Evaluation of a New Thermostable Formulation of FLOLAN in Japanese Subjects
An Open Label, Single-arm Study Evaluating a New Thermostable Formulation of FLOLAN™ in Japanese Subjects With Pulmonary Arterial Hypertension (PAH)
Panoramica dello studio
Stato
Stato
Condizioni
Condizioni
Intervento / Trattamento
Intervento / Trattamento
Tipo di studio
Tipo di studio
Iscrizione (Effettivo)
Iscrizione
Fase
Fase
- Fase 4
Contatti e Sedi
Luoghi di studio
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Okayama, Giappone, 701-1192
- GSK Investigational Site
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Criteri di partecipazione
Criteri di ammissibilità
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
Inclusion Criteria:
- Male or female subjects at least 18 to 75 years at the time of screening;
- Subjects must be on FLOLAN therapy for pulmonary arterial hypertension (PAH) as approved in the product label;
- Subjects must receive FLOLAN therapy at 45 nanograms (ng)/kilogram (kg)/minute (min) or higher;
- Subjects must be on stable doses of their existing FLOLAN treatment for a minimum of one month prior to screening; it is acceptable to adjust within 10% of dose during the last one month period;
- Subjects must be on stable doses of any current PAH treatments other than FLOLAN therapy in the last 30 days prior to screening;
Subjects who meet any of the following: A female subject is eligible to participate if she is not pregnant (as confirmed by a negative [serum or urine] human chorionic gonadotrophin [hCG] test), not lactating, and at least one of the following conditions applies:
- Non-reproductive potential defined as: Pre-menopausal females with any of the following: documented tubal ligation, documented hysteroscopic tubal occlusion procedure with follow-up confirmation of bilateral tubal occlusion, hysterectomy, or documented bilateral oophorectomy. Postmenopausal defined as 12 months of spontaneous amenorrhea (in questionable cases a blood sample with simultaneous follicle stimulating hormone [FSH] and estradiol levels consistent with menopause). Females on hormone replacement therapy (HRT) and whose menopausal status is in doubt will be required to use one of the highly effective contraception methods if they wish to continue their HRT during the study. Otherwise, they must discontinue HRT to allow confirmation of post-menopausal status prior to study enrolment.
- Reproductive potential and agrees to follow one of the options listed below in the GSK Modified List of Highly Effective Methods for Avoiding Pregnancy in Females of Reproductive Potential (FRP) from 30 days prior to the first dose of thermostable formulation of FLOLAN therapy until completion of the follow-up visit.
- Subject must agree not to participate in a clinical study involving another investigational drug or device throughout this study;
- Subjects must be competent to understand the information given in the Institutional Review Board (IRB) or Independent Ethics Committee (IEC) approved informed consent form and must sign the form prior to the initiation of any study procedures.
Exclusion Criteria:
- Subjects who are given FLOLAN therapy for a condition or in a manner that is outside the approved indication.
- Subjects with congestive heart failure arising from severe left ventricular dysfunction.
- Subjects, with or without supplemental oxygen, who have a resting arterial oxygen saturation (SaO2) <90% as measured by pulse oximetry at screening.
- Subjects have been hospitalised as an emergency or visited the emergency room for a condition related to PAH or treatment for PAH in the last 3 months.
- The subject's clinical condition is such that they are not expected to remain clinically stable for the duration of the study.
- Female subjects who are pregnant or breastfeeding.
- Subjects who have demonstrated noncompliance with previous medical regimens.
- Subjects who have a history of abusing alcohol or illicit drugs within 1 year.
- Subjects who have participated in a clinical study involving another investigational drug or device within four weeks before screening.
- Any concurrent condition that would affect the safety of the subject or in the opinion of the investigator (or subinvestigator) it is not in the best interest of the patient to participate in the study.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: N / A
- Modello interventistico: Assegnazione di gruppo singolo
- Mascheramento: Nessuno (etichetta aperta)
Numero di armi
Armi e interventi
Gruppo di partecipanti / ArmGruppo di partecipanti / Arm |
Intervento / TrattamentoIntervento / Trattamento |
|---|---|
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Sperimentale: FLOLAN arm
Subjects will receive the existing FLOLAN treatment (i.e., FLOLAN injection prepared with the currently marketed diluent) for a run-in period of a maximum of 4 weeks, the thermostable formulation of FLOLAN injection (i.e., FLOLAN injection prepared with the reformulated diluent) for a 4-week treatment period and there will be a one-week follow-up visit.
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FLOLAN injection (epoprostenol 0.5 or 1.5) prepared with the currently marketed diluent (epoprostenol sodium+ powder of hydrogen [pH] 10.2 - 10.8 diluent) for Injection.
FLOLAN injection (epoprostenol 0.5 or 1.5) prepared with the reformulated diluent (epoprostenol sodium + pH 11.7 - 12.3 diluent) for Injection.
