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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-000073-12 | EudraCT Number |
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Business decision
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This study will compare the efficacy and safety of unesbulin plus dacarbazine versus placebo plus dacarbazine in participants with unresectable or metastatic, relapsed or refractory LMS who have received at least 1 prior line of systemic therapy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Unesbulin and Dacarbazine | Experimental | Participants will receive unesbulin 300 milligrams (mg) tablets administered orally twice weekly in each 3-week treatment cycle in combination with dacarbazine 1000 mg/meter squared (m^2) intravenously (IV) once every 21 days. Treatment will continue for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason. |
|
| Placebo and Dacarbazine | Placebo Comparator | Participants will receive placebo matching to unesbulin tablets administered orally twice weekly in each 3-week treatment cycle in combination with dacarbazine 1000 mg/m^2 IV once every 21 days. Treatment will continue for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Unesbulin | Drug | Unesbulin will be administered as per the dose and schedule specified in the arm description. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Progression Free Survival (PFS) Per Independent Central Review Using Response Evaluation Criteria in Solid Tumors (RECIST) V1.1 | PFS was defined as the time from randomization to the documented disease progression or death due to any cause, whichever occurred first. Disease progression was defined as at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 millimeters (mm). The appearance of one or more new lesions was also considered progression. | Up to approximately 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival | Overall survival was defined as the time in months from the randomization date to the date of death from any cause or date last known alive for those who did not die. | Up to approximately 2 years |
| Objective Response Rate (ORR) Per Independent Central Review Using RECIST V1.1 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other inclusion and exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| Mark Rance, MD | PTC Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| City of Hope | Duarte | California | 91010 | United States | ||
| University of California, Los Angeles (UCLA) - Jonsson Comprehensive Cancer Center |
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Participants were randomized 2:1 to 1 of the following treatment groups: 1. Unesbulin and Dacarbazine or 2. Placebo and Dacarbazine.
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| ID | Title | Description |
|---|---|---|
| FG000 | Unesbulin and Dacarbazine | Participants received unesbulin 300 milligrams (mg) tablets administered orally twice weekly in each 3-week treatment cycle in combination with dacarbazine 1000 mg/square meter (m^2) intravenously (IV) once every 21 days. Treatment was continued for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 28, 2023 | Jun 3, 2025 |
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| Dacarbazine | Drug | Dacarbazine will be administered as per the dose and schedule specified in the arm description. |
|
|
| Placebo | Other | Placebo will be administered as per the schedule specified in the arm description. |
|
ORR was defined as percentage of participants who achieved a confirmed best overall response (BOR) of complete response (CR) or partial response (PR). CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. |
| Up to approximately 2 years |
| Disease Control Rate (DCR) Per Independent Central Review Using RECIST V1.1 | DCR was defined as percentage of participants who achieved a confirmed BOR of CR, PR, or at least 3 months of stable disease (SD). CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. SD: Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study. | Up to approximately 2 years |
| Duration of Response Per Independent Central Review Using RECIST V1.1 | Duration of response was defined as the time from the date of first confirmed response of CR or PR to the date of the first documented disease progression or death due to any cause, whichever occurred first. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. Disease progression: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions was also considered progression. | Up to approximately 2 years |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A TEAE was defined as an AE that had an onset date on or after the first dose of study drug until 30 days after last dose or occurred prior to first dose of study drug and worsened in severity after first dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. | From first dose of study drug up to approximately 2 years |
