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Clinical Trials
A clinical trial is a rigorously controlled test of a new drug or a new invasive medical device on human subjects. In the United States, it is conducted under the direction of the FDA before being made available for general clinical use. Typically clinical studies go through Phase I (safety), Phase II (initial efficacy and dosing), and Phase III (large scale studies assessing both safety and efficacy and may represent a pivotal study that provides the evidence to file a New Drug Application for drug approval).
What are the benefits and risks of participating in a clinical trial?
Benefits
Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:
Risks
There are risks to clinical trials.
HCV Updates:@Clinical Connection
Centerwatch CenterWatch Clinical Trials Listing Services
www.celsion.com - Oncology Drug Development
www.clinicaltrials.gov - Listing of research studies
www.ifpma.org/clinicaltrials- Links information about clinical trials worldwide
HCV Advocate- All the information in one place
Clinical studies at Rockefeller University
Clinical trials at Weill Cornell Medical Center
The Center for the Study of Hepatitis C is actively involved in both clinical research studies and clinical trials for Hepatitis C.
The aim of the clinical research studies is to learn more about the hepatitis C virus, how it replicates, what causes the damage to the liver and what role the immune system plays in the disease.
We participated in the original multicenter trial for hepatitis C treatment and conducted the largest hepatitis-C related clinical trial to date. We continue to participate in current clinical trials with novel therapies for hepatitis C.
Click here for information on Clinical Research Studies currently enrolling participants. You may also find information on our clinical studies at http://www.rucares.org/clinicalstudies/list.php?cat=11&listby=
Click here for information on Clinical Trials at the Center for the Study of Hepatitis C or call 646-962-HEPC (4372). You may also find information on our clinical trials at
In the news
Nov 2012
Hepatitis C-Avoiding Missteps When Evaluating New HCV Trial Data
Oct 2012
Missing Data May Skew Clinical Trials
Hepatitis C And Clinical Trials
Creative Marketing, Clinical Trials, and You
Of Interest
Oct 2012
Geno 1-4: Hepatitis C Drug Development Goes from Pony Ride to Rocket Launch
A few things to remember about clinical trials :
Trial Phase.
A phase 2 trial is riskier then a phase 3 trial and so on..
Why ?
There are a few reasons and one is that the dosage profile is still being adjusted early on in the trial and adverse effects are still being identified.
;Phases of Clinical Trials
In order to find treatments for chronic hepatitis C that are safe and effective, new drugs, including interferons, must undergo rigorous testing. Drug research involves many stages. Much of the knowledge about drugs is derived from initial laboratory research in animals. Animals are used during the drug development phase, primarily to confirm lack of toxicity. If laboratory tests show that a drug has potential therapeutic value without major toxicity, it may advance to the next stage.
The next step involves an extensive process of applying to the FDA for permission to proceed. After careful review of the preliminary data, if the FDA gives its approval, then drug testing – clinical trials – can begin on humans.
Clinical trials may have up to four phases. The initial introduction of an investigational new drug using humans occurs in
Phase I.
The subjects in this phase are usually healthy volunteers (usually 20 to 80 subjects). Sometimes the subjects are those with the disease that is being studied.The goals of a Phase I trial are to evaluate safety and tolerability (i.e., lack of major side effects) as well as the dosage range. This is determined by testing a range of doses (called a dose-ranging trial). Study participants initially receive a low dose of the drug; this is gradually increased as long as the drug appears to be safe.
Phase I studies may provide early indications of the drug’s effectiveness, but whether or not a drug works is the primary focus of Phase II and Phase III studies.
In Phase II trials, clinical studies are conducted using patients with the disease for which the drug is being tested. The goal of this phase is to obtain preliminary data on the effectiveness (also known as efficacy) of the test drug. This phase also allows further collection of data on the common short-term side effects and risks associated with the drug. A relatively small number of participants enroll in Phase II studies (100 to 300 subjects).
After confirming preliminary evidence of effectiveness in Phase II studies, the goal of Phase III studies is to gain additional information on effectiveness and safety. In this phase, several hundred to several thousand subjects receive the test drug.
In Phase III studies, the new drug is often compared to current standard therapy. After the drug is approved and marketed, the FDA may require acompany to obtain more information about the drug. These studies occur as Phase IV trials.
Examples are the safety and efficacy of varying doses,how the drug interacts with other drugs, or how it works in people with other diseases.
Phase IV trials may include small or large numbers o fsubjects and may reveal uncommon side effects that are too rare to show up in Phase II or III studies.
http://www.hcvadvocate.org/
Biotech terms
Fast Track Development Program
Fast Track designation is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. The designation typically enables a Company to submit a New Drug Application (NDA) on a “rolling” basis with ongoing FDA review during the submission process. NDAs with Fast Track designation are also usually granted priority review by FDA at the time of submission.
=======================================================
Special Protocol Assessment
A Special Protocol Assessment (SPA) from the FDA is an agreement that the Phase III trial protocol design, clinical endpoints, and statistical analyses are acceptable to support regulatory approval. An SPA is binding upon the FDA unless a substantial scientific issue essential to determining safety or efficacy is identified after the testing is begun.
========================================================
Abbreviated New Drug Application
An Abbreviated New Drug Application (ANDA) is an application for a U.S. generic drug approval for an existing licensed medication or approved drug. The ANDA contains data which when submitted to FDA's Center for Drug Evaluation and Research, Office of Generic Drugs, provides for the review and ultimate approval of a generic drug product. Once approved, an applicant may manufacture and market the generic drug product to provide a safe, effective, low cost alternative to the American public.
=========================================================
Investigational New Drug
The United States Food and Drug Administration's Investigational New Drug (IND) program is the means by which a pharmaceutical company obtains permission to ship an experimental drug across state lines (usually to clinical investigators) before a marketing application for the drug has been approved. The FDA reviews the IND application for safety to assure that research subjects will not be subjected to unreasonable risk. If the application is approved, the candidate drug usually enters a Phase 1 clinical trial..
==========================================================
New Drug Application (NDA)
Since 1938, every new drug has been the subject of an approved NDA before U.S. commercialization. The NDA application is the vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the U.S. The data gathered during the animal studies and human clinical trials of an Investigational New Drug (IