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NCT00553358: Neo ALTTO

Neo ALTTO (Neoadjuvant Lapatinib and/or Trastuzumab Treatment Optimisation) Study

Completed Phase 3 Results posted Last updated 21 September 2021
What this trial tests

Phase 3 trial testing Lapatinib in Neoplasms, Breast in 455 participants. Completed in 23 December 2019.

Timeline
5 January 2008
Primary endpoint
27 May 2010
23 December 2019

Quick facts

Lead sponsorNovartis Pharmaceuticals
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Maskingnone
Primary purposetreatment
Enrollment455
Start date5 January 2008
Primary completion27 May 2010
Estimated completion23 December 2019
Sites121 locations across Hong Kong, Italy, Taiwan, South Korea, Russia, Belgium, Sweden, Lithuania

Drugs / interventions tested

Conditions studied

Sponsor

Novartis Pharmaceuticals — full company profile →

Who can join

18 and older, female only, with Neoplasms, Breast. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Pathological Complete Response (pCR) at the Time of Surgery Primary · Weeks 20 to 22

Pathological complete response is defined as no invasive cancer in the breast or only non-invasive in situ cancer in the breast specimen. Surgical breast and axillary node resection specimens were evaluated for pathologic tumor response according to National Surgical Adjuvant Breast and Bowel Project (NSABP) guidelines, which do not take into account the histological nodal status.

GroupValue95% CI
Lapatinib 1500 mg38
Trastuzumab 2 mg/kg44
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg78
Number of Participants With Overall Response at Week 6 Secondary · Week 6

The number of participants with overall response (complete response and/or partial response) was evaluated using World Health Organization (WHO) criteria by clinical examination and by mammography and breast echography with bi-dimensional measurements at Week 6. As per WHO criteria: complete response is defined as the disappearance of all lesions; partial response is defined as a greater than 50% decrease in the sum of products of the greatest length and width of the largest lesion; progressive disease is defined as a greater than 25% increase in the sum of products of all measurable lesions.

Overall Response
GroupValue95% CI
Lapatinib 1500 mg81
Trastuzumab 2 mg/kg45
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg102
No Change
GroupValue95% CI
Lapatinib 1500 mg57
Trastuzumab 2 mg/kg81
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg33
Progressive Disease
GroupValue95% CI
Lapatinib 1500 mg5
Trastuzumab 2 mg/kg11
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg2
Not Evaluated
GroupValue95% CI
Lapatinib 1500 mg7
Trastuzumab 2 mg/kg9
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg12
Missing Data
GroupValue95% CI
Lapatinib 1500 mg4
Trastuzumab 2 mg/kg3
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg3
Overall Response at the Time of Surgery Secondary · Time of surgery (Weeks 20 to 22)

The number of participants with overall response (complete response and/or partial response) was evaluated using WHO criteria by clinical examination and mammography and breast echography with bi-dimensional measurements at the time of surgery (Weeks 20 to 22). As per WHO criteria: complete response is defined as the disappearance of all lesions; partial response is defined as a greater than 50% decrease in the sum of products of the greatest length and width of the largest lesion; progressive disease is defined as a greater than 25% increase in the sum of products of all measurable lesions.

Overall Response
GroupValue95% CI
Lapatinib 1500 mg114
Trastuzumab 2 mg/kg105
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg122
No Change
GroupValue95% CI
Lapatinib 1500 mg8
Trastuzumab 2 mg/kg16
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg7
Progressive Disease
GroupValue95% CI
Lapatinib 1500 mg0
Trastuzumab 2 mg/kg2
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg1
Not Evaluated
GroupValue95% CI
Lapatinib 1500 mg19
Trastuzumab 2 mg/kg20
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg14
Missing Data
GroupValue95% CI
Lapatinib 1500 mg13
Trastuzumab 2 mg/kg6
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg8
Number of Participants With Negative Lymph Nodes at the Time of Surgery Secondary · Time of surgery (Weeks 20 to 22)

Participants were assessed for node-negative lymph nodes at the time of surgery. As per the pathological TNM (Tumor, Node, Metastases) classification (pTNM) of malignant tumors: pN, absence or presence and extent of regional lymph node metastasis. Node-negative (pN0) participants had no regional lymph node metastasis. Although not assessed in this measure, pT is the extent of primary tumor, and pM is the absence or presence of distant metastasis.

