scholarly journals Long-term effectiveness of carglumic acid in patients with propionic acidemia (PA) and methylmalonic acidemia (MMA): a randomized clinical trial

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Majid Alfadhel ◽  
Marwan Nashabat ◽  
Mohammed Saleh ◽  
Mohammed Elamin ◽  
Ahmed Alfares ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Standard Treatment ◽  
Propionic Acidemia ◽  
Methylmalonic Acidemia ◽  
First Episode ◽  
Controlled Clinical Trial ◽  
Open Label ◽  
Significant Difference ◽  
Secondary Outcomes

Abstract Background Propionic acidemia (PA) and methylmalonic acidemia (MMA) are rare, autosomal recessive inborn errors of metabolism that require life-long medical treatment. The trial aimed to evaluate the effectiveness of the administration of carglumic acid with the standard treatment compared to the standard treatment alone in the management of these organic acidemias. Methods The study was a prospective, multicenter, randomized, parallel-group, open-label, controlled clinical trial. Patients aged ≤ 15 years with confirmed PA and MMA were included in the study. Patients were followed up for two years. The primary outcome was the number of emergency room (ER) admissions because of hyperammonemia. Secondary outcomes included plasma ammonia levels over time, time to the first episode of hyperammonemia, biomarkers, and differences in the duration of hospital stay. Results Thirty-eight patients were included in the study. On the primary efficacy endpoint, a mean of 6.31 ER admissions was observed for the carglumic acid arm, compared with 12.76 for standard treatment, with a significant difference between the groups (p = 0.0095). Of the secondary outcomes, the only significant differences were in glycine and free carnitine levels. Conclusion Using carglumic acid in addition to standard treatment over the long term significantly reduces the number of ER admissions because of hyperammonemia in patients with PA and MMA.

Abstract 16567: Long-Term Vascular Function in CTO Recanalization. A Randomized Clinical Trial of Ticagrelor vs. Clopidogrel

Circulation ◽  
2020 ◽  
Vol 142 (Suppl_3) ◽  
Author(s):  
Juan Jose Rodriguez Arias ◽  
Josep Gomez-Lara ◽  
Juan Caballero-Borrego ◽  
Luis Ortega-Paz ◽  
Luis Teruel ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Vascular Function ◽  
Drug Loading ◽  
Controlled Clinical Trial ◽  
Loading Dose ◽  
Clinical Value ◽  
Open Label ◽  
Total Occlusion

Background: Coronary vascular function of a chronic coronary total occlusion (CTO) immediately after recanalization is known to be poor and to be partially improved by pre-treatment with loading dose of ticagrelor vs. clopidogrel. It is unknown if this vascular dysfunction is maintained at long-term follow-up and may be improved by 1-year dual antiplatelet therapy (DAPT) with ticagrelor vs. clopidogrel. Methods: The TIGER is a prospective, open-label, two parallel-group controlled clinical trial, which 1:1 randomized 50 CTO patients to pre-PCI loading dose and subsequent 1-year DAPT with ticagrelor vs. clopidogrel. Coronary blood flow (CBF) under stepwise adenosine infusion was assessed after drug loading dose and at follow-up and compared between the two drug groups, adjusting for time of follow-up. Results: Out of 50 patients with index CBF evaluation, 38 (76%) patients underwent angiographic follow-up (23 and 15 at 1 and 3-year, respectively). A higher CBF area under the curve (AUC), already observed after loading dose in ticagrelor vs. clopidogrel group (p=0.027), was maintained at follow-up (AUC 34815.22±24206.06 vs. AUC 22712.47±13768.95; p=0.071). Specifically, whereas high ticagrelor loading dose-related CBF was sustained at follow-up (p=0.933), clopidogrel loading dose-related CBF increased at follow-up (p=0.039). Conclusion: The TIGER trial showed that DAPT with ticagrelor may maintain a higher CBF in a recanalized CTO as compared to clopidogrel, whose treated patients exhibit a lower CBF immediately after PCI with slight increase at follow-up. The clinical value of such sustained higher coronary flow should be evaluated in a larger group of patients.