| Los Angeles |
| California |
| 90024 |
| United States |
| Sarcoma Oncology Research Center | Santa Monica | California | 90403 | United States |
| Stanford Cancer Center | Stanford | California | 94305 | United States |
| University of Colorado Denver | Denver | Colorado | 80045 | United States |
| Yale University | New Haven | Connecticut | 06511 | United States |
| Mayo Clinic | Jacksonville | Florida | 32224 | United States |
| University of Florida (UF) Health Cancer Center - Orlando Health | Orlando | Florida | 32806 | United States |
| Moffitt | Tampa | Florida | 33612 | United States |
| Northwestern Memorial Hospital | Chicago | Illinois | 60611 | United States |
| Northwestern Medicine - Warrenville Cancer Center | Warrenville | Illinois | 60555 | United States |
| Johns Hopkins Oncology Group | Baltimore | Maryland | 21231 | United States |
| University of Michigan | Ann Arbor | Michigan | 48109 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| The Trustees of Columbia University | New York | New York | 10027 | United States |
| David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center | New York | New York | 11101 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| The Ohio State University (OSU) | Columbus | Ohio | 43210 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | United States |
| Thomas Jefferson University | Philadelphia | Pennsylvania | 19144 | United States |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15232 | United States |
| MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
| Chris O'Brien Lifehouse | Camperdown | 2050 | Australia |
| Peter MacCallum Cancer Institute | East Melbourne | 3000 | Australia |
| Prince of Wales Hospital | Randwick | 2031 | Australia |
| Fundacao PIO XII - Hospital de Amor | Barretos | Brazil |
| Santa Casa de Misericordia de Porto Alegre | Porto Alegre | Brazil |
| INCA I - Instituto Nacional de Cancer | Rio de Janeiro | 20220-410 | Brazil |
| Hospital Sao Rafael - Instituto D'Or da Bahia | Salvador | Brazil |
| CIP - Centro Integrado de Pesquisas do Hospital de Base de Sao Jose do Rio Preto | São José do Rio Preto | Brazil |
| Instituto do Cancer do Estado de São Paulo (ICESP) | São Paulo | Brazil |
| The Ottawa Hospital Cancer Centre | Ottawa | Ontario | K1H8L6 | Canada |
| Princess Margaret Hospital | Toronto | Ontario | Canada |
| Universite de Montreal - Hopital Maisonneuve-Rosemont (HMR) | Montreal | Quebec | Canada |
| Institut Bergonie | Bordeaux | 33076 | France |
| Centre Leon Berard | Lyon | 69008 | France |
| Institut Curie | Paris | 75005 | France |
| Gustave Roussy | Villejuif | France |
| Universitaetsmedizin Mannheim | Mannheim | 68167 | Germany |
| Klinikum der Ludwig-Maximilians-Universitaet Muenchen | München | 81377 | Germany |
| Eszak-Pesti Centrumkorhaz - Honvedkorhaz | Budapest | Hungary |
| Fondazione IRCCS Istituto Nazionale dei Tumori | Milan | 20133 | Italy |
| La Fondazione e l'Istituto di Candiolo | Torino | 10060 | Italy |
| Leids Universitair Medisch Centrum | Leiden | Netherlands |
| Niepubliczny Zaklad Opieki Zdrowotnej Zespól Poradni Specjalistycznych "TERMEDICA" | Poznan | Poland |
| Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie - Panstwowy Instytut Badawczy, Klinika Nowotworow Tkanek Miekkich, Kosci i Czerniakow | Warsaw | 02-781 | Poland |
| Institut Catala d'Oncologia (Hospital Duran y Reynals) | Barcelona | 08908 | Spain |
| Hospital Universitario Vall d'Hebron | Barcelona | Spain |
| Hospital Fundacion Jimenez Diaz | Madrid | 28040 | Spain |
| Hospital Universitario 12 de Octubre | Madrid | Spain |
| Beatson, West of Scotland Cancer Centre | Glasgow | G12 0YN | United Kingdom |
| Royal Marsden Hospital | London | SW3 6JJ | United Kingdom |
| The Christie NHS Foundation Trust | Manchester | United Kingdom |
| FG001 | Placebo and Dacarbazine | Participants received placebo matching to unesbulin tablets administered orally twice weekly in each 3-week treatment cycle in combination with dacarbazine 1000 mg/m^2 IV once every 21 days. Treatment was continued for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason. |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
The safety analysis set included all participants who received at least 1 dose of study drug (unesbulin/placebo or dacarbazine).
