A control group refers to a group of patients in a study, that any intervention group is compared to. This helps to show that the intervention actually caused what was seen and that it wouldn’t have happened anyway.
One common type of control group is to use a placebo.
In the example above, all patients get the best treatment with or without the new drug.
If, for example, this is a new HIV drug and the best treatment already includes 3 active drugs, then it could be difficult to see any difference between the new drug and the placebo, because both groups will already do very well.
Another type of control group is a group where no intervention takes place.
This example might be used where there is something about the trial drug that makes using a placebo difficult – perhaps because it is given by injection.
The difficulty of not randomising the control group to having a placebo is that you can never be sure whether some of the things (both good and bad) that happened to patients in the active drug arm, are not due to chance.
More importantly, people in each arm may behave differently because they know they are getting active drug, for example, by reporting more side effects.
The example below uses a drug or combination that has already been studied as a control group.
This is still generally the type of trial design used for studying a new HIV drug in people who have not yet used HIV treatment. This is generally okay, so long as the new drug turns out to be better than, or at least as good as, the current standard of care.
For this reason, early trials with this design should not enrol people who have advanced HIV (for example with CD4 counts less than 100 cells/mm3) as these people will need to rely on a proven treatment.
Randomising patients should mean that important factors – both known and unknown – are likely to be distributed between each group. For example, having the similar numbers of women, Caucasians, smokers, CD4 counts etc in each group.
Last updated: 21 July 2009.
Intervention: Icatibant (Drug); Placebo (Drug)
Status: Recruiting
Sponsored by: Shire
Official(s) and/or principal investigator(s):
Vipin Aggarwal, MD, MPH, Study Director, Affiliation: Shire
Overall contact:
ShireHGT Call Center, Phone: +1-866-842-5335, Email: US_ShireHGT_Medicalinformation@shire. com
Summary
This study is being conducted to compare the safety and efficacy of icatibant with placebo in the treatment for Angiotensin-Converting Enzyme Inhibitor (ACE-I)-Induced Angioedema in Adults.
Clinical Details
Official title: Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Study Evaluating the Safety & Efficacy of Icatibant as a Treatment for Angiotensin-Converting Enzyme Inhibitor (ACE-I)-Induced Angioedema in Adults
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: Time to Meeting Discharge Criteria (TMDC).
Safety and tolerability of Icatibant.
Secondary outcome: Time to Onset of Symptom Relief (TOSR).
Occurrence of airway intervention due to ACE-I-induced angioedema.
Admission to the hospital or intensive care unit (ICU).
Angioedema attack following study drug administration.
Time to meeting discharge criteria (TMDC).
Detailed description: Angiotensin-converting enzyme inhibitors (ACE-Is) are the class of medications prescribed most frequently for the treatment of hypertension. They are also used post myocardial infarction as well as in patients with heart failure, diabetes mellitus, and chronic kidney disease. Approximately 35 to 40 million patients are on ACE-Is worldwide. Study HGT-FIR-096 is a multicenter, Phase III, randomized, double-blind, two-armed, placebo-controlled trial. The study population will consist of 118 adult patients, 18 years of age or older, who present with an acute ACE-I-induced angioedema attack. The primary aim of the study is to demonstrate that icatibant is significantly more effective than placebo in resolving attacks of angioedema caused by ACE-I based on the Time to Meeting Discharge Criteria (TMDC). Safety and tolerability, as well as the pharmacokinetics (PK), of icatibant will also be evaluated. Eligible patients will be randomized at a 1: 1 ratio to receive a single sub-cutaneous injection of either 30 mg icatibant or placebo.
Eligibility
Minimum age: 18 Years. Maximum age: N/A. Gender(s): Both.