Randomized use of anti-GD2 antibody dinutuximab beta (DB) long-term infusion with and without subcutaneous interleukin-2 (scIL-2) in high-risk neuroblastoma patients with relapsed and refractory disease: Results from the SIOPEN LTI-trial.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 10014-10014
Author(s):  
Holger N. Lode ◽  
Dominique Valteau-Couanet ◽  
Juliet Gray ◽  
Roberto Luksch ◽  
Aleksandra Wieczorek ◽  
...  
Keyword(s):  
Clinical Trial ◽  
High Risk ◽  
Interleukin 2 ◽  
Hematological Toxicity ◽  
Antibody Concentration ◽  
Mycn Amplification ◽  
Open Label ◽  
Significant Difference ◽  
Enzyme Elevation

10014 Background: We determined the role of scIL-2 combined with long term infusion (LTI) of DB in patients (pts) with high-risk relapsed/refractory neuroblastoma. Methods: 160 pts were enrolled into an open label SIOPEN Phase II clinical trial (EudraCT 2009-018077-31). Pts were randomly assigned to receive up to 5 cycles of 100 mg/m2 DB-LTI (d8-17) and 160 mg/m2 oral isotretinoin (d19-32) (81 pts) with and without 6x106IU/m2 scIL-2 (d1-5; 8-12) (79 pts). Endpoints were toxicity, response rates and 2yrs-event free and -overall survival. Results: Between 07/2014 and 07/2017, 160 pts from 11 countries were randomised. Median follow-up is 2.6 years. Pts were well balanced between arms according to stage, age, MYCN amplification, patients with relapse and remission status. The 2yrs-EFS and -OS for DB (81 pts) vs. DB combined with scIL-2 (79 pts) was 59%±6% vs 65%±6% (p = 0.721) and 79%±5% vs 84%±4% (p = 0.904). In 97 pts with evaluable disease, a response rate of 49% (9% CR, 40% PR) vs 52% (26% CR, 26% PR) after treatment with DB vs DB and scIL-2 was observed. Grade 3&4 fever (16% vs 46%, P = 0.000), allergic reaction (1% vs 14%, P = 0.004), hematological toxicity (46% vs 66%, P = 0.013) and neurotoxicity (0% vs 8%, p = 0.003) were significantly worse in the combination arm, but no difference was seen for capillary leak, gastrointestinal, liver enzyme elevation and pain. Paraplegia possibly related to the treatment was observed in 2 pts in the combination arm, none in the arm without scIL-2, and one resolved to baseline. A subgroup of 34 pts who had a relapse and measurable disease at treatment start, showed a 2yrs-EFS and -OS in DB (17 pts) vs DB combined with scIL-2 (17 pts) of 35%±12% vs 69%±12% (p = 0.116) and 59%±12% vs 81%±10% (p = 0.167). However, this trend was statistically not significant. Pharmacokinetic and HACA response between both arms was not different with overlapping antibody concentration-time curves and a HACA response of 15/81 (19%) (DB) vs 16/79 (20%) (DB and scIL-2). Conclusions: No significant difference in efficacy of DB combined with scIL-2 and increased toxicity in this arm suggests that this schedule of scIL-2 is of no additional benefit. Clinical trial information: 2009-018077-31.

scholarly journals Efficacy of doxycycline as a combination therapy in the treatment of rheumatoid arthritis: a randomized controlled clinical trial

2021 ◽  
Vol 33 (1) ◽  
Author(s):  
Eman M. Ibrahem ◽  
Salwa S. El-gendi ◽  
Amal A. Mahmoud ◽  
Sherif M. Abdel-Aal ◽  
Hanan Sharaf El-Deen Mohammed
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Trial ◽  
Combination Therapy ◽  
Cost Effective ◽  
Controlled Clinical Trial ◽  
Open Label ◽  
American College Of Rheumatology ◽  
Effective Combination ◽  
Number Of Patients ◽  
Significant Difference

