Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2019-002463-10 | EudraCT Number | ||
| BLU-667-2303 | Registry Identifier | CT.Gov | |
| 2023-505035-12-00 | Other Identifier | EU CT number |
Not provided
Not provided
Early termination of the study resulted from organizational and commercial decisions that led to the discontinuation of pralsetinib's global marketing and development in all territories (excluding US and Greater China).
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is an international, randomized, open-label, Phase 3 study designed to evaluate whether the potent and selective RET inhibitor, pralsetinib, improves outcomes when compared to a platinum chemotherapy-based regimen chosen by the Investigator from a list of standard of care treatments, as measured primarily by progression free survival (PFS), for participants with RET fusion-positive metastatic NSCLC who have not previously received systemic anticancer therapy for metastatic disease.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pralsetinib | Experimental | Participants randomized to the Experimental Arm will receive Pralsetinib |
|
| Platinum-based chemotherapy with or without pembrolizumab | Active Comparator | Participants randomized to the Active Comparator Arm will receive 1 of 6 platinum-based chemotherapy treatment regimens (with or without pembrolizumab) at the study center as chosen by the treating Investigator (based on histology) Nonsquamous histology
Squamous histology
|
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pralsetinib | Drug | Administered orally |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Arm A vs Arm B: Treatment Period: Progression-free Survival (PFS) | PFS was defined as the time from randomization to the date of first documented PD as determined by the investigator with the use of Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST v1.1) or death from any cause, whichever occurred first. PD was defined as at least a 20% increase in the sum of diameters (SOD) of target lesions, taking as reference the smallest SOD at prior timepoints, including baseline. In addition to the relative increase of 20%, the SOD must also demonstrate an absolute increase of at least 5 millimeters (mm). Kaplan-Meier (K-M) method was used to estimate median PFS. 95% CI for median was computed using the method of Brookmeyer and Crowley. | Up to approximately 50 months |
| Measure | Description | Time Frame |
|---|---|---|
| Arm A vs Arm B: Objective Response Rate (ORR) | ORR was defined as the percentage of participants with a complete response (CR) or a partial response (PR) on two consecutive occasions ≥4 weeks apart, as determined by the investigator with the use of RECIST v1.1. CR was defined as disappearance of all target lesions or any pathological lymph nodes must have reduction in short axis to < 10 mm. PR was defined as at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in the absence of CR. 95% CI for rates were constructed using the Clopper-Pearson method. Percentages have been rounded off. |
Not provided
Inclusion criteria:
Participant has pathologically confirmed, definitively diagnosed, locally advanced (not able to be treated with surgery or radiotherapy) or metastatic NSCLC and has not been treated with systemic anticancer therapy for metastatic disease.
Participant must have a documented RET-fusion
Participant has measurable disease based on RECIST 1.1 as determined by the local site Investigator/radiology assessment.
Participant has an ECOG Performance Status of 0 or 1.
Participant should not have received any prior anticancer therapy for metastatic disease.
Participant is an appropriate candidate for and agrees to receive 1 of the Investigator choice platinum-based chemotherapy regimens if randomized to Arm B.
For women of childbearing potential: participants who agree to remain abstinent (refrain from heterosexual intercourse) or use contraception.
For men: participants who agree to remain abstinent (refrain from heterosexual intercourse) or use a condom and agree to refrain from donating sperm.
Exclusion criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital Britanico | Buenos Aires | C1280AEB | Argentina | |||
| Centro Oncologico Riojano Integral (CORI) |
For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
Not provided
Not provided
Not provided
Not provided
Participants in treatment period were randomized in 1:1 ratio to receive standard of care (SOC) platinum containing anticancer treatment regimens (Arm A) or pralsetinib (Arm B). Participants with disease progression (PD) in Arm A had the option to crossover & receive pralsetinib (Arm C), which was later discontinued as of Protocol Version 5. 11 randomized participants did not receive any study treatment and were excluded from safety analysis.
A total of 223 participants with rearranged during transfection (RET) fusion-positive metastatic non-small cell lung cancer (NSCLC) took part in the study at 74 investigative sites across 22 countries from 24 July 2020 to 27 January 2025. The study consists of a treatment period and a crossover period.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Arm A: Treatment Period- SOC | Participants received platinum-containing anticancer treatment regimens (with or without pembrolizumab) at the study center as chosen by the treating investigator based on NSCLC histology. Participants with NSCLC of non-squamous histology received carboplatin + pemetrexed or cisplatin + pemetrexed or pembrolizumab + carboplatin + pemetrexed or pembrolizumab + cisplatin + pemetrexed. Participants with NSCLC of squamous histology received carboplatin or cisplatin + gemcitabine or carboplatin + paclitaxel/nab-paclitaxel + pembrolizumab until PD or for up to a maximum of 2 years. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Treatment Period |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Oct 23, 2024 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Carboplatin | Drug | Administered IV |
|
| Cisplatin | Drug | Administered IV |
|
| Pemetrexed | Drug | Administered IV |
|
| Pembrolizumab | Drug | Administered IV |
|
| Gemcitabine | Drug | Administered IV |
|
| Paclitaxel | Drug | Administered IV |
|
| Nab-Paclitaxel | Drug | Administered IV |
|
| Up to approximately 50 months |
| Arm A vs Arm B: Overall Survival (OS) | OS was defined as the time from randomization to death from any cause. K-M method was used to estimate median OS. 95% CI for median was computed using the method of Brookmeyer and Crowley. | From randomization to death (up to approximately 50 months) |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. SAE is any significant hazard, contraindication, side effect that is fatal or life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is medically significant or requires intervention to prevent one or other of the outcomes listed above. | Up to approximately 50 months |
| Number of Participants With Change From Baseline in Eastern Cooperative Oncology Group Performance Status (ECOG PS) Score | ECOG is a 6-point scale (0-5) used to assess participants functional status, where, 0= Fully active, able to carry on all pre-disease performance without restriction; 1= restricted in physically strenuous activity but ambulatory & able to carry out work of a light or sedentary nature, e.g., light housework, office work; 2= ambulatory & capable of all self-care but unable to carry out any work activities. Up & about more than 50% of waking hours; 3= capable of only limited self-care, confined to bed or chair more than 50% of waking hours; 4= completely disabled, cannot carry on any self-care, totally confined to bed or chair; 5= dead. | Baseline up to 50 months |
| Arm A vs Arm B: Duration of Response (DOR) | DOR was defined as the time from the first occurrence of a documented objective response (OR) to PD or death from any cause, whichever occurred first, as determined by the investigator with the use of RECIST v1.1. CR was defined as disappearance of all target lesions or any pathological lymph nodes must have reduction in short axis to < 10 mm. PR was defined as at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in the absence of CR. PD was defined as at least a 20% increase in the SOD of target lesions, taking as reference the smallest SOD at prior timepoints, including baseline. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. K-M method was used to estimate median DOR. 95% CI for median was computed using the method of Brookmeyer and Crowley. | Up to approximately 50 months |
| Arm A vs Arm B: Clinical Benefit Rate (CBR) | CBR was defined as the percentage of participants who experienced the best response of stable disease (SD) with a minimum duration of 6 months, a CR, or a PR, as assessed by investigator with use of RECIST v1.1. SD was defined as neither sufficient shrinkage to qualify for CR or PR nor sufficient increase to qualify for PD. CR was defined as the disappearance of all target lesions or any pathological lymph nodes must have reduction in short axis to < 10 mm. PR was defined as at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in the absence of CR. PD was defined as at least a 20% increase in the SOD of target lesions, taking as reference the smallest SOD at prior timepoints, including baseline. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. 95% CI for rates were constructed using the Clopper-Pearson method. Percentages are rounded off. | Up to approximately 50 months |