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| ID | Title | Description |
|---|---|---|
| BG000 | Unesbulin and Dacarbazine | Participants received unesbulin 300 mg tablets administered orally twice weekly in each 3-week treatment cycle in combination with dacarbazine 1000 mg/m^2 IV once every 21 days. Treatment was continued for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason. |
| BG001 | Placebo and Dacarbazine | Participants received placebo matching to unesbulin tablets administered orally twice weekly in each 3-week treatment cycle in combination with dacarbazine 1000 mg/m^2 IV once every 21 days. Treatment was continued for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Progression Free Survival (PFS) Per Independent Central Review Using Response Evaluation Criteria in Solid Tumors (RECIST) V1.1 | PFS was defined as the time from randomization to the documented disease progression or death due to any cause, whichever occurred first. Disease progression was defined as at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 millimeters (mm). The appearance of one or more new lesions was also considered progression. | The modified intent-to-treat (mITT) set included randomized participants with 1 to 3 prior lines of therapy. | Posted | Median | 95% Confidence Interval | months | Up to approximately 2 years |
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| Secondary | Overall Survival | Overall survival was defined as the time in months from the randomization date to the date of death from any cause or date last known alive for those who did not die. | The mITT set included randomized participants with 1 to 3 prior lines of therapy. | Posted | Median | 95% Confidence Interval | months | Up to approximately 2 years |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Objective Response Rate (ORR) Per Independent Central Review Using RECIST V1.1 | ORR was defined as percentage of participants who achieved a confirmed best overall response (BOR) of complete response (CR) or partial response (PR). CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. | The mITT set included randomized participants with 1 to 3 prior lines of therapy. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to approximately 2 years |
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| Secondary | Disease Control Rate (DCR) Per Independent Central Review Using RECIST V1.1 | DCR was defined as percentage of participants who achieved a confirmed BOR of CR, PR, or at least 3 months of stable disease (SD). CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. SD: Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study. | The mITT set included randomized participants with 1 to 3 prior lines of therapy. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to approximately 2 years |
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| Secondary | Duration of Response Per Independent Central Review Using RECIST V1.1 | Duration of response was defined as the time from the date of first confirmed response of CR or PR to the date of the first documented disease progression or death due to any cause, whichever occurred first. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. Disease progression: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions was also considered progression. | The mITT set included randomized participants with 1 to 3 prior lines of therapy. | Posted | Median | 95% Confidence Interval | months | Up to approximately 2 years |
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| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A TEAE was defined as an AE that had an onset date on or after the first dose of study drug until 30 days after last dose or occurred prior to first dose of study drug and worsened in severity after first dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. | The safety analysis set included all participants who received at least 1 dose of study drug (unesbulin/placebo or dacarbazine). | Posted | Count of Participants | Participants | From first dose of study drug up to approximately 2 years |
|
From first dose of study drug up to approximately 2 years
The safety analysis set included all participants who received at least 1 dose of study drug (unesbulin/placebo or dacarbazine).
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Unesbulin and Dacarbazine | Participants received unesbulin 300 mg tablets administered orally twice weekly in each 3-week treatment cycle in combination with dacarbazine 1000 mg/m^2 IV once every 21 days. Treatment was continued for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason. | 49 | 238 | 81 | 238 | 234 | 238 |
| EG001 | Placebo and Dacarbazine | Participants received placebo matching to unesbulin tablets administered orally twice weekly in each 3-week treatment cycle in combination with dacarbazine 1000 mg/m^2 IV once every 21 days. Treatment was continued for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason. | 27 | 120 | 43 | 120 | 112 | 120 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 25.0 | Systematic Assessment |
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| Bicytopenia | Blood and lymphatic system disorders | MedDRA 25.0 | Systematic Assessment |
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| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 25.0 | Systematic Assessment |
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| Haematotoxicity | Blood and lymphatic system disorders | MedDRA 25.0 | Systematic Assessment |
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| Neutropenia | Blood and lymphatic system disorders | MedDRA 25.0 | Systematic Assessment |
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| Pancytopenia | Blood and lymphatic system disorders | MedDRA 25.0 | Systematic Assessment |
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| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 25.0 | Systematic Assessment |
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| Angina pectoris | Cardiac disorders | MedDRA 25.0 | Systematic Assessment |
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| Atrial fibrillation | Cardiac disorders | MedDRA 25.0 | Systematic Assessment |
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| Atrial flutter | Cardiac disorders | MedDRA 25.0 | Systematic Assessment |
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| Atrial tachycardia | Cardiac disorders | MedDRA 25.0 | Systematic Assessment |
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| Atrial thrombosis | Cardiac disorders | MedDRA 25.0 | Systematic Assessment |
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| Bundle branch block left | Cardiac disorders | MedDRA 25.0 | Systematic Assessment |
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| Cardiac failure | Cardiac disorders | MedDRA 25.0 | Systematic Assessment |
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| Myocardial infarction | Cardiac disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Supraventricular tachycardia | Cardiac disorders | MedDRA 25.0 | Systematic Assessment |
