS1+ NSCLC iii. EXP-3: 2 Prior ROS1 TKIs ROS1+ NSCLC (No Chemo or IO) iv. EXP-4: 1 Prior ROS1 TKI ROS1+ NSCLC (No Chemo or IO) v. EXP-5: TRK TKI-naïve NTRK+ solid tumors vi. EXP-6: TRK TKI-pretreated NTRK+ solid tumors 8. Subjects with asymptomatic CNS metastases (treated or untreated) and/or asymptomatic leptomeningeal carcinomatosis are eligible to enroll if they satisfy the protocol specified criteria. 9. Baseline laboratory values fulfilling the following requirements:Absolute neutrophils count (ANC) ≥1500/mm3 (1.5 × 109/L); Platelets (PLTs) ≥100,000/mm3 (100 × 109/L); Hemoglobin ≥ 9.0 g/dL transfusions are allowed; Serum creatinine or creatinine clearance \> 40 mL/min; Total serum bilirubin \< 1.5 × ULN; Liver transaminases (ASTs/ALTs) \< 2.5 × ULN; \< 5 × ULN if liver metastases are present Alkaline phosphatase (ALP); \< 2.5 × ULN; \< 5 × ULN if liver and/or bone metastasis are present; Serum calcium, magnesium, and potassium Normal or CTCAE grade ≤ 1 with or without supplementation 10. Life expectancy ≥ 3 months. Key Exclusion Criteria PHASE 1 and PHASE 2 1. Concurrent participation in another therapeutic clinical trial. 2. Symptomatic brain metastases or leptomeningeal involvement. 3. History of previous cancer, except for squamous cell or basal-cell carcinoma of the skin, or any in situ carcinoma that has been completely resected, requiring therapy within the previous 2 years. 4. Major surgery within 4 weeks of start of repotrectinib treatment. Radiation therapy (except palliative to relieve bone pain) within 2 weeks of study entry. Palliative radiation (≤10 fractions) must have been completed at least 48 hours prior to study entry 5. Clinically significant cardiovascular disease (either active or within 6 months prior to enrollment): myocardial infarction, unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure (New York Heart Association Classification Class ≥ II), cerebrovascular accident or transient ischemic attack, symptomatic bradycardia, requirement for anti-arrhythmic medication. Ongoing cardiac dysrhythmias of NCI CTCAE grade ≥2 6. Any of the following cardiac criteria: Mean resting corrected QT interval (ECG interval measured from the onset of the QRS complex to the end of the T wave) for heart rate (QTcF) \> 470 msec obtained from 3 ECGs, using the screening clinic ECG machine-derived QTc value Any clinically important abnormalities in rhythm, conduction or morphology of resting ECG (e.g., complete left bundle branch block, third degree heart block, second degree heart block, PR interval \> 250 msec) Any factors that increase the risk of QTc prolongation or risk of arrhythmic events such as heart failure, hypokalemia, congenital long QT syndrome, family history of long QT syndrome, or any concomitant medication known to prolong the QT interval. 7. Known active infections (bacterial, fungal, viral including HIV positivity). 8. Gastrointestinal disease (e.g., Crohn's disease, ulcerative colitis, or short gut syndrome) or other malabsorption syndromes that would impact drug absorption. 9. Peripheral neuropathy of CTCAE ≥grade 2. 10. History of extensive, disseminated, bilateral, or presence of CTCAE grade 3 or 4 interstitial fibrosis or interstitial lung disease including a history of pneumonitis, hypersensitivity pneumonitis, interstitial pneumonia, interstitial lung disease, obliterative bronchiolitis, and pulmonary fibrosis. Subjects with history of prior radiation pneumonitis are not excluded.
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Get notified about this study
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
65 sites in 10 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Anhui Provincial Hospital
RECRUITINGHefei, 230001, China
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Asan Medical Center
RECRUITINGSeoul, 05505, South Korea
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Baptist Memorial Hospital Baptist Cancer Center
RECRUITINGMemphis, Tennessee, 38120, United States
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Beijing Cancer hospital
RECRUITINGBeijing, Beijing Municipality, 100142, China
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Centre Antoine-Lacassagne
RECRUITINGNice, 06189, France
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Centre Georges-Francois Leclerc
RECRUITINGDijon, 21079, France
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Centre Hospitalier Universitarie Grenoble Alpes (Chuga)
RECRUITINGGrenoble, 38043, France
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Centro di Riferimento Oncologico
RECRUITINGPordenone, 33081, Italy
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Chonnam National University Hwasun Hospital
RECRUITINGHwasun-eup, Hwasun-gun, Jeollanam-do, 519-763, South Korea
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Chris O'Brien LifeHouse
RECRUITINGCamperdown, New South Wales, 2050, Australia
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Daping Hospital, the Third Affiliated Hospital of Third Military Medical University /Cancer Center
RECRUITINGDaping, Chongqing Municipality, 00000, China
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Ehime University Hospital
RECRUITINGTōon, Ehime, 791-0295, Japan
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Fondazione IRCCS Istituto Nazionale dei Tumori
RECRUITINGMilan, MI, 20133, Italy
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Guangdong Provincial People'S Hospital
RECRUITINGGuangzhou, Guangdong, 510120, China
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Henan Cancer Hospital/The 1st pneumology department
RECRUITINGZhengzhou, 00000, China
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Hokkaido University Hospital
RECRUITINGSapporo, Hokkaido, 0608648, Japan
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Hospital Universitario HM Sanchinarro CIOCC
RECRUITINGMadrid, 28050, Spain
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Hospital Unversitario Val D'Hebrón
RECRUITINGBarcelona, 8035, Spain
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Hunan Cancer Hospital-thoracic oncology II
RECRUITINGChangsha, Hunan, 410013, China
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IRCCS Istituto Regina Elena Oncologia Medica 2
RECRUITINGRoma, 144, Italy
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Institute Gustave Roussy
RECRUITINGVillejuif, 98405, France
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Jilin Cancer Hospital/Medical Oncology Department
RECRUITINGChangchun, Jilin, 130012, China
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Jilin Cancer Hospital/Medical Oncology Department
RECRUITINGChangchun, Jilin, 130012, China
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Kanagawa cancer center
RECRUITINGYokohama, Kanagawa, 2418515, Japan
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Liaoning Cancer Hospital
RECRUITINGShenyang, Liaoning, 110801, China
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Local Institution - 1001
COMPLETEDOrange, California, 92868, United States
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Local Institution - 1002
ACTIVE_NOT_RECRUITINGNew York, New York, 10065, United States
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Local Institution - 1003
COMPLETEDAurora, Colorado, 80045, United States
-
Local Institution - 1004
COMPLETEDBoston, Massachusetts, 02214, United States
-
Local Institution - 2101
COMPLETEDOrange, California, 92868, United States
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Local Institution - 2102
COMPLETEDNew York, New York, 10065, United States
-
Local Institution - 2103
COMPLETEDAurora, Colorado, 80045, United States
-
Local Institution - 2105
COMPLETEDAnn Arbor, Michigan, 48109, United States
-
Local Institution - 2106
COMPLETEDWashington D.C., District of Columbia, 20007, United States
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Local Institution - 2107
COMPLETEDSeattle, Washington, 98109, United States
-
Local Institution - 2108
COMPLETEDPhiladelphia, Pennsylvania, 19111-2497, United States
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Local Institution - 2109
COMPLETEDCleveland, Ohio, 44195, United States
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Local Institution - 2110
COMPLETEDWashington D.C., District of Columbia, 20016, United States
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Local Institution - 2111
COMPLETEDDetroit, Michigan, 48201, United States
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Local Institution - 2112
COMPLETEDCanton, Ohio, 44718, United States
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Local Institution - 2113
COMPLETEDTampa, Florida, 33612, United States
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Local Institution - 2114
COMPLETEDLa Jolla, California, 92093, United States
