Small clinical trials: Are they all bad?

1995 ◽  
Vol 14 (2) ◽  
pp. 115-126 ◽  
Author(s):  
John N. S. Matthews
Keyword(s):  
Clinical Trials ◽  
Small Clinical Trials

scholarly journals Merged block randomisation: A novel randomisation procedure for small clinical trials

2019 ◽  
Vol 16 (3) ◽  
pp. 246-252 ◽  
Author(s):  
Stéphanie L van der Pas
Keyword(s):  
Clinical Trials ◽  
Simple Procedure ◽  
Good Choice ◽  
Main Concern ◽  
Treatment Groups ◽  
Biased Coin Design ◽  
Block Randomisation ◽  
Number Of Patients ◽  
Single Centre Study ◽  
Small Clinical Trials

Background/Aims: Randomisation in small clinical trials is a delicate matter, due to the tension between the conflicting aims of balanced groups and unpredictable allocations. The commonly used method of permuted block randomisation has been heavily criticised for its high predictability. This article introduces merged block randomisation, a novel and conceptually simple restricted randomisation design for small clinical trials (less than 100 patients per stratum). Merged block randomisation is a simple procedure that can be carried out without need for a computer. Merged block randomisation is not restricted to 1:1 randomisation, but is readily applied to unequal target allocations and to more than two treatment groups. Methods: The position of merged block randomisation on the spectrum of balance and predictability is investigated in a simulation study, in two common situations: a single-centre study and a multicentre study (with sampling stratified per centre). Methods included for comparison were permuted block randomisation, Efron’s biased coin design, the maximal procedure, the block urn design and the big stick design. Results: Compared to permuted block randomisation with blocks of size 4, merged block randomisation has the same maximum tolerated imbalance and is thus as impervious to chronological bias, with the added benefit of being less predictable. Each method in the study takes a different position on the balance/determinism spectrum, and none was uniformly best. Merged block randomisation was either less predictable or more balanced than the other methods, in all simulation settings. Conclusion: Merged block randomisation is a versatile restricted randomisation method that outperforms permuted block randomisation and is a good choice for small clinical trials where imbalance is a main concern, especially in multicentre trials where the number of patients per centre may be small.

Pharmacotherapy of Epilepsy: Focus on Levetiracetam

2009 ◽  
Vol 1 ◽  
pp. CMT.S1096
Author(s):  
Molly E. Adams ◽  
Allison M. Chung ◽  
Lea S. Eiland
Keyword(s):  
United States ◽  
Adverse Effect ◽  
Clinical Trials ◽  
Antiepileptic Drug ◽  
Common Adverse Effect ◽  
The United States ◽  
Behavioral Effects ◽  
Good Tolerability ◽  
Refractory Seizures ◽  
Small Clinical Trials

Levetiracetam is a second-generation antiepileptic drug which first came to the United States market in 1999. It has a mechanism of action that is not well elucidated. However, it is a very favorable antiepileptic drug due to its reliable pharmacokinetics, minimal drug interactions, seizure efficacy and good tolerability. It is an agent that has established efficacy as an adjunct therapy agent for partial and refractory seizures. As a monotherapy agent, levetiracetam also appears to be an attractive agent with observed efficacy and tolerability. Since levetiracetam has recently become available intravenously, it is also being reviewed as an agent for acute status epilepticus. In pediatrics, levetiracetam is widely used with efficacy seen in small clinical trials for a variety of seizure types. Levetiracetam is well tolerated: the most common adverse effect being somnolence and behavioral effects. Overall, levetiracetam is a notable antiepileptic drug that has added significantly to the current antiepileptic armamentarium.

AGAIN, THE PERFIDY OF SMALL CLINICAL TRIALS

PEDIATRICS ◽  
1989 ◽  
Vol 83 (5) ◽  
pp. A63-A63
Author(s):  
Student
Keyword(s):  
Clinical Trials ◽  
Prophylactic Treatment ◽  
Study Groups ◽  
Small Study ◽  
Febrile Convulsions ◽  
Small Clinical Trials

The analysis [of British trials of phenobarbitone and valproate for the prophylactic treatment of febrile convulsions] shows well how small study groups serve to exaggerate benefits of drawbacks of treatment. All the trials have been too small for paediatricians to use their findings with confidence.

Issues in designing and interpreting small clinical trials

2021 ◽  
Author(s):  
Andrew D. Althouse ◽  
Jonathan G. Yabes ◽  
Kaleab Z. Abebe
Keyword(s):  
Clinical Trials ◽  
Small Clinical Trials

scholarly journals When the MAGIC stops: magnesium in acute coronary syndromes — a lesson in medical humility

CJEM ◽  
2004 ◽  
Vol 6 (02) ◽  
pp. 123-125 ◽  
Author(s):  
Shannon Patrick ◽  
Mel Herbert
Keyword(s):  
Myocardial Infarction ◽  
Acute Myocardial Infarction ◽  
Clinical Trials ◽  
Acute Coronary Syndromes ◽  
Animal Studies ◽  
Coronary Syndromes ◽  
Small Clinical Trials

ABSTRACT There has been much debate as to whether magnesium, a well-tolerated, readily available and cheap therapy, should be used to treat patients with suspected myocardial infarction. Despite promising results from animal studies and small clinical trials conducted in the 1980s, two large recent trials have concluded that the once phenomenal treatment is ineffective. The story of magnesium for acute myocardial infarction is a lesson in medical humility.

scholarly journals Novel treatments for progressive multifocal leukoencephalopathy

2020 ◽  
Vol 81 (7) ◽  
pp. 1-9
Author(s):  
Karina M Bennett ◽  
Peter M Fernandes
Keyword(s):  
Clinical Trials ◽  
Progressive Multifocal Leukoencephalopathy ◽  
Treatment Options ◽  
Bk Virus ◽  
Novel Therapies ◽  
Immune Systems ◽  
Novel Treatments ◽  
Demyelinating Disorder ◽  
Blocking Antibodies ◽  
Small Clinical Trials

Progressive multifocal leukoencephalopathy is a rare demyelinating disorder of the CNS, caused by John Cunningham virus, that occurs in those with impaired immune systems. Existing treatment options are ineffective or unproven. This article reviews research into novel therapies: immune checkpoint-blocking antibodies (nivolumab and pembrolizumab), allogenic BK virus-specific T cell treatment and filgrastim. Results for these therapies in small clinical trials are promising, but further research is required to assess efficacy fully.

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