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  1. Article ; Online: Long-term Efficacy and Safety of Elamipretide in Patients with Barth Syndrome: 168-Week Open-label Extension Results of TAZPOWER.

    Thompson, William R / Manuel, Ryan / Abbruscato, Anthony / Carr, Jim / Campbell, John / Hornby, Brittany / Vaz, Frédéric M / Vernon, Hilary J

    Genetics in medicine : official journal of the American College of Medical Genetics

    2024  , Page(s) 101138

    Abstract: Purpose: Evaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome (BTHS) .: Methods: TAZPOWER was a 28-week randomized, double-blind, placebo-controlled ... ...

    Abstract Purpose: Evaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome (BTHS) .
    Methods: TAZPOWER was a 28-week randomized, double-blind, placebo-controlled trial followed by a 168-week OLE. Patients entering the OLE continued elamipretide 40mg subcutaneous daily. OLE primary endpoints were safety and tolerability; secondary endpoints included change from baseline in the 6-minute walk test (6MWT) and BarTH Syndrome Symptom Assessment (BTHS-SA) Total Fatigue. Muscle strength, physician- and patient-assessed outcomes, echocardiographic parameters, and biomarkers, including cardiolipin (CL) and monolysocardiolipin (MLCL), were assessed.
    Results: Ten patients entered the OLE; 8 reached the Week 168 visit. Elamipretide was well tolerated, with injection site reactions being the most common adverse events. Significant improvements from OLE baseline on 6MWT occurred at all OLE timepoints (cumulative 96.1 meters of improvement [Week 168, p=0.003]). Mean BTHS-SA Total Fatigue scores were below baseline (improved) at all OLE timepoints. 3-D left ventricular stroke, end-diastolic, and end-systolic volumes improved, showing significant trends for improvement from baseline to Week 168. MLCL/CL values showed improvement, correlating to important clinical outcomes.
    Conclusion: Elamipretide was associated with sustained long-term tolerability and efficacy, with improvements in functional assessments and cardiac function in BTHS.
    Language English
    Publishing date 2024-04-07
    Publishing country United States
    Document type Journal Article
    ZDB-ID 1455352-1
    ISSN 1530-0366 ; 1098-3600
    ISSN (online) 1530-0366
    ISSN 1098-3600
    DOI 10.1016/j.gim.2024.101138
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    In stock of ZB MED Cologne/Königswinter

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  2. Article ; Online: Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome.

    Hornby, Brittany / Thompson, William Reid / Almuqbil, Mohammed / Manuel, Ryan / Abbruscato, Anthony / Carr, Jim / Vernon, Hilary J

    Orphanet journal of rare diseases

    2022  Volume 17, Issue 1, Page(s) 336

    Abstract: Background: Natural history studies are increasingly recognized as having an important role in drug development for rare diseases. A phase 3, observational, retrospective, and non-interventional study was designed to establish a natural history control ( ...