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Local Institution - 2116
NOT_YET_RECRUITINGNew Orleans, Louisiana, 70121, United States
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Local Institution - 2117
COMPLETEDNew York, New York, 10016, United States
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Local Institution - 2119
COMPLETEDToledo, Ohio, 43614, United States
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Local Institution - 2120
COMPLETEDGlendale, California, 91206, United States
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Local Institution - 2121
COMPLETEDLong Beach, California, 90813, United States
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Local Institution - 2122
COMPLETEDNew Brunswick, New Jersey, 08901, United States
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Local Institution - 2127
COMPLETEDHouston, Texas, 77030, United States
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Local Institution - 2129
COMPLETEDDuarte, California, 91010, United States
-
Local Institution - 2131
COMPLETEDBoston, Massachusetts, 02215, United States
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Local Institution - 2132
COMPLETEDSaint Paul, Minnesota, 55101, United States
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Local Institution - 2133
COMPLETEDBaltimore, Maryland, 21210, United States
-
Local Institution - 2134
COMPLETEDColumbus, Georgia, 31904, United States
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Local Institution - 2136
WITHDRAWNLa Jolla, California, 92037, United States
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Local Institution - 2137
COMPLETEDFairfax, Virginia, 22031, United States
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Local Institution - 2140
COMPLETEDDetroit, Michigan, 48202-2608, United States
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Local Institution - 2141
WITHDRAWNTacoma, Washington, 98405, United States
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Local Institution - 2142
COMPLETEDPeoria, Illinois, 61615, United States
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Local Institution - 2143
COMPLETEDCincinnati, Ohio, 45220, United States
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Local Institution - 2144
COMPLETEDGoldsboro, North Carolina, 27534, United States
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Local Institution - 2145
COMPLETEDAppleton, Wisconsin, 54911, United States
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Local Institution - 2146
COMPLETEDKingwood, Texas, 77339, United States
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Local Institution - 2147
COMPLETEDBolivar, Missouri, 65613, United States
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Local Institution - 2201
COMPLETEDToronto, Ontario, M5G 2M9, Canada
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Local Institution - 2202
COMPLETEDEdmonton, Alberta, T6G 1Z2, Canada
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Local Institution - 2203
COMPLETEDOntario, L6R 37R, Canada
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Local Institution - 2204
COMPLETEDOttawa, K1H 8L6, Canada
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Local Institution - 2205
WITHDRAWNVancouver, British Columbia, V5Z 4E7, Canada
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Local Institution - 3001
COMPLETEDSeoul, 03080, South Korea
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Local Institution - 3002
COMPLETEDSeoul, Seodaemun-gu, 03722, South Korea
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Local Institution - 3002
WITHDRAWNSeoul, Seoul Teugbyeolsi, 03080, South Korea
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Local Institution - 3003
COMPLETEDSeoul, Gangnam-gu, 06351, South Korea
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Local Institution - 3301
COMPLETEDEast Melbourne, 3002, Australia
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Local Institution - 4102
COMPLETEDBarcelona, 08028, Spain
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Local Institution - 4103
COMPLETEDMadrid, 28041, Spain
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Local Institution - 4106
COMPLETEDMadrid, 28033, Spain
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Local Institution - 4107
COMPLETEDValencia, 46009, Spain
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Local Institution - 4108
COMPLETEDPamplona, 31008, Spain
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Local Institution - 4201
COMPLETEDMarseille, Bouches-du-Rhône, 13005, France
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Local Institution - 4203
COMPLETEDSaint-Mandé, 94163, France
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Local Institution - 4207
COMPLETEDBrest, 29200, France
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Local Institution - 4208
COMPLETEDPoitiers, 86000, France
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Local Institution - 4302
COMPLETEDTerni, 05100, Italy
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Local Institution - 4304
NOT_YET_RECRUITINGRavenna, 48121, Italy
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Local Institution - 4305
COMPLETEDReggio Emilia, 42123, Italy
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Local Institution - 4306