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Number of Participants With Any Adverse Event (AE) or Serious Adverse Event (SAE)
Lasso di tempo: Up to Week 4
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An AE is defined as any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
A SAE is defined as any untoward medical occurrence that, at any dose, results in death, is life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, or is a congenital anomaly/birth defect, important medical events which may require medical or surgical interventions.
Intention-to-Treat (ITT) population: comprised of all participants who have received at least one dose of the thermostable formulation of FLOLAN.
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Up to Week 4
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Number of Participants With Mild, Moderate or Severe AEs
Lasso di tempo: Up to Week 4
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Intensity for an AE and SAE is categorized as mild if an event is easily tolerated by the participant, causing minimal discomfort and not interfering with everyday activities; moderate if an event is sufficiently discomforting to interfere with normal everyday activities; severe if that prevents normal everyday activities.
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Up to Week 4
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Percentage of Basophils, Eosinophils, Lymphocytes, Monocytes, and Total Neutrophils in Blood at Baseline and Week 4
Lasso di tempo: Baseline and Week 4
|
Blood samples were collected for the measurement of percentage of basophils, eosinophils, lymphocytes, monocytes, and total neutrophils in blood at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hour (hr) prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline and Week 4
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Absolute Values of Hemoglobin at Baseline and Week 4
Lasso di tempo: Baseline and Week 4
|
Blood samples were collected for measurement of hemoglobin values at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline and Week 4
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Absolute Values of Hematocrit at Baseline and Week 4
Lasso di tempo: Baseline and Week 4
|
Blood samples were collected for measurement of hematocrit values at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline and Week 4
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Absolute Values of Platelet Count and White Blood Cell Count at Baseline and Week 4
Lasso di tempo: Baseline and Week 4
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Blood samples were collected for measurement of platelets and white blood cells (WBC) at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline and Week 4
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Absolute Values of Red Blood Cell Count at Baseline and Week 4
Lasso di tempo: Baseline and Week 4
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Blood samples were collected for measurement of red blood cells at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline and Week 4
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Absolute Values of Albumin and Total Protein at Baseline and Week 4
Lasso di tempo: Baseline and Week 4
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Blood samples were collected for measurement of albumin and total protein at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline and Week 4
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Absolute Values of Total and Direct Bilirubin, Creatinine, and Uric Acid at Baseline and Week 4
Lasso di tempo: Baseline and Week 4
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Blood samples were collected for measurement of total and direct bilirubin, creatinine (CRT), and uric acid (UA) at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline and Week 4
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Absolute Values of Alanine Aminotransferase, Aspartate Aminotransferase, Alkaline Phosphatase, Gamma Glutamyltransferase, Lactate Dehydrogenase and Creatine Kinase at Baseline and Week 4
Lasso di tempo: Baseline and Week 4
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Blood samples were collected for measurement of Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Alkaline Phosphatase (ALP), Gamma Glutamyltransferase (GGT), Lactate Dehydrogenase (LDH) and Creatine Kinase (CK) at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline and Week 4
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Absolute Values of Urea/Blood Urea Nitrogen, Glucose, Chloride, Sodium, Potassium, Magnesium, Phosphorus (Inorganic), and Calcium at Baseline and Week 4
Lasso di tempo: Baseline and Week 4
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Blood samples were collected for measurement of urea/Blood Urea Nitrogen (Urea/BUN), glucose, chloride, sodium, potassium, magnesium, phosphorus, inorganic, and calcium at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline and Week 4
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Absolute Values of Free Triiodothyronine and Free Thyroxine at Baseline and Week 4
Lasso di tempo: Baseline and Week 4
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Blood samples were collected for measurement of Free Triiodothyronine (FT3) and Free Thyroxine (FT4) at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline and Week 4
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Absolute Values of Thyroid Stimulating Hormone at Baseline and Week 4
Lasso di tempo: Baseline and Week 4
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Blood samples were collected for measurement of Thyroid Stimulating Hormone (TSH) at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline and Week 4
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Number of Participants With the Indicated Urinalysis Findings
Lasso di tempo: Baseline and Week 4
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Urine protein, urine glucose, and occult blood were assessed at Baseline (BL) and Week 4 (W4).
Dipstick test was performed for routine urinalysis.
Abnormal values such as trace, 1+, 2+, 3+ and positive have been reported.
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline').
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Baseline and Week 4
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Number of Participants With the Indicated Electrocardiogram (ECG) Findings
Lasso di tempo: Baseline, 24 hour and Week 4
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A safety 12-lead ECG was performed at Baseline (BL), 24 hr after switching to the new Flolan diluent and Week 4 (W4).
Any abnormal clinically significant (CS) and not clinically significant (NCS) findings were reported.