| Arm A vs Arm B: Disease Control Rate (DCR) | DCR was defined as the percentage of participants who experienced the best response of CR, or PR, or SD, as assessed by investigator according to RECIST v1.1. SD was defined as neither sufficient shrinkage to qualify for CR or PR nor sufficient increase to qualify for PD. CR was defined as the disappearance of all target lesions or any pathological lymph nodes must have reduction in short axis to <10 mm. PR was defined as at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in the absence of CR. PD was defined as at least a 20% increase in the SOD of target lesions, taking as reference the smallest SOD at prior timepoints, including baseline. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. 95% CI for rates were constructed using the Clopper-Pearson method. Percentages are rounded off. | Up to approximately 50 months |
| La Rioja |
| F5300COE |
| Argentina |
| Royal North Shore Hospital | St Leonards | New South Wales | 2065 | Australia |
| UZ Antwerpen | Edegem | 2650 | Belgium |
| Hospital Sao Lucas - PUCRS | Porto Alegre | Rio Grande do Sul | 90610-000 | Brazil |
| Hospital A. C. Camargo | São Paulo | São Paulo | 01509-010 | Brazil |
| Clinica CIMCA | San José | 10103 | Costa Rica |
| Institut Bergonie CLCC Bordeaux | Bordeaux | 33000 | France |
| Hôpital Ambroise Paré - Boulogne-Billancourt | Boulogne-Billancourt | 92100 | France |
| Hôpital Louis Pradel, Hospices Civils de Lyon | Bron | 69677 | France |
| CHRU Lille Service de Pneumologie et Oncologie Thoracique | Lille | 59000 | France |
| Institut Paoli Calmettes | Marseille | 13273 | France |
| Hopital Bichat Claude Bernard | Paris | 75018 | France |
| Hopital Tenon | Paris | 75970 | France |
| Hopital de Pontchaillou | Rennes | 35033 | France |
| Ico Rene Gauducheau | Saint-Herblain | 44805 | France |
| CHU Strasbourg - Nouvel Hopital Civil | Strasbourg | 67091 | France |
| CHU de Toulouse - Hôpital Larrey | Toulouse | 31100 | France |
| Institut Gustave Roussy | Villejuif | 94805 | France |
| Universitätsklinikum Carl Gustav Carus, Medizinische Klinik I, Pneumologie MK1-A13 | Dresden | 01307 | Germany |
| Asklepios-Fachkliniken Muenchen-Gauting | Gauting | 82131 | Germany |
| Pius-Hospital | Oldenburg | 26121 | Germany |
| Leopoldina-Krankenhaus Medizinische Klinik II | Schweinfurt | 97422 | Germany |
| Klinik Schillerhöhe | Stuttgart | 70376 | Germany |
| St. James Hospital | Dublin | D08 HNY1 | Ireland |
| Ospedale Clinicizzato SS Annunziata | Chieti | Abruzzo | 66100 | Italy |
| IRCCS Giovanni Paolo II Istituto Oncologico | Bari | Apulia | 70124 | Italy |
| Istituto Nazionale per lo Studio e la Cura dei Tumori Fondazione G. Pascale | Naples | Campania | 80131 | Italy |
| Ospedale Provinciale Santa Maria Delle Croci | Ravenna | Emilia-Romagna | 48100 | Italy |
| Istituto Nazionale Tumori Regina Elena | Rome | Lazio | 00144 | Italy |
| AZ. Ospedaliera San Giovanni - Addolorata | Rome | Lazio | 00184 | Italy |
| Irccs Ospedale San Raffaele | Milan | Lombardy | 20132 | Italy |
| Fondazione IRCCS Istituto Nazionale dei Tumori | Milan | Lombardy | 20133 | Italy |
| Istituto Europeo Di Oncologia | Milan | Lombardy | 20141 | Italy |
| Azienda Ospedaliera Universitaria Pisana - Ospedale Cisanello | Pisa | Tuscany | 56124 | Italy |
| IOV - Istituto Oncologico Veneto - IRCCS | Padova | Veneto | 35128 | Italy |
| A.O.U. INTEGRATA DI VERONA-Ospedale Civile Maggiore Borgo Trento | Verona | Veneto | 37126 | Italy |
| Kyushu University Hospital | Fukuoka | 812-8582 | Japan |
| National Hospital Organization Himeji Medical Center | Hyōgo | 670-8520 | Japan |
| Osaka International Cancer Institute | Osaka | 541-8567 | Japan |
| Kansai Medical University Hospital | Osaka | 573-1191 | Japan |
| Juntendo University Hospital | Tokyo | 113-8431 | Japan |
| The Cancer Institute Hospital of JFCR | Tokyo | 135-8550 | Japan |
| National Hospital Organization Yamaguchi - Ube Medical Center | Yamaguchi | 755-0241 | Japan |