Abstract Background This single-center randomized open-label clinical trial evaluates the effectiveness of doxycycline as a combination therapy for active rheumatoid arthritis (RA) with methotrexate (MTX). Materials and methods One hundred and sixty RA patients were recruited who fulfilled the 2010 American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification criteria. Subjects were randomly allocated in a 1:1 ratio into one of two treatment arms; one group was maintained on MTX alone and the other group on MTX together with doxycycline orally 200 mg daily. Follow-up clinical response, erythrocyte sedimentation rate (ESR), levels of C-reactive protein (CRP), and disease activity score 28 (DAS28-CRP) after 3 months were done. Results There was a significant difference regarding DAS28-CRP between the two groups (median (IQR) 4.26 (3.6–5) for those treated with MTX alone compared with 2.8 (2.37–3.5) for those treated with MTX together with doxycycline) (p = 0.005). A higher number of patients treated with doxycycline in combination with MTX achieved remission (40.5%) compared to patients who received MTX alone (13.5%). The levels of ESR and CRP were lower in patients treated with MTX and doxycycline and this was statistically significant (p = 0.005, p = 0.003 respectively). Conclusion Doxycycline as a cost-effective combination therapy with MTX can achieve higher rates of remission than MTX alone in RA patients without causing increase in the adverse events profile. Trial registration Clinical Trials.gov, NCT03194204. Registered on 21 June 2017

scholarly journals Atorvastatin and standard treatment of Helicobacter pylori: Single Blinded Randomized Controlled Clinical Trial

2020 ◽  
Author(s):  
Mohammad Reza Mohammad Hoseini Azar ◽  
Parham Portaghali ◽  
Ali Jafari ◽  
Amin Sedokani
Keyword(s):  
Clinical Trial ◽  
Helicobacter Pylori ◽  
Standard Treatment ◽  
Intervention Group ◽  
Control Group ◽  
Controlled Clinical Trial ◽  
P Value ◽  
Bismuth Subcitrate ◽  
Significant Difference ◽  
H Pylori

AbstractBackgroundConsidering the increase in drug resistance over time to Helicobacter pylori treatment relying on the anti-inflammatory and antibacterial effects of atorvastatin to increase the success rate of H. pylori eradication, we examined the effect of adding atorvastatin to standard treatment of H. pylori eradication.Materials and MethodsA total of 186 symptomatic patients who had been diagnosed with Helicobacter pylori infection and tested for H. pylori eradication were examined by a pathological response or positive urea breath test. Patients who received atorvastatin in addition to standard treatment were also identified based on a table of random numbers. Standard treatment included a 240mg bismuth subcitrate tablet, a 40mg pantoprazole tablet, a 500mg metronidazole tablet, and 2 capsules of 500mg amoxicillin, all taken BID for 14 days. After 4 weeks of treatment, all patients underwent stool testing for H. pylori fecal antigen. If the test was positive, the request was considered a failure of treatment, and if the test was negative, it was considered a successful eradication of H. pylori. The clinical trial registration code for this study is IRCT20190823044589N1.ResultsThe eradication rate of H. pylori was 80% in the control group and 80.9% in the intervention group, which did not show a statistically significant difference between the two groups (P-value = 0.971).ConclusionAdding atorvastatin to 4-drug regimen of PPI, bismuth subcitrate, amoxicillin, and metronidazole as the first line of treatment for H. pylori eradication is ineffective.

A pilot study of the ticagrelor role in ischemic stroke secondary prevention

2021 ◽  
pp. 1-6
Author(s):  
Mohamed G. Zeinhom ◽  
Hany M. Aref ◽  
Hala El-khawas ◽  
Tamer M. Roushdy ◽  
Hossam M. Shokri ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Ischemic Stroke ◽  
Acute Ischemic Stroke ◽  
Controlled Clinical Trial ◽  
Loading Dose ◽  
University Hospitals ◽  
Stroke Patients ◽  
Open Label ◽  
Significant Difference

<b><i>Introduction:</i></b> Ticagrelor is one of the most recent antiplatelet drugs used to treat ischemic heart disease. Its efficacy may equal or exceed aspirin in improving clinical outcomes in patients with acute ischemic stroke who are ineligible for rt-PA. <b><i>Aim of the Work:</i></b> We aimed at evaluating the safety (as a primary endpoint) and efficacy (as a secondary endpoint) of a 180 mg loading dose of ticagrelor given within 9 h from the onset of first-ever ischemic stroke. <b><i>Methods:</i></b> We conducted an open-label, randomized prospective controlled clinical trial between May 2019 and September 2020 on patients who presented with their first-ever ischemic stroke and were recruited from the emergency department, of Kafr el-sheik University Hospitals, Egypt. Eligible patients randomly received aspirin or ticagrelor loading and maintenance doses. Treatment began within 9 h of stroke onset. <b><i>Results:</i></b> Aspirin was given to 84 patients; ticagrelor was given to 85. There was no significant difference between the 2 groups regarding the hemorrhagic and nonhemorrhagic complications. Patients who received ticagrelor had a better outcome regarding NIHSS improvement at 2 days and 1 week or discharge and a favorable mRS score after 1 week or discharge and at 90-day follow-up. <b><i>Conclusion:</i></b> Ticagrelor was noninferior to aspirin regarding safety profile. Compared with aspirin, ticagrelor had a better clinical outcome based on NIHSS and mRS in first-ever acute ischemic stroke patients who received it within 9 h from symptom onset, leading to a shorter hospital stay.

scholarly journals The effect of oat bran consumption on gestational diabetes: a randomized controlled clinical trial

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Zahra Barati ◽  
Mina Iravani ◽  
Majid Karandish ◽  
Mohammad Hosein Haghighizadeh ◽  
Sara Masihi
Keyword(s):  
Clinical Trial ◽  
Blood Glucose ◽  
Gestational Diabetes ◽  
Fasting Blood Glucose ◽  
Intervention Group ◽  
Control Group ◽  
Controlled Clinical Trial ◽  
Standard Diet ◽  
Oat Bran ◽  
Significant Difference

Abstract Background Gestational diabetes is the most common medical complication in pregnancy, and it has many side effects for the mother and the fetus. The aim of this study was to evaluate the effect of oat bran consumption on gestational diabetes. Methods This study is a randomized clinical trial that was performed on 112 women with gestational diabetes treated with diet. Participants were randomly divided into two groups of 56. Participants in both groups were given a diet for gestational diabetes. In addition to the diet, the intervention group received 30 g of oat bran daily for 4 weeks at lunch and dinner. Tests of fasting blood glucose and two-hour postprandial (2hpp) glucose were taken from both groups: before the intervention, and 2 and 4 weeks after the start of the intervention. Data analysis was performed using SPSS statistical software (version 22) using independent t-test, as well as Chi-square and Mann-Whitney tests. P values less than 0.05 were considered statistically significant. Results There was no statistically significant difference between the two groups in terms of mean blood glucose before the intervention, while 2 and 4 weeks after the intervention, mean fasting blood glucose and two-hour postprandial (2hpp) glucose decreased significantly in the intervention group compared with the control group (P < 0.001). Conclusion Based on the results of this study, the addition of oat bran to the standard diet for pregnant women with gestational diabetes reduced fasting blood glucose and two-hour postprandial (2hpp) glucose. More detailed studies with higher sample sizes are recommended to prove the effectiveness of this valuable dietary supplement. Trial registration IRCT registration number:IRCT20191220045828N1. Registration date: 2020-04-18. Registered while recruiting.

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