ECG abnormaility with respect to CS and NCS findings were judged by the investigator.
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Baseline, 24 hour and Week 4
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Change From Baseline in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)
Lasso di tempo: Baseline, 1 hour, 3 hour, 24 hour and Week 4
|
SBP and DBP were measured at Baseline, 1 hr, 3 hr, 24 hr, Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline').
Change from Baseline is defined as the difference between the post-dose visit value and the Baseline value.
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Baseline, 1 hour, 3 hour, 24 hour and Week 4
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Change From Baseline in Heart Rate
Lasso di tempo: Baseline and up to Week 4
|
Heart rate was measured at Baseline, 1 hr, 3 hr, 24 hr, Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline').
Change from Baseline is defined as the difference between the post-dose visit value and the Baseline value.
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Baseline and up to Week 4
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Change From Baseline in Body Weight
Lasso di tempo: Baseline and Week 4
|
Body weight was measured at Baseline (BL) and Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline').
Change from Baseline is defined as the difference between the post-dose visit value and the Baseline value.
Participants with body weight outside and within the clinical concern reference range (<50kg) has been presented.
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Baseline and Week 4
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Absolute Values of Oxygen Saturation
Lasso di tempo: Baseline and up to Week 4
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Oxygen saturation was measured by pulse oximetry at Baseline (BL), 1 hr, 3 hr, 24 hr, Week 4 (W4).
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline').
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Baseline and up to Week 4
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Misure di risultato secondarie
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Number of Events to Adjust Dose of FLOLAN Based on the Change From Baseline to 3 Hours in Mean Pulmonary Artery Pressure (mPAP)
Lasso di tempo: Up to Week 4
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To assess the frequency of dose adjustment requirements based on the changes from baseline in mPAP up to 3 hours after dosing.The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline').
Participants who gave consent to undergo right heart catheterisation (RHC) over 24-hour and at week 4 were assessed.
Change from Baseline is defined as the difference between the post-dose visit value and the Baseline value.
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Up to Week 4
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Number of Participants With the Reason for the Change Dose of the Thermostable Formulation of FLOLAN
Lasso di tempo: Up to Week 4
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All reasons for FLOLAN dose adjustments after the switch were listed.
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Up to Week 4
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Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT Pro BNP)
Lasso di tempo: Baseline and up to Week 4
|
Blood samples were collected at Baseline, 24 hours after the first dose of thermostable formulation of FLOLAN, and Week 4 for measurement of NT pro BNP.
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline').
Change from Baseline is defined as the difference between the post-dose visit value and the Baseline value.
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Baseline and up to Week 4
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Number of Participants in Each World Health Organization (WHO) Functional Class
Lasso di tempo: Baseline and Week 4
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The WHO functional classes of PAH range from Class I (without limitation in physical activity) to Class IV (inability to perform a physical activity without any symptoms).
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Baseline and Week 4
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Number of Participants With Change of WHO Functional Class From Previous Visit
Lasso di tempo: Up to Week 4
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WHO Functional Classification of physical activity limitations: I (no limitation) and IV (unable to carry out any physical activity without symptoms).
The change from baseline in WHO class was classified as Improved (decrease in functional class), No Change (functional class stayed the same), and Deteriorated (functional class increased).
The change from baseline in WHO functional class at Week 4 has been presented in the table.
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline')
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Up to Week 4
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Change From Baseline in Mean Pulmonary Arterial Pressure (mPAP) and Mean Right Atrial Pressure (mRAP)
Lasso di tempo: Baseline and up to Week 4
|
mPAP and mRAP are hemodynamic parameters.
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline').
Change from Baseline is defined as the difference between the post-dose visit value and the Baseline value.
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Baseline and up to Week 4
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Change From Baseline in Pulmonary Vascular Resist (PVR)
Lasso di tempo: Baseline and up to Week 4
|
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline').
Change from Baseline is defined as the difference between the post-dose visit value and the Baseline value.
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Baseline and up to Week 4
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Change From Baseline in Cardiac Output (CO)
Lasso di tempo: Baseline and up to Week 4
|
Cardiac output is the volume of blood pumped by the heart per minute.
The Baseline values are those collected within 0.5 hr prior to the first dose of the new diluent formulation ('Visit 2 - Baseline').
Change from Baseline is defined as the difference between the post-dose visit value and the Baseline value.
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Baseline and up to Week 4
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Collaboratori e investigatori
Sponsor
Sponsor
Pubblicazioni e link utili
Studiare le date dei record
Studia le date principali
Inizio studio
Inizio studio
Completamento primario (Effettivo)
Completamento primario
Completamento dello studio (Effettivo)
Completamento dello studio
Date di iscrizione allo studio
Primo inviato
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Stima)
Primo Inserito
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento pubblicato
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
Altri numeri di identificazione dello studio
- 201614
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