| Health Pharma Professional Research | Mexico City | Mexico CITY (federal District) | 03100 | Mexico |
| NKI/AvL | Amsterdam | 1066 CX | Netherlands |
| Maastricht University Medical Center | Maastricht | 6229 HX | Netherlands |
| Oslo universitetssykehus HF, Ullevål, Kreftsenteret | Oslo | 0450 | Norway |
| Hemato Oncología de Panamá Especializada | Panama City | 0801 | Panama |
| Narod.Inst.Onkol. im. M.Sklodowskiej - Curie-Panst.Inst.Bad | Warsaw | 02-781 | Poland |
| IPO do Porto | Porto | 4200-072 | Portugal |
| National Cancer Center | Goyang-si | 10408 | South Korea |
| Seoul National University Hospital | Seoul | 03080 | South Korea |
| Severance Hospital, Yonsei University Health System | Seoul | 03722 | South Korea |
| Asan Medical Center | Seoul | 05505 | South Korea |
| Insititut Catala D'Oncologia | L'Hospitalet de Llobregat | Barcelona | 08908 | Spain |
| Hospital Universitario Marques de Valdecilla | Santander | Cantabria | 39008 | Spain |
| Complejo Hospitalario Universitario A Coruña | A Coruña | LA Coruna | 15006 | Spain |
| Hospital Universitari Vall d'Hebron | Barcelona | 08035 | Spain |
| Hospital General Universitario Gregorio Marañon | Madrid | 28007 | Spain |
| Hospital Ramon y Cajal | Madrid | 28034 | Spain |
| Hospital de Madrid Norte Sanchinarro- Centro Integral Oncologico Clara Campal | Madrid | 28050 | Spain |
| Hospital Regional Universitario de Malaga | Málaga | 29010 | Spain |
| Hosp Clinico Univ Lozano Blesa | Zaragoza | 50009 | Spain |
| Karolinska Universitetssjukhuset, Solna | Stockholm | 171 76 | Sweden |
| UniversitätsSpital Zürich | Zurich | 8091 | Switzerland |
| Adana City Hospital, Medical Oncology | Adana | 01060 | Turkey (Türkiye) |
| Ankara Bilkent City Hospital | Ankara | 06490 | Turkey (Türkiye) |
| ?zmir Medical Point | Kar?iyaka | 35575 | Turkey (Türkiye) |
| Velindre Cancer Centre | Cardiff | CF14 2TL | United Kingdom |
| Leicester Royal Infirmary | Leicester | LE1 5WW | United Kingdom |
| Guys & St Thomas Hospital | London | SE1 9RT | United Kingdom |
| Royal Marsden Hospital | London | SW3 6JJ | United Kingdom |
| Christie Hospital Nhs Trust | Manchester | M2O 4BX | United Kingdom |
| FG001 | Arm B: Treatment Period- Pralsetinib | Participants received pralsetinib, 400 milligrams (mg), orally, once a day (QD) on Day 1 of each cycle until PD, post-trial access program (PTAP), pralsetinib was no longer available, unacceptable toxicity, non-compliance, withdrawal of consent, closure of the study by the sponsor, or death, whichever occurred first (cycle length=21 days). |
| FG002 | Arm C: Crossover Period- Pralsetinib | Participants with confirmed PD as confirmed by blinded independent central review (BICR) in Arm A crossed over to receive pralsetinib, 400 mg, orally, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity or death, whichever occurred first (cycle length=21 days). |
| Safety-evaluable Population | Safety-evaluable population included all participants who received any amount of any study drug. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Crossover Period |
|
|
Intent-to-Treat (ITT) population included all randomized participants whether or not the assigned study treatment is received.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Arm A: Treatment Period- SOC | Participants received platinum-containing anticancer treatment regimens (with or without pembrolizumab) at the study center as chosen by the treating investigator based on NSCLC histology. Participants with NSCLC of non-squamous histology received carboplatin + pemetrexed or cisplatin + pemetrexed or pembrolizumab + carboplatin + pemetrexed or pembrolizumab + cisplatin + pemetrexed. Participants with NSCLC of squamous histology received carboplatin or cisplatin + gemcitabine or carboplatin + paclitaxel/nab-paclitaxel + pembrolizumab until PD or for up to a maximum of 2 years. |
| BG001 | Arm B: Treatment Period- Pralsetinib | Participants received pralsetinib, 400 mg, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity, non-compliance, withdrawal of consent, closure of the study by the sponsor, or death, whichever occurred first (cycle length=21 days). |