COMPLETEDMilan, 20122, Italy
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Local Institution - 4307
WITHDRAWNPalermo, 90146, Italy
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Local Institution - 4401
COMPLETEDLondon, SW3 6JJ, United Kingdom
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Local Institution - 4402
COMPLETEDLondon, W12 OHS, United Kingdom
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Local Institution - 4403
COMPLETEDManchester, M20 4BX, United Kingdom
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Local Institution - 4404
COMPLETEDLondon, W1G 6AD, United Kingdom
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Local Institution - 4405
COMPLETEDSutton, SM2 5PT, United Kingdom
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Local Institution - 4501
COMPLETEDGroningen, 9713 GZ, Netherlands
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Local Institution - 4502
COMPLETEDAmsterdam, 1066 CX, Netherlands
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Local Institution - 4602
COMPLETEDWarsaw, 02-781, Poland
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Local Institution - 4603
COMPLETEDSzczecin, 70-784, Poland
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Local Institution - 4604
COMPLETEDLublin, 20-609, Poland
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Local Institution - 4605
COMPLETEDPoznan, 60-693, Poland
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Local Institution - 4701
COMPLETEDCologne, 50937, Germany
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Local Institution - 4702
COMPLETEDHeidelberg, 69120, Germany
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Local Institution - 4703
COMPLETEDDresden, 01307, Germany
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Local Institution - 4704
COMPLETEDBerlin, 13125, Germany
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Local Institution - 4801
COMPLETEDLeuven, 3000, Belgium
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Local Institution - 4802
COMPLETEDAntwerp, 2650, Belgium
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Local Institution - 4901
COMPLETEDCopenhagen, 2100, Denmark
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Local Institution - 5101
COMPLETEDBudapest, 1083, Hungary
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Local Institution - 5103
COMPLETEDBudapest, 1121, Hungary
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Local Institution - 6103
COMPLETEDAdelaide, South Australia, 5042, Australia
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Local Institution - 6201
ACTIVE_NOT_RECRUITINGTaiepi, 100, Taiwan
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Local Institution - 6202
COMPLETEDTaipei, 10449, Taiwan
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Local Institution - 6203
ACTIVE_NOT_RECRUITINGTainan, 704, Taiwan
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Local Institution - 6304
COMPLETEDSeoul, 06591, South Korea
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Local Institution - 6306
COMPLETEDCheongju-si, 28644, South Korea
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Local Institution - 6307
COMPLETEDSeoul, 05030, South Korea
-
Local Institution - 6501
ACTIVE_NOT_RECRUITINGHong Kong, Hong Kong
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Local Institution - 6502
ACTIVE_NOT_RECRUITINGHong Kong, 0, Hong Kong
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Local Institution - 6503
ACTIVE_NOT_RECRUITINGToronto, Ontario, M5G 2M9, Canada
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Local Institution - 6504
ACTIVE_NOT_RECRUITINGShatin, HONG KONG, 999077, China
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Local Institution - 6505
ACTIVE_NOT_RECRUITINGShenzhen, Guangdong, 518053, China
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Local Institution - 6702
COMPLETEDBeijing, Beijing Municipality, 100021, China
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Local Institution - 6705
COMPLETEDChangsha, Hunan, 410011, China
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Local Institution - 6710
COMPLETEDWuhan, Hubei, 430022, China
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Local Institution - 6719
COMPLETEDFuzhou, Fujian, 000000, China
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Local Institution - 6725
COMPLETEDHangzhou, Zhejiang, 310016, China
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Local Institution - 6733
COMPLETEDGuangzhou, Guangdong, 510120, China
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Local Institution - 6748
COMPLETEDNanjing, Jiangsu, 210008, China
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MD Anderson Cancer Center
RECRUITINGHouston, Texas, 77030, United States
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Massachusetts General Hospital,
RECRUITINGBoston, Massachusetts, 02114, United States
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Memorial Healthcare System
RECRUITINGHollywood, Florida, 33021, United States
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Nagoya University Hospital
RECRUITINGNagoya, 466-8560, Japan
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National Cancer Center Hospital East
RECRUITINGKashiwa, 277-8577, Japan
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National Cancer Center Hospital.