    Abstract Background: Natural history studies are increasingly recognized as having an important role in drug development for rare diseases. A phase 3, observational, retrospective, and non-interventional study was designed to establish a natural history control (NHC) cohort of patients with Barth syndrome (BTHS) to provide further analysis of the efficacy of elamipretide observed in an open-label extension (OLE) phase of the TAZPOWER trial, a clinical trial that tested the efficacy of 40 mg daily of elamipretide in patients with BTHS.
    Methods: This was a retrospective, non-interventional study. A propensity score model was used to compare elamipretide-treated patients and NHCs. The analysis included 8 patients from the TAZPOWER OLE and 19 untreated NHCs (including 12 with serial echocardiographic assessments).
    Results: For the 6-min walk test (6MWT, primary endpoint), the least squares (LS) mean difference between groups was 79.7 m (P = 0.0004) at week 64 and 91.0 m (P = 0.0005) at week 76 in favor of elamipretide. Significant improvements in muscle strength (secondary endpoint), as assessed by handheld dynamometry (HHD) were also observed with elamipretide, with LS mean differences of 40.8 Newtons at 64 weeks (P = 0.0002) and 56.7 Newtons at 76 weeks (P = 0.0005). Patients continuously treated with elamipretide also experienced statistically significant improvements in other secondary endpoints (i.e., 5 times sit-to-stand [5XSST], multi-domain responder index [MDRI]). The functional improvements were robust to sensitivity analyses. Left ventricular stroke volume increased from baseline in patients with elamipretide but decreased in NHCs.
    Conclusions: Overall, the study established a NHC for use in assessing the efficacy of therapeutic interventions in patients with BTHS and the results suggest that elamipretide may improve natural history of BTHS at least in part by attenuating the natural decline in heart function and provide meaningful improvements in heart function and functional capacity in patients with BTHS compared to NHCs.
    Highlights: A matched Natural History Control (NHC) was used to evaluate elamipretide in BTHS Elamipretide may improve natural history of BTHS by attenuating natural decline in heart function Elamipretide was associated with meaningful clinical improvements in skeletal muscle and cardiovascular parameters that were not observed in NHCs The study established a NHC for use in assessing the efficacy of therapeutic interventions in BTHS.
    MeSH term(s) Barth Syndrome/drug therapy ; Humans ; Oligopeptides/therapeutic use ; Retrospective Studies ; Treatment Outcome ; Walk Test
    Chemical Substances Oligopeptides ; arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide
    Language English
    Publishing date 2022-09-02
    Publishing country England
    Document type Clinical Trial, Phase III ; Journal Article ; Observational Study ; Research Support, Non-U.S. Gov't
    ZDB-ID 2225857-7
    ISSN 1750-1172 ; 1750-1172
    ISSN (online) 1750-1172
    ISSN 1750-1172
    DOI 10.1186/s13023-022-02469-5
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article ; Online: A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism.

    Reid Thompson, W / Hornby, Brittany / Manuel, Ryan / Bradley, Elena / Laux, Janice / Carr, Jim / Vernon, Hilary J

    Genetics in medicine : official journal of the American College of Medical Genetics

    2020  Volume 23, Issue 3, Page(s) 471–478

    Abstract: Purpose: To evaluate effectiveness of elamipretide in Barth syndrome (BTHS), a genetic condition of defects in TAZ, which causes abnormal cardiolipin on the inner mitochondrial membrane.: Methods: We performed a randomized, double-blind, placebo- ... ...

    Abstract Purpose: To evaluate effectiveness of elamipretide in Barth syndrome (BTHS), a genetic condition of defects in TAZ, which causes abnormal cardiolipin on the inner mitochondrial membrane.
    Methods: We performed a randomized, double-blind, placebo-controlled crossover trial followed by an open-label extension in BTHS to test the effect of elamipretide, a mitochondrial tetrapeptide that interacts with cardiolipin. In part 1, 12 subjects were randomized to 40 mg per day of elamipretide or placebo for 12 weeks, followed by a 4-week washout and then 12 weeks on the opposite arm. Ten subjects continued on the open-label extension (part 2) of 40 mg per day of elamipretide, with eight subjects reaching 36 weeks. Primary endpoints were improvement on the 6-minute walk test (6MWT) and improvement on a BTHS Symptom Assessment (BTHS-SA) scale.
    Results: In part 1 neither primary endpoint was met. At 36 weeks in part 2, there were significant improvements in 6MWT (+95.9 m, p = 0.024) and BTHS-SA (-2.1 points, p = 0.031). There were also significant improvements in secondary endpoints including knee extensor strength, patient global impression of symptoms, and some cardiac parameters.
    Conclusion: In this interventional clinical trial in BTHS, daily administration of elamipretide led to improvement in BTHS symptoms.
    MeSH term(s) Barth Syndrome ; Cardiolipins ; Humans ; Mitochondria ; Oligopeptides
    Chemical Substances Cardiolipins ; Oligopeptides ; arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide
    Language English
    Publishing date 2020-10-20
    Publishing country United States
    Document type Clinical Trial, Phase II ; Clinical Trial, Phase III ; Journal Article ; Randomized Controlled Trial ; Research Support, Non-U.S. Gov't
    ZDB-ID 1455352-1
    ISSN 1530-0366 ; 1098-3600
    ISSN (online) 1530-0366
    ISSN 1098-3600
    DOI 10.1038/s41436-020-01006-8
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  4. Article: Taking It to the Streets: Figuring Out and Communicating What's Really Important in Children's Health and Well-being Research.