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| Tachycardia | Cardiac disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Aplasia | Congenital, familial and genetic disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Ascites | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Haemoperitoneum | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Intestinal perforation | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Large intestine perforation | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Asthenia | General disorders | MedDRA 25.0 | Systematic Assessment |
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| Disease progression | General disorders | MedDRA 25.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 25.0 | Systematic Assessment |
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| General physical health deterioration | General disorders | MedDRA 25.0 | Systematic Assessment |
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| Malaise | General disorders | MedDRA 25.0 | Systematic Assessment |
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| Non-cardiac chest pain | General disorders | MedDRA 25.0 | Systematic Assessment |
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| Pain | General disorders | MedDRA 25.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 25.0 | Systematic Assessment |
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| Systemic inflammatory response syndrome | General disorders | MedDRA 25.0 | Systematic Assessment |
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| Hepatic failure | Hepatobiliary disorders | MedDRA 25.0 | Systematic Assessment |
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| Subcapsular hepatic haematoma | Hepatobiliary disorders | MedDRA 25.0 | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Cystitis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Device related sepsis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Escherichia urinary tract infection | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Pertussis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Pyelonephritis acute | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Septic shock | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Soft tissue infection | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Spinal cord infection | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Vascular device infection | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Accidental overdose | Injury, poisoning and procedural complications | MedDRA 25.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 25.0 | Systematic Assessment |
| |
| Hip fracture | Injury, poisoning and procedural complications | MedDRA 25.0 | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA 25.0 | Systematic Assessment |
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| Overdose | Injury, poisoning and procedural complications | MedDRA 25.0 | Systematic Assessment |
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| Toxicity to various agents | Injury, poisoning and procedural complications | MedDRA 25.0 | Systematic Assessment |
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| Blood bilirubin increased | Investigations | MedDRA 25.0 | Systematic Assessment |
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| Electrocardiogram QT prolonged | Investigations | MedDRA 25.0 | Systematic Assessment |
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| Neutrophil count decreased | Investigations | MedDRA 25.0 | Systematic Assessment |
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| Platelet count decreased | Investigations | MedDRA 25.0 | Systematic Assessment |
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| Transaminases increased | Investigations | MedDRA 25.0 | Systematic Assessment |
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| White blood cell count decreased | Investigations | MedDRA 25.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Malnutrition | Metabolism and nutrition disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 25.0 | Systematic Assessment |
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| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Malignant neoplasm progression | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.0 | Systematic Assessment |
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| Metastases to lung | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.0 | Systematic Assessment |
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| Tumour haemorrhage | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.0 | Systematic Assessment |
| |
| Tumour pain | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.0 | Systematic Assessment |
| |
| Brain oedema | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Central nervous system lesion | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Facial paralysis | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Neurotoxicity | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Spinal cord compression | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Device occlusion | Product Issues | MedDRA 25.0 | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Urinary tract obstruction | Renal and urinary disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Vaginal haemorrhage | Reproductive system and breast disorders | MedDRA 25.0 | Systematic Assessment | This is a sex-specific AE. Only female participants were at risk. |
|
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 25.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 25.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 25.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 25.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 25.0 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 25.0 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA 25.0 | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA 25.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
|
The study was terminated early due to business decision.
The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the Sponsor for review. The Sponsor may consult with the PI to require changes to the communication or extend the embargo.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Patient Advocacy | PTC Therapeutics, Inc. | 1-866-562-4620 | medinfo@ptcbio.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 25, 2024 | Jun 3, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D007890 | Leiomyosarcoma |
| ID | Term |
|---|---|
| D009379 | Neoplasms, Muscle Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D012509 | Sarcoma |
Not provided
Not provided
| ID | Term |
|---|---|
| C000712133 | PTC596 |
| D003606 | Dacarbazine |
| ID | Term |
|---|---|
| D014226 | Triazenes |
| D009930 | Organic Chemicals |
| D007093 | Imidazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| American Indian/Alaska Native |
|
| Black/African American |
|
| Native Hawaiian/Other Pacific Islander |
|
| White/Caucasian |
|
| Other |
|
| Missing |
|
| Multiple |
|
| Participants |
|
|
|
|
|
|
|
|
Participants received placebo matching to unesbulin tablets administered orally twice weekly in each 3-week treatment cycle in combination with dacarbazine 1000 mg/m^2 IV once every 21 days. Treatment was continued for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason. |
|
|