| 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 | Arm A vs Arm B: Treatment Period: Progression-free Survival (PFS) | PFS was defined as the time from randomization to the date of first documented PD as determined by the investigator with the use of Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST v1.1) or death from any cause, whichever occurred first. PD was defined as at least a 20% increase in the sum of diameters (SOD) of target lesions, taking as reference the smallest SOD at prior timepoints, including baseline. In addition to the relative increase of 20%, the SOD must also demonstrate an absolute increase of at least 5 millimeters (mm). Kaplan-Meier (K-M) method was used to estimate median PFS. 95% CI for median was computed using the method of Brookmeyer and Crowley. | ITT population included all randomized participants whether or not the assigned study treatment was received. As specified in the protocol, assessment of this outcome measure was limited to Arms A and B, as the objective was to compare pralsetinib with SOC platinum-containing anticancer treatment regimens. | Posted | Median | 95% Confidence Interval | months | Up to approximately 50 months |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Arm A vs Arm B: Objective Response Rate (ORR) | ORR was defined as the percentage of participants with a complete response (CR) or a partial response (PR) on two consecutive occasions ≥4 weeks apart, as determined by the investigator with the use of RECIST v1.1. CR was defined as disappearance of all target lesions or any pathological lymph nodes must have reduction in short axis to < 10 mm. PR was defined as at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in the absence of CR. 95% CI for rates were constructed using the Clopper-Pearson method. Percentages have been rounded off. | ITT population included all randomized participants whether or not the assigned study treatment is received. As specified in the protocol, assessment of this outcome measure was limited to Arms A and B, as the objective was to compare pralsetinib with SOC platinum-containing anticancer treatment regimens. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to approximately 50 months |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Arm A vs Arm B: Overall Survival (OS) | OS was defined as the time from randomization to death from any cause. K-M method was used to estimate median OS. 95% CI for median was computed using the method of Brookmeyer and Crowley. | ITT population included all randomized participants whether or not the assigned study treatment was received. As specified in the protocol, assessment of this outcome measure was limited to Arms A and B, as the objective was to compare pralsetinib with SOC platinum-containing anticancer treatment regimens. | Posted | Median | 95% Confidence Interval | months | From randomization to death (up to approximately 50 months) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. SAE is any significant hazard, contraindication, side effect that is fatal or life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is medically significant or requires intervention to prevent one or other of the outcomes listed above. | Safety-evaluable population included all participants who receive any amount of any study drug. | Posted | Count of Participants | Participants | Up to approximately 50 months |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Change From Baseline in Eastern Cooperative Oncology Group Performance Status (ECOG PS) Score | ECOG is a 6-point scale (0-5) used to assess participants functional status, where, 0= Fully active, able to carry on all pre-disease performance without restriction; 1= restricted in physically strenuous activity but ambulatory & able to carry out work of a light or sedentary nature, e.g., light housework, office work; 2= ambulatory & capable of all self-care but unable to carry out any work activities. Up & about more than 50% of waking hours; 3= capable of only limited self-care, confined to bed or chair more than 50% of waking hours; 4= completely disabled, cannot carry on any self-care, totally confined to bed or chair; 5= dead. | Safety-evaluable population included all participants who receive any amount of any study drug. As specified in the protocol, assessment of this outcome measure was limited to the treatment period, as the objective was to compare pralsetinib with SOC platinum-containing anticancer treatment regimens. | Posted | Count of Participants | Participants | Baseline up to 50 months |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Arm A vs Arm B: Duration of Response (DOR) | DOR was defined as the time from the first occurrence of a documented objective response (OR) to PD or death from any cause, whichever occurred first, as determined by the investigator with the use of RECIST v1.1. CR was defined as disappearance of all target lesions or any pathological lymph nodes must have reduction in short axis to < 10 mm. PR was defined as at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in the absence of CR. PD was defined as at least a 20% increase in the SOD of target lesions, taking as reference the smallest SOD at prior timepoints, including baseline. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. K-M method was used to estimate median DOR. 95% CI for median was computed using the method of Brookmeyer and Crowley. | ITT population included all randomized participants whether or not the assigned study treatment is received. Overall number analyzed is the number of participants with an objective response of CR or PR. As specified in the protocol, assessment of this outcome measure was limited to Arms A and B, as the objective was to compare pralsetinib with SOC platinum-containing anticancer treatment regimens. | Posted | Median | 95% Confidence Interval | months | Up to approximately 50 months |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Arm A vs Arm B: Clinical Benefit Rate (CBR) | CBR was defined as the percentage of participants who experienced the best response of stable disease (SD) with a minimum duration of 6 months, a CR, or a PR, as assessed by investigator with use of RECIST v1.1. SD was defined as neither sufficient shrinkage to qualify for CR or PR nor sufficient increase to qualify for PD. CR was defined as the disappearance of all target lesions or any pathological lymph nodes must have reduction in short axis to < 10 mm. PR was defined as at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in the absence of CR. PD was defined as at least a 20% increase in the SOD of target lesions, taking as reference the smallest SOD at prior timepoints, including baseline. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. 95% CI for rates were constructed using the Clopper-Pearson method. Percentages are rounded off. | ITT population included all randomized participants whether or not the assigned study treatment is received. As specified in the protocol, assessment of this outcome measure was limited to Arms A and B, as the objective was to compare pralsetinib with SOC platinum-containing anticancer treatment regimens. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to approximately 50 months |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Arm A vs Arm B: Disease Control Rate (DCR) | DCR was defined as the percentage of participants who experienced the best response of CR, or PR, or SD, as assessed by investigator according to RECIST v1.1. SD was defined as neither sufficient shrinkage to qualify for CR or PR nor sufficient increase to qualify for PD. CR was defined as the disappearance of all target lesions or any pathological lymph nodes must have reduction in short axis to <10 mm. PR was defined as at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in the absence of CR. PD was defined as at least a 20% increase in the SOD of target lesions, taking as reference the smallest SOD at prior timepoints, including baseline. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. 95% CI for rates were constructed using the Clopper-Pearson method. Percentages are rounded off. | ITT population included all randomized participants whether or not the assigned study treatment is received. As specified in the protocol, assessment of this outcome measure was limited to Arms A and B, as the objective was to compare pralsetinib with SOC platinum-containing anticancer treatment regimens. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to approximately 50 months |
|
Up to approximately 50 months
All-cause mortality: ITT population included all randomized participants, regardless of whether the assigned study treatment was received.
AEs: Safety-evaluable population included all participants who receive any amount of any study drug. 11 randomized participants did not receive any study treatment and were excluded from safety analysis.
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 | Arm A: Treatment Period- SOC | Participants received platinum-containing anticancer treatment regimens (with or without pembrolizumab) at the study center as chosen by the treating investigator based on NSCLC histology. Participants with NSCLC of non-squamous histology received carboplatin + pemetrexed or cisplatin + pemetrexed or pembrolizumab + carboplatin + pemetrexed or pembrolizumab + cisplatin + pemetrexed. Participants with NSCLC of squamous histology received carboplatin or cisplatin + gemcitabine or carboplatin + paclitaxel/nab-paclitaxel + pembrolizumab until PD or for up to a maximum of 2 years. | 27 | 113 | 38 | 104 | 103 | 104 |
| EG001 | Arm B: Treatment Period- Pralsetinib | Participants received pralsetinib, 400 mg, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity, non-compliance, withdrawal of consent, closure of the study by the sponsor, or death, whichever occurred first (cycle length=21 days). | 33 | 110 | 67 | 108 | 106 | 108 |
| EG002 | Arm C: Crossover Period- Pralsetinib | Participants with confirmed PD as confirmed by blinded independent central review (BICR) in Arm A crossed over to receive pralsetinib, 400 mg, orally, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity or death, whichever occurred first (cycle length=21 days). | 8 | 38 | 22 | 38 | 38 | 38 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Febrile bone marrow aplasia | Blood and lymphatic system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pancytopenia | Blood and lymphatic system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Acute myocardial infarction | Cardiac disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Cardiac tamponade | Cardiac disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Cardio-respiratory arrest | Cardiac disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Myocarditis | Cardiac disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Keratitis | Eye disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Scleral disorder | Eye disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Enteritis | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Intestinal perforation | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Intra-abdominal haematoma | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Death | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| General physical health deterioration | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Atypical pneumonia | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Escherichia infection | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Kidney infection | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Pleural infection | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Pneumocystis jirovecii pneumonia | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Pneumonia cytomegaloviral | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Pneumonia legionella | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Pseudomembranous colitis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Pyelonephritis acute | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Q fever | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Spontaneous bacterial peritonitis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA version 27.1 | Systematic Assessment |
| |
| Clavicle fracture | Injury, poisoning and procedural complications | MedDRA version 27.1 | Systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA version 27.1 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA version 27.1 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA version 27.1 | Systematic Assessment |
| |
| Lumbar vertebral fracture | Injury, poisoning and procedural complications | MedDRA version 27.1 | Systematic Assessment |
| |
| Pelvic fracture | Injury, poisoning and procedural complications | MedDRA version 27.1 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Eating disorder symptom | Metabolism and nutrition disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pathological fracture | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Anal cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 27.1 | Systematic Assessment |
| |
| Cancer pain | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 27.1 | Systematic Assessment |
| |
| Colorectal adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 27.1 | Systematic Assessment |
| |
| Endometrial adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 27.1 | Systematic Assessment |
| |
| Metastases to central nervous system | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 27.1 | Systematic Assessment |
| |
| Non-small cell lung cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 27.1 | Systematic Assessment |
| |
| Thyroid adenoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 27.1 | Systematic Assessment |
| |
| Tumour pain | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 27.1 | Systematic Assessment |
| |
| Brain oedema | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Intracranial pressure increased | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Device dislocation | Product Issues | MedDRA version 27.1 | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Adnexa uteri cyst | Reproductive system and breast disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Prostatitis | Reproductive system and breast disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Bronchopleural fistula | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Interstitial lung disease | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pharyngeal inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pulmonary fibrosis | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pulmonary toxicity | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Stevens-Johnson syndrome | Skin and subcutaneous tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Embolism | Vascular disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Thrombophlebitis migrans | Vascular disorders | MedDRA version 27.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Lymphopenia | Blood and lymphatic system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hypothyroidism | Endocrine disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Eyelid oedema | Eye disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Lacrimation increased | Eye disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Face oedema | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Mucosal inflammation | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA version 27.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Blood bicarbonate decreased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA version 27.1 | Systematic Assessment |