    Roos, Noralou P / Freemantle, Jane / Farthing, Gerald / Carr, Jim

    Healthcare policy = Politiques de sante

    2014  Volume 6, Issue Spec Issue, Page(s) 86–87

    Title translation Sortir au grand jour: compréhension et communication de ce qui compte vraiment en matière de recherche sur la santé et le bien-être des enfants.
    Language English
    Publishing date 2014-06-26
    Publishing country Canada
    Document type Journal Article
    ZDB-ID 2481657-7
    ISSN 1715-6572
    ISSN 1715-6572
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  5. Article ; Online: Effects of Elamipretide on Left Ventricular Function in Patients With Heart Failure With Reduced Ejection Fraction: The PROGRESS-HF Phase 2 Trial.

    Butler, Javed / Khan, Muhammad Shahzeb / Anker, Stefan D / Fonarow, Gregg C / Kim, Raymond J / Nodari, Savina / O'Connor, Christopher M / Pieske, Burkert / Pieske-Kraigher, Elisabeth / Sabbah, Hani N / Senni, Michele / Voors, Adriaan A / Udelson, James E / Carr, Jim / Gheorghiade, Mihai / Filippatos, Gerasimos

    Journal of cardiac failure

    2020  Volume 26, Issue 5, Page(s) 429–437

    Abstract: Background: Elamipretide, a novel mitochondrial modulating agent, improves myocardial energetics; however, it is unknown whether this mechanistic benefit translates into improved cardiac structure and function in heart failure (HF) with reduced ejection ...

    Abstract Background: Elamipretide, a novel mitochondrial modulating agent, improves myocardial energetics; however, it is unknown whether this mechanistic benefit translates into improved cardiac structure and function in heart failure (HF) with reduced ejection fraction (HFrEF). The objective of this study was to evaluate the effects of multiple subcutaneous doses of elamipretide on left ventricular end systolic volume (LVESV) as assessed by cardiac magnetic resonance imaging.
    Methods: We randomized 71 patients with HFrEF (LVEF ≤ 40%) in a double-blind, placebo-controlled trial in a 1:1:1 ratio to receive placebo, 4 mg or 40 mg elamipretide once daily for 28 consecutive days.
    Results: The mean age (standard deviation) of the study population was 65 ± 10 years, 24% were females, and the mean EF was 31% ± 7%. The change in LVESV from baseline to week 4 was not significantly different between elamipretide 4 mg (89.4 mL to 85 mL; difference, -4.4 mL) or 40 mg (77.9 mL to 76.6 mL; difference, -1.2 mL) compared with placebo (77.7 mL to 74.6 mL; difference, -3.8 mL) (4 mg vs placebo: difference of means, -0.3; 95% CI, -4.6 to 4.0; P  =  0.90; and 40 mg vs placebo: difference of means, 2.3; 95% CI, -1.9 to 6.5; P  =  0.28). Also, no significant differences in change in LVESV and LVEF were observed between placebo and either of the elamipretide groups. Rates of any study drug-related adverse events were similar in the 3 groups.
    Conclusions: Elamipretide was well tolerated but did not improve LVESV at 4 weeks in patients with stable HFrEF compared with placebo.
    MeSH term(s) Aged ; Female ; Heart Failure/drug therapy ; Humans ; Middle Aged ; Oligopeptides ; Stroke Volume ; Ventricular Function, Left
    Chemical Substances Oligopeptides ; arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide
    Language English
    Publishing date 2020-02-14
    Publishing country United States
    Document type Clinical Trial, Phase II ; Journal Article ; Randomized Controlled Trial
    ZDB-ID 1281194-4
    ISSN 1532-8414 ; 1071-9164
    ISSN (online) 1532-8414
    ISSN 1071-9164
    DOI 10.1016/j.cardfail.2020.02.001
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    Zs.A 4340: Show issues Location:
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  6. Article ; Online: Relation of Left Ventricular Mass and Infarct Size in Anterior Wall ST-Segment Elevation Acute Myocardial Infarction (from the EMBRACE STEMI Clinical Trial).