GroupValue95% CI
Lapatinib 1500 mg72
Trastuzumab 2 mg/kg82
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg100
Number of Participants With Actual Indicated Surgery Secondary · At surgery (Weeks 20 to 22)

Participants were assessed for the type of surgery they underwent for breast cancer. Non-conservative surgery is defined as a radical or modified radical mastectomy. Conservative surgery is comprised of a lumpectomy, a quadrantectomy/segmentectomy, or a partial mastectomy. Participants who were not assessed as being candidates for non-conservative or conservative surgery were classified as non-operable.

Conservative
GroupValue95% CI
Lapatinib 1500 mg66
Trastuzumab 2 mg/kg58
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg63
Non-conservative
GroupValue95% CI
Lapatinib 1500 mg77
Trastuzumab 2 mg/kg85
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg80
Non-operable
GroupValue95% CI
Lapatinib 1500 mg11
Trastuzumab 2 mg/kg6
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg9
Mean Change From Baseline in Tumor Size at Week 6 and at Surgery Secondary · Week 6 and surgery (Weeks 20 to 22)

Mean change from baseline in tumor in tumor size. Change from baseline in tumor size was defined as tumor size at Week 6/ surgery (Weeks 20 to 22) minus tumor size at baseline. The difference in treatment arms was estimated for Lapatinib 1500 mg versus Trastuzumab 2 mg/kg and for Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg versus Trastuzumab 2 mg/kg.

Week 6
GroupValue95% CI
Lapatinib 1500 mg-20.45± 18.43
Trastuzumab 2 mg/kg-13.42± 16.44
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg-25.77± 19.91
Surgery (Weeks 20 to 22)
GroupValue95% CI
Lapatinib 1500 mg-41.01± 23.81
Trastuzumab 2 mg/kg-35.47± 22.95
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg-43.59± 26.88
Number of Participants Starting Paclitaxel Before Completing 6 Weeks of Treatment With Either Lapatinib or Trastuzumab Secondary · Week 6

Participants with progressive disease at 4 week assessment that were permitted to commence treatment with paclitaxel.

GroupValue95% CI
Lapatinib 1500 mg8
Trastuzumab 2 mg/kg12
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg6
Event-free Survival (EFS) - Median Clinical Follow-up Secondary · From randomization up to approximately year 10

Event free survival (EFS) is defined as the time from randomization to first EFS event. For subjects who had breast cancer surgery, EFS events were post-surgery breast cancer relapse, second primary malignancy or death without recurrence. For subjects who did not have breast cancer surgery, EFS events were death during clinical follow-up or non-completion of any neoadjuvant investigational product due to disease progression.

GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg9.699.55 – 9.73
Lapatinib 1500 mg9.608.21 – 9.69
Trastuzumab 2 mg/kg9.669.50 – 9.72
Event-free Survival (EFS) - Events and Censoring Secondary · From randomization up to approximately year 10

Event free survival (EFS) is defined as the time from randomization to first EFS event. For subjects who had breast cancer surgery, EFS events were post-surgery breast cancer relapse, second primary malignancy or death without recurrence. For subjects who did not have breast cancer surgery, EFS events were death during clinical follow-up or non-completion of any neoadjuvant investigational product due to disease progression.

Number of subjects with EFS events
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg43
Lapatinib 1500 mg47
Trastuzumab 2 mg/kg47
Number of subjects censored - total
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg109
Lapatinib 1500 mg107
Trastuzumab 2 mg/kg102
Number of subjects censored - Clinical follow-up ongoing
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg0
Lapatinib 1500 mg0
Trastuzumab 2 mg/kg0
Number of subjects censored - Clinical follow-up ended - total
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg103
Lapatinib 1500 mg105
Trastuzumab 2 mg/kg99
Number of subjects censored -Clinical follow-up ended - Completed study follow-up
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg61
Lapatinib 1500 mg49
Trastuzumab 2 mg/kg58
Number of subjects censored - Clinical follow-up ended - Lost to follow-up
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg17
Lapatinib 1500 mg20
Trastuzumab 2 mg/kg10
Number of subjects censored - Clinical follow-up ended - Withdrew (but consent for survival f/u)
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg3
Lapatinib 1500 mg6
Trastuzumab 2 mg/kg4
Number of subjects censored - Clinical follow-up ended - Withdrew
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg22
Lapatinib 1500 mg30
Trastuzumab 2 mg/kg27
Overall Survival (OS) - Median Survival Follow-up Secondary · From randomization up to approximately year 10

Overall survival is defined as the period from randomization until death (from any cause). OS was assessed annually for up to 10 years after the randomization of the last participant into the study.

GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg9.709.60 – 9.76
Lapatinib 1500 mg9.628.86 – 9.67
Trastuzumab 2 mg/kg9.649.35 – 9.71
Overall Survival (OS) - Deaths and Censoring Secondary · From randomization up to approximately year 10

Overall survival is defined as the period from randomization until death (from any cause). OS was assessed annually for up to 10 years after the randomization of the last participant into the study.

Number of deaths due to any cause
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg26
Lapatinib 1500 mg31
Trastuzumab 2 mg/kg32
Number of subjects censored - total
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg126
Lapatinib 1500 mg123
Trastuzumab 2 mg/kg117
Number of subjects censored - Survival follow-up ongoing
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg0
Lapatinib 1500 mg0
Trastuzumab 2 mg/kg0
Number of subjects censored - Survival follow-up ended - total
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg120
Lapatinib 1500 mg121
Trastuzumab 2 mg/kg114
Number of subjects censored -Survival follow-up ended - Completed study follow-up
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg74
Lapatinib 1500 mg59
Trastuzumab 2 mg/kg62
Number of subjects censored - Survival follow-up ended - Lost to follow-up
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg22
Lapatinib 1500 mg27
Trastuzumab 2 mg/kg18
Number of subjects censored - Survival follow-up ended - Withdrew
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg24
Lapatinib 1500 mg35
Trastuzumab 2 mg/kg34
Number of subjects censored - Other
GroupValue95% CI
Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg6
Lapatinib 1500 mg2
Trastuzumab 2 mg/kg3
Assess Associations Between Locoregional Pathological Complete Response (pCR) and Event-free Survival (EFS) - Median Clinical Follow-up (EFS Landmark Population) Secondary · up to year 10

The landmark date is 30 weeks after a subject's randomization. Subjects with missing pCR status were not included in the landmark analysis. Clinical follow-up is the period during which the patient is monitored such that all recurrence or second primary malignancy (SPM) or contralateral breast cancer (CBC) events would be reported. Patients are considered in clinical follow-up from randomisation until one of the following occurs: lost to follow-up, withdrawal of consent, end of follow-up due to completion of year 10 visit, termination of study follow-up, or death.

Median clinical follow-up - all subjects in the EFS landmark analysis
GroupValue95% CI
Pathological Complete Response (pCR)9.058.86 – 9.14
No Pathological Complete Response (pCR)9.119.05 – 9.14
Overall9.099.03 – 9.13
Median clinical follow-up- EFS landmark analysis - lapatinib + trastuzumab arm (n=67,71,138)
GroupValue95% CI
Pathological Complete Response (pCR)9.138.97 – 9.23
No Pathological Complete Response (pCR)9.107.24 – 9.15
Overall9.128.97 – 9.15
Median clinical follow-up - subjects in the EFS landmark analysis - lapatinib arm (n=30,104,134)
GroupValue95% CI
Pathological Complete Response (pCR)8.086.08 – 9.12
No Pathological Complete Response (pCR)9.098.52 – 9.19
Overall9.058.23 – 9.12
Median clinical follow-up - subjects in the EFS landmark analysis - trastuzumab arm (n=39,99,138)
GroupValue95% CI
Pathological Complete Response (pCR)8.988.38 – 9.21
No Pathological Complete Response (pCR)9.129.03 – 9.25
Overall9.118.96 – 9.17

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 31 weeks.. Reporting threshold: 2%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Lapatinib 1000/750 mg + Trastuzumab 2 mg/kg
Serious: 61/149 (41%)
Deaths: 1/149
Lapatinib 1500 mg
Serious: 58/151 (38%)
Deaths: 2/151
Trastuzumab 2 mg/kg
Serious: 36/148 (24%)
Deaths: 0/148

Serious adverse events (79 terms)

ReactionSystemLapatinib 1000/750 mg + Tr…Lapatinib 1500 mgTrastuzumab 2 mg/kg
HYPERTRANSAMINASAEMIAHepatobiliary disorders
NEUTROPENIABlood and lymphatic system disorders
DIARRHOEAGastrointestinal disorders
FEBRILE NEUTROPENIABlood and lymphatic system disorders
PYREXIAGeneral disorders
HYPERBILIRUBINAEMIAHepatobiliary disorders
CARDIAC FAILURE CONGESTIVECardiac disorders
VOMITINGGastrointestinal disorders
URINARY TRACT INFECTIONInfections and infestations
LEUKOPENIABlood and lymphatic system disorders
NAUSEAGastrointestinal disorders
CELLULITISInfections and infestations
MASTITISInfections and infestations
PNEUMONIAInfections and infestations
DEHYDRATIONMetabolism and nutrition disorders
ACUTE KIDNEY INJURYRenal and urinary disorders
INTERSTITIAL LUNG DISEASERespiratory, thoracic and mediastinal disorders
AGRANULOCYTOSISBlood and lymphatic system disorders
ANAEMIABlood and lymphatic system disorders
PANCYTOPENIABlood and lymphatic system disorders
CARDIO-RESPIRATORY ARRESTCardiac disorders
MYOCARDIAL INFARCTIONCardiac disorders
DUODENITISGastrointestinal disorders
ENTERITISGastrointestinal disorders
GASTRITIS EROSIVEGastrointestinal disorders
Other adverse events (178 terms — click to expand)

ReactionSystemLapatinib 1000/750 mg + Tr…Lapatinib 1500 mgTrastuzumab 2 mg/kg
DIARRHOEAGastrointestinal disorders
ALOPECIASkin and subcutaneous tissue disorders
NAUSEAGastrointestinal disorders
RASHSkin and subcutaneous tissue disorders
VOMITINGGastrointestinal disorders
HYPERTRANSAMINASAEMIAHepatobiliary disorders
FATIGUEGeneral disorders
NEUTROPENIABlood and lymphatic system disorders
ASTHENIAGeneral disorders
DECREASED APPETITEMetabolism and nutrition disorders
EPISTAXISRespiratory, thoracic and mediastinal disorders
ANAEMIABlood and lymphatic system disorders
MUCOSAL INFLAMMATIONGeneral disorders
NAIL DISORDERSkin and subcutaneous tissue disorders
PYREXIAGeneral disorders
MYALGIAMusculoskeletal and connective tissue disorders
INSOMNIAPsychiatric disorders
ARTHRALGIAMusculoskeletal and connective tissue disorders
HEADACHENervous system disorders
DRY SKINSkin and subcutaneous tissue disorders
PRURITUSSkin and subcutaneous tissue disorders
ABDOMINAL PAINGastrointestinal disorders
ABDOMINAL PAIN UPPERGastrointestinal disorders
STOMATITISGastrointestinal disorders
DYSPEPSIAGastrointestinal disorders
HYPERBILIRUBINAEMIAHepatobiliary disorders
COUGHRespiratory, thoracic and mediastinal disorders
PARAESTHESIANervous system disorders
HYPERPHOSPHATASAEMIAMetabolism and nutrition disorders
BACK PAINMusculoskeletal and connective tissue disorders
ACNESkin and subcutaneous tissue disorders
HOT FLUSHVascular disorders
LEUKOPENIABlood and lymphatic system disorders
NEUROPATHY PERIPHERALNervous system disorders
PAIN IN EXTREMITYMusculoskeletal and connective tissue disorders
OROPHARYNGEAL PAINRespiratory, thoracic and mediastinal disorders
PERIPHERAL SENSORY NEUROPATHYNervous system disorders
CONSTIPATIONGastrointestinal disorders
RADIATION SKIN INJURYInjury, poisoning and procedural complications
BONE PAINMusculoskeletal and connective tissue disorders

Most-reported serious reactions: HYPERTRANSAMINASAEMIA, NEUTROPENIA, DIARRHOEA, FEBRILE NEUTROPENIA, PYREXIA, HYPERBILIRUBINAEMIA, CARDIAC FAILURE CONGESTIVE, VOMITING.

Data from ClinicalTrials.gov NCT00553358 adverse events section.