| |
| Cell death | Metabolism and nutrition disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Amnesia | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Sciatica | Nervous system disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Erectile dysfunction | Reproductive system and breast disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Prostatitis | Reproductive system and breast disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Lung disorder | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hypertrichosis | Skin and subcutaneous tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA version 27.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA version 27.1 | Systematic Assessment |
|
The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Communications | Hoffmann-La Roche | 800 821-8590 | genentech@druginfo.com |
| Jan 5, 2026 |
| Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| D008175 | Lung Neoplasms |
| D002289 | Carcinoma, Non-Small-Cell Lung |
| D012142 | Respiratory Tract Neoplasms |
| D013899 | Thoracic Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D002283 | Carcinoma, Bronchogenic |
| D001982 | Bronchial Diseases |
| D006258 | Head and Neck Neoplasms |
| D000230 | Adenocarcinoma |
| D002277 | Carcinoma |
| D009370 | Neoplasms by Histologic Type |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009380 | Neoplasms, Nerve Tissue |
| ID | Term |
|---|---|
| D001984 | Bronchial Neoplasms |
| D009375 | Neoplasms, Glandular and Epithelial |
Not provided
Not provided
| ID | Term |
|---|---|
| C000655704 | pralsetinib |
| D016190 | Carboplatin |
| D002945 | Cisplatin |
| D000068437 | Pemetrexed |
| C582435 | pembrolizumab |
| D000093542 | Gemcitabine |
| D017239 | Paclitaxel |
| C520255 | 130-nm albumin-bound paclitaxel |
| ID | Term |
|---|---|
| D056831 | Coordination Complexes |
| D009930 | Organic Chemicals |
| D017606 | Chlorine Compounds |
| D007287 | Inorganic Chemicals |
| D017672 | Nitrogen Compounds |
| D017671 | Platinum Compounds |
| D006147 | Guanine |
| D007042 | Hypoxanthines |
| D011688 | Purinones |
| D011687 | Purines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
| D005971 | Glutamates |
| D024342 | Amino Acids, Acidic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
| D000600 | Amino Acids, Dicarboxylic |
| D003841 | Deoxycytidine |
| D003562 | Cytidine |
| D011741 | Pyrimidine Nucleosides |
| D011743 | Pyrimidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D043823 | Taxoids |
| D043822 | Cyclodecanes |
| D003516 | Cycloparaffins |
| D006840 | Hydrocarbons, Alicyclic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D004224 | Diterpenes |
| D013729 | Terpenes |
Not provided
Not provided
| Informed Consent Withdrawn |
|
| Reason Not Specified |
|
| Progressive Disease |
|
| Study Terminated by Sponsor |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Black or African American |
|
| White |
|
| Other |
|
| Unknown |
|
| Not Reported |
|
| OG001 | Arm B: Treatment Period- Pralsetinib | Participants received pralsetinib, 400 mg, orally, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity, non-compliance, withdrawal of consent, closure of the study by the sponsor, or death, whichever occurred first (cycle length=21 days). |
|
|
|
|
|
|
| OG001 | Arm B: Treatment Period- Pralsetinib | Participants received pralsetinib, 400 mg, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity, non-compliance, withdrawal of consent, closure of the study by the sponsor, or death, whichever occurred first (cycle length=21 days). |
| OG002 | Arm C: Crossover Period- Pralsetinib | Participants with confirmed PD as confirmed by blinded independent central review (BICR) in Arm A crossed over to receive pralsetinib, 400 mg, orally, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity or death, whichever occurred first (cycle length=21 days). |
|
|
| OG001 | Arm B: Treatment Period- Pralsetinib | Participants received pralsetinib, 400 mg, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity, non-compliance, withdrawal of consent, closure of the study by the sponsor, or death, whichever occurred first (cycle length=21 days). |
| OG002 | Arm C: Crossover Period- Pralsetinib | Participants with confirmed PD as confirmed by blinded independent central review (BICR) in Arm A crossed over to receive pralsetinib, 400 mg, orally, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity or death, whichever occurred first (cycle length=21 days). |
|
|
| OG001 | Arm B: Treatment Period- Pralsetinib | Participants received pralsetinib, 400 mg, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity, non-compliance, withdrawal of consent, closure of the study by the sponsor, or death, whichever occurred first (cycle length=21 days). |
|
|
|
| OG001 | Arm B: Treatment Period- Pralsetinib | Participants received pralsetinib, 400 mg, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity, non-compliance, withdrawal of consent, closure of the study by the sponsor, or death, whichever occurred first (cycle length=21 days). |
|
|
| OG001 | Arm B: Treatment Period- Pralsetinib | Participants received pralsetinib, 400 mg, QD on Day 1 of each cycle until PD, PTAP, pralsetinib was no longer available, unacceptable toxicity, non-compliance, withdrawal of consent, closure of the study by the sponsor, or death, whichever occurred first (cycle length=21 days). |
|
|