Criteria: Inclusion Criteria: 1. Male or female, 18 years of age or older. 2. Patient is currently being treated with an ACE inhibitor. 3. Patient presenting with an ACE inhibitor-induced angioedema attack of the head and/or neck region within 12 hours of onset (must be sufficiently less than 12 hours to allow study drug to be given with 12 hours of attack onset). 4. Angioedema must be considered at least moderate in severity for at least one of the four angioedema-associated airway symptoms (difficulty breathing, difficulty swallowing, voice changes, tongue swelling). 5. Patient must have voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient. 6. Females must have a negative urine pregnancy test prior to administration of the study medication, with the exception of those females who have had a total hysterectomy or bilateral oophorectomy, or who are 2 years post-menopausal. Exclusion Criteria: 1. Patient has a diagnosis of angioedema of other etiology (eg, hereditary or acquired angioedema, allergic angioedema [eg, food, insect bite or sting, evident clinical response to antihistamines and/or corticosteroids], anaphylaxis, trauma, abscess or infection or associated disease, local inflammation, local tumor, post-operative or post-radiogenic edema, salivary gland disorders, other [non-ACE inhibitor] drug-induced angioedema). 2. Patients with a family history of recurrent angioedema. 3. Patients who have had a previous episode(s) of angioedema while not on ACE inhibitor therapy. 4. Patients with acute urticaria (itchy, erythematous wheals). 5. Patients who have an intervention to support the airway (eg, intubation, tracheotomy, cricothyrotomy) due to the current attack of angioedema. 6. Patient has any of the following vascular conditions that, in the judgment of the investigator, would be a contraindication to participation in the study.
- Unstable angina pectoris or acute myocardial ischemia
- Hypertensive urgency or emergency (diastolic blood pressure [DBP] >120 mm Hg or
systolic blood pressure [SBP] >180 mm Hg)
- Within 1 month of a stroke or transient ischemic attack
- New York Heart Association (NYHA) heart failure class IV
7. Patient has a serious or acute condition or illness that, in the judgment of the investigator, would interfere with evaluating the safety and/or efficacy assessments of the study (eg, a condition or illness requiring hemodialysis). 8. Patient is pregnant or breast feeding. 9. Patient has participated in another investigational study in the past 30 days. 10. Patient is unable to understand the nature, scope, and possible consequences of the protocol, or is unlikely or unable to comply with the protocol assessments, or is unlikely to complete the study for any reason. 11. Patients who are not suitable for the study in the opinion of the investigator. 12. Patient has experienced hypersensitivity to the active substance of the investigational product or to any of its excipients.
Locations and Contacts
ShireHGT Call Center, Phone: +1-866-842-5335, Email: US_ShireHGT_Medicalinformation@shire. com Hals - Nasen - Ohrenklinik und Poliklinik, Munich 81675, Germany; Withdrawn
Klinik fГјr Hals-, Nasen - und Ohrenheilkunde, Ulm 89075, Germany; Withdrawn
Soroka University Medical Center, Beer Sheva 84101, Israel; Recruiting
Rita Troitsa, Phone: +97286244245, Email: ritaTr@clalit. org. il
Carmi Bartal, MD, Principal Investigator
Bnai Zion Medical Center, Haifa 31048, Israel; Recruiting
Regina Peri, Phone: +97248359659, Email: regina. peri@b-zion. org. il
Elias Toubi, MD, Principal Investigator
Ziv Medical Center, Safed 13100, Israel; Recruiting
Karina Shestatski, Phone: +97246828114, Email: clinical@ziv. health. gov. il
Osamah Hussein, MD, Principal Investigator
Tel-Aviv Sourasky Medical Center, Tel Aviv 64239, Israel; Recruiting
Dania Dror, Phone: +972524498779, Email: daniadror@gmail. com
Shmuel Kivity, MD, Principal Investigator