Sponsor's own description

This is a randomised, open label multicenter Phase III study comparing the efficacy of neoadjuvant lapatinib plus paclitaxel, versus trastuzumab plus paclitaxel, versus concomitant lapatinib and trastuzumab plus paclitaxel given as neoadjuvant treatment in HER2/ErbB2 over-expressing and/or amplified primary breast cancer. Patients will be randomised to receive either: lapatinib 1500 mg daily, trastuzumab 4 mg/kg intravenous (IV) load followed by 2 mg/kg IV weekly, or lapatinib 1000 mg daily with trastuzumab 4 mg/kg IV load followed by 2 mg/kg IV weekly for a total of 6 weeks. After this biological window, patients on monotherapy arms will continue on the same targeted therapy plus weekly paclitaxel 80 mg/m\^2 for a further 12 weeks, up to definitive surgery. In the combination arm, patients will receive lapatinib 750 mg daily in combination with trastuzumab 2 mg/kg IV plus weekly paclitaxel 80mg/m\^2 IV for a further 12 weeks, up to definitive surgery. After surgery, patients will receive three courses of adjuvant chemotherapy with 5-Fluorouracil Epirubicin Cyclophosphamide (FEC) followed by the same targeted therapy as in the biological window of the neoadjuvant setting for a further 34 weeks (in the combination arm, lapatinib dose will be 1000 mg daily in combination with trastuzumab). The planned total duration of the anti-HER2 therapy one year. Primary objective is to evaluate and compare the rate of pathological complete response (pCR) at the time of surgery in patients with HER2/ErbB2 overexpressing or amplified operable breast cancer randomised to lapatinib followed by lapatinib plus paclitaxel versus trastuzumab followed by trastuzumab plus paclitaxel versus lapatinib in combination with trastuzumab followed by lapatinib, trastuzumab plus paclitaxel.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Lapatinib with trastuzumab for HER2-positive early breast cancer (NeoALTTO): a randomised, open-label, multicentre, phase 3 trial.
    Baselga J, Bradbury I, Eidtmann H, Di Cosimo S, et al · · 2012 · cited 982× · PMID 22257673 · DOI 10.1016/s0140-6736(11)61847-3
  2. Tumor-Infiltrating Lymphocytes and Associations With Pathological Complete Response and Event-Free Survival in HER2-Positive Early-Stage Breast Cancer Treated With Lapatinib and Trastuzumab: A Secondary Analysis of the NeoALTTO Trial.
    Salgado R, Denkert C, Campbell C, Savas P, et al · · 2015 · cited 479× · PMID 26181252 · DOI 10.1001/jamaoncol.2015.0830
  3. Lapatinib with trastuzumab for HER2-positive early breast cancer (NeoALTTO): survival outcomes of a randomised, open-label, multicentre, phase 3 trial and their association with pathological complete response.
    de Azambuja E, Holmes AP, Piccart-Gebhart M, Holmes E, et al · · 2014 · cited 336× · PMID 25130998 · DOI 10.1016/s1470-2045(14)70320-1
  4. PIK3CA mutations are associated with decreased benefit to neoadjuvant human epidermal growth factor receptor 2-targeted therapies in breast cancer.
    Majewski IJ, Nuciforo P, Mittempergher L, Bosma AJ, et al · · 2015 · cited 183× · PMID 25559818 · DOI 10.1200/jco.2014.55.2158
  5. RNA Sequencing to Predict Response to Neoadjuvant Anti-HER2 Therapy: A Secondary Analysis of the NeoALTTO Randomized Clinical Trial.
    Fumagalli D, Venet D, Ignatiadis M, Azim HA, et al · · 2017 · cited 133× · PMID 27684533 · DOI 10.1001/jamaoncol.2016.3824
  6. Mechanism, safety and efficacy of three tyrosine kinase inhibitors lapatinib, neratinib and pyrotinib in HER2-positive breast cancer.
    Xuhong JC, Qi XW, Zhang Y, Jiang J. · · 2019 · cited 118× · PMID 31720077
  7. Interaction of host immunity with HER2-targeted treatment and tumor heterogeneity in HER2-positive breast cancer.
    Griguolo G, Pascual T, Dieci MV, Guarneri V, et al · · 2019 · cited 91× · PMID 30922362 · DOI 10.1186/s40425-019-0548-6
  8. Intrinsic subtypes, PIK3CA mutation, and the degree of benefit from adjuvant trastuzumab in the NSABP B-31 trial.
    Pogue-Geile KL, Song N, Jeong JH, Gavin PG, et al · · 2015 · cited 85× · PMID 25559813 · DOI 10.1200/jco.2014.56.2439

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00553358.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing