scholarly journals A double-blind, placebo-controlled phase II study of the efficacy and safety of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease

2014 ◽  
Vol 89 (7) ◽  
pp. 709-713 ◽  
Author(s):  
Marvin E. Reid ◽  
Amal El Beshlawy ◽  
Adlette Inati ◽  
Abdullah Kutlar ◽  
Miguel R. Abboud ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Phase Ii ◽  
Phase Ii Study ◽  
Efficacy And Safety ◽  
Cell Disease ◽  
Double Blind ◽  
Double Blind Placebo

Sevuparin for the treatment of acute pain crisis in patients with sickle cell disease: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

2021 ◽  
Vol 8 (5) ◽  
pp. e334-e343
Author(s):  
Bart J Biemond ◽  
Anil Tombak ◽  
Yurdanur Kilinc ◽  
Murtadha Al-Khabori ◽  
Miguel Abboud ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Acute Pain ◽  
Phase 2 ◽  
Cell Disease ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Phase 2 Trial ◽  
Pain Crisis

scholarly journals A collaborative, double-blind randomized study of cetiedil citrate in sickle cell crisis

Blood ◽  
1986 ◽  
Vol 67 (5) ◽  
pp. 1442-1447 ◽  
Author(s):  
LJ Benjamin ◽  
LR Berkowitz ◽  
E Orringer ◽  
VN Mankad ◽  
AS Prasad ◽  
...  
Keyword(s):  
Body Weight ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Adverse Reactions ◽  
Randomized Study ◽  
Drug Dosage ◽  
Cell Disease ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Sickle Cell Crisis

Abstract We have recently completed a double-blind, placebo-controlled, noncrossover study, the goal of which was to determine whether cetiedil citrate (cetiedil) could affect the course of vaso-occlusive crises in sickle cell disease. Patients, who presented to the emergency room at least 4 but no more than 24 hours after the onset of a painful vasoocclusive crisis severe enough to require hospitalization, were considered candidates for the study. Each patient received either placebo or cetiedil at one of the following three dosages: 0.2, 0.3, or 0.4 mg/kg body weight. The assigned drug dosage was given as a 30 minute intravenous infusion every 8 hours for 4 consecutive days. A total of 67 patients was enrolled in the study. Cetiedil, at its highest dosage (0.4 mg/kg body weight), was found to be significantly superior to placebo both in reducing the number of painful sites present on all 4 treatment days and in shortening the total time in crisis. No serious adverse reactions were observed during the course of the study. We conclude that cetiedil, given at a dosage of 0.4 mg/kg body weight, is therapeutically advantageous for sickle cell crisis.

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