RECRUITINGChuo-ku, Tokyo, 1040045, Japan
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National Cancer Center Singapore
RECRUITINGSingapore, 169610, Singapore
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National University Hospital
RECRUITINGSingapore, 119074, Singapore
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Osaka City General Hospital
RECRUITINGOsaka, Osaka, 5340021, Japan
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Osaka International Cancer institute
RECRUITINGOsaka, 5418567, Japan
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Ośrodek Badań Klinicznych Wczesnych Faz, Uniwersyteckie Centrum Kliniczne
RECRUITINGGdansk, 80-214, Poland
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Peter MacCallum Cancer Centre
RECRUITINGMelbourne, Victoria, 3000, Australia
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START Madrid-FJD
RECRUITINGMadrid, 28040, Spain
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Samsung Medical Center
RECRUITINGSeoul, Seoul-teukbyeolsi [Seoul], 06351, South Korea
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Seoul National University Hospital
RECRUITINGSeoul, Seoul-teukbyeolsi [Seoul], 03080, South Korea
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Shanghai Chest Hospital
RECRUITINGShanghai, 200030, China
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Shanghai Chest Hospital
RECRUITINGShanghai, 200030, China
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Shanxi Bethune Hospital
RECRUITINGTaiyuan, Shanxi, 030032, China
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Sichuan Cancer Hospital/Medical Oncology Department
RECRUITINGChengdu, Sichuan, 00000, China
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St Joseph Heritage Healthcare
RECRUITINGSanta Rosa, California, 95403, United States
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Tangdu Hospital
RECRUITINGXi'an, Shan3xi, 710038, China
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The Affiliated Tumor Hospital of Harbin Medical University
RECRUITINGHarbin, Heilongjiang, 150081, China
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The First Affiliated Hospital - Zhejiang University School of Medicine
RECRUITINGHangzhou, 310003, China
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The First Affiliated hospital of Xiamen University-oncology
RECRUITINGXiamen, Fujian, 361003, China
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The First Hospital Affiliated To AMU - Southwest Hospital
RECRUITINGChongqing, Sichuan, 400030, China
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The Ohio State University Wexner Medical Center
RECRUITINGColumbus, Ohio, 43210, United States
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The Third Xiangya Hospital of Central South University/Department of Respiratory and Critical Care Medicine
RECRUITINGChangsha, 00000, China
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The first hospital of Jilin university-Oncology Department
RECRUITINGChangchun, Jilin, 130021, China
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Tottori University Hospital
RECRUITINGYonago, Tottori, 683-8504, Japan
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UT Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
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University Cancer and Blood Center
RECRUITINGAthens, Georgia, 30607, United States
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University of Chicago
RECRUITINGChicago, Illinois, 60637, United States
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Washington University Infusion Center Pharmacy
RECRUITINGSt Louis, Missouri, 63110, United States
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Weifang People's Hospital/Medical Oncology Department
RECRUITINGWeifang, 00000, China
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West China Hospital Sichuan University/Lung cancer center
RECRUITINGChengdu, 00000, China
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XuZhou Central Hospital/Oncology Department
RECRUITINGXuzhou, Jiangsu, 00000, China
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Yonsei University Health System, Severance Hospital
RECRUITINGSeoul, 03722, South Korea
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Zhejiang Cancer Hospital-Oncology
RECRUITINGHangzhou, Zhejiang, 310022, China
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