Brighton and Sussex University Hospitals NHS Trust, Brighton BN2 5BE, United Kingdom; Recruiting
Manchester Royal Infirmary, Manchester M13 9WL, United Kingdom; Recruiting
Rachel Pearson, Phone: +441612766777, Email: rachel. pearson2@cmft. nhs. uk
Richard Body, MD, Principal Investigator
Queen's Medical Centre, Nottingham NG7 2UH, United Kingdom; Recruiting
Philip Miller, Phone: +441159249924, Ext: 67898, Email: philip. miller@nuh. nhs. uk
Christopher Gough, MD, Principal Investigator
University of California San Diego, La Jolla, California 92093, United States; Recruiting
Jennifer Blair, Phone: 858-822-6599, Email: jcblair@ucsd. edu
Marc Riedl, MD, Principal Investigator
University of California San Diego Medical Center, LaJolla, California 92037, United States; Recruiting
Jill Nyland, Phone: 224-610-1310, Email: jill. nyland@va. gov
Rohit Arora, MD, Principal Investigator
Saint Francis Medical Center, Peoria, Illinois 61606, United States; Withdrawn
University of Kansas Cancer Center, Kansas City, Kansas 66160, United States; Recruiting
Jeanie Whipple, Phone: 913-588-4643, Email: jwhipple@kumc. edu
Chris Cannon, MD, Principal Investigator
Ochsner Medical Center, New Orleans, Louisiana 70121, United States; Recruiting
Kelly Boudet, Phone: 504-842-5257, Email: kboudet@ochsner. org
Eiman Jahangir, MD, Principal Investigator
Louisiana State University Health Science Center, New OrlГ©ans, Louisiana 70070, United States; Withdrawn
Brigham and Womens Hospital, Boston, Massachusetts 02115, United States; Recruiting
Marian Tilearcio, Phone: 617-732-5638, Email: mtilearcio@partners. org
Daniel Pallin, MD, Principal Investigator
Massachusetts General Hospital, Boston, Massachusetts 02114, United States; Recruiting
Matthew Iandoli, Phone: 617-724-1714, Email: miandoli@mgh. harvard. edu
Aleena Banerji, MD, Principal Investigator
Baystate Medical Center, Springfield, Massachusetts 01199, United States; Recruiting
Kye Poronsky, BS, Phone: 413-794-4421, Email: kye. poronsky@baystatehealth. org
Howard Smithline, MD, Principal Investigator
Detroit Receiving Hospital and University Health Center, Detroit, Michigan 48201, United States; Recruiting
Temple University Hospital, Philadelphia, Pennsylvania 19140, United States; Recruiting
Patricia McNelis, Phone: 215-707-2235, Email: patricia. mcnelis@tuhs. temple. edu
Megan Healy, MD, Principal Investigator
Allegheny General Hospital, Pittsburgh, Pennsylvania 15143, United States; Recruiting
SueAnn Livingston, Phone: 412-359-8763, Email: saliving@wpahs. org
Arvind Venkat, MD, Principal Investigator
Rhode Island Hospital, Providence, Rhode Island 02903, United States; Recruiting
Karina Bertsch, CCRP, Phone: 401-444-9264, Email: kbertsch@lifespan. org
Gregory Jay, MD, Principal Investigator
University of South Carolina School of Medicine, Columbia, South Carolina 29203, United States; Recruiting
By Steven Bratman, M. D.
I once took alternative medicine on faith. For decades, I practiced it on patients and myself and my family, and assumed that pretty much all of it worked. Then I learned about double-blind studies, and it was like a tornado blowing down a house of cards. I discovered that I, like most people who love alternative medicine, had made a huge (though understandable) mistake.
Steven Bratman, M. D.
I had thought it was possible to know whether a treatment worked by trying it. I had also thought I could trust tradition, anecdote, and authority. I now see otherwise. The insights of the double-blind trial have cut through my wishful thinking and idealism, and turned me into a hard-nosed skeptic. Show me the double-blind studies, and I’ll pay attention. Otherwise, so far as I’m concerned, it’s little more than hot air.
Warning: This isn’t an easy subject. But if you read this through, and think about it, you will never look at alternative medicine (or any form of medicine) the same way again.
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