    Daaboul, Yazan / Korjian, Serge / Weaver, W Douglas / Kloner, Robert A / Giugliano, Robert P / Carr, Jim / Neal, Brandon J / Chi, Gerald / Cochet, Madeleine / Goodell, Laura / Michalak, Nathan / Rusowicz-Orazem, Luke / Alkathery, Turky / Allaham, Haytham / Routray, Sujit / Szlosek, Donald / Jain, Purva / Gibson, C Michael

    The American journal of cardiology

    2016  Volume 118, Issue 5, Page(s) 625–631

    Abstract: Biomarker measures of infarct size and myocardial salvage index (MSI) are important surrogate measures of clinical outcomes after a myocardial infarction. However, there is variability in infarct size unaccounted for by conventional adjustment factors. ... ...

    Abstract Biomarker measures of infarct size and myocardial salvage index (MSI) are important surrogate measures of clinical outcomes after a myocardial infarction. However, there is variability in infarct size unaccounted for by conventional adjustment factors. This post hoc analysis of Evaluation of Myocardial Effects of Bendavia for Reducing Reperfusion Injury in Patients With Acute Coronary Events (EMBRACE) ST-Segment Elevation Myocardial Infarction (STEMI) trial evaluates the association between left ventricular (LV) mass and infarct size as assessed by areas under the curve for creatine kinase-MB (CK-MB) and troponin I release over the first 72 hours (CK-MB area under the curve [AUC] and troponin I [TnI] AUC) and the MSI. Patients with first anterior STEMI, occluded left anterior descending artery, and available LV mass measurement in EMBRACE STEMI trial were included (n = 100) (ClinicalTrials.govNCT01572909). MSI, end-diastolic LV mass on day 4 cardiac magnetic resonance, and CK-MB and troponin I concentrations were evaluated by a core laboratory. After saturated multivariate analysis, dominance analysis was performed to estimate the contribution of each independent variable to the predicted variance of each outcome. In multivariate models that included age, gender, body surface area, lesion location, smoking, and ischemia time, LV mass remained independently associated with biomarker measures of infarct size (CK-MB AUC p = 0.02, TnI AUC p = 0.03) and MSI (p = 0.003). Dominance analysis demonstrated that LV mass accounted for 58%, 47%, and 60% of the predicted variances for CK-MB AUC, TnI AUC, and MSI, respectively. In conclusion, LV mass accounts for approximately half of the predicted variance in biomarker measures of infarct size. It should be considered as an adjustment variable in studies evaluating infarct size.
    MeSH term(s) Aged ; Anterior Wall Myocardial Infarction/blood ; Anterior Wall Myocardial Infarction/diagnosis ; Anterior Wall Myocardial Infarction/drug therapy ; Anterior Wall Myocardial Infarction/therapy ; Antioxidants/therapeutic use ; Biomarkers/blood ; Creatine Kinase, MB Form/blood ; Double-Blind Method ; Female ; Heart Ventricles/drug effects ; Heart Ventricles/pathology ; Humans ; Magnetic Resonance Imaging ; Male ; Middle Aged ; Myocardium/enzymology ; Myocardium/pathology ; Oligopeptides/therapeutic use ; Percutaneous Coronary Intervention/methods ; Predictive Value of Tests ; ST Elevation Myocardial Infarction/blood ; ST Elevation Myocardial Infarction/diagnosis ; ST Elevation Myocardial Infarction/drug therapy ; ST Elevation Myocardial Infarction/therapy ; Sensitivity and Specificity ; Time Factors ; Treatment Outcome ; Troponin I/blood
    Chemical Substances Antioxidants ; Biomarkers ; Oligopeptides ; Troponin I ; arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide ; Creatine Kinase, MB Form (EC 2.7.3.2)
    Language English
    Publishing date 2016-06-15
    Publishing country United States
    Document type Clinical Trial, Phase II ; Journal Article ; Randomized Controlled Trial ; Research Support, Non-U.S. Gov't
    ZDB-ID 80014-4
    ISSN 1879-1913 ; 0002-9149
    ISSN (online) 1879-1913
    ISSN 0002-9149
    DOI 10.1016/j.amjcard.2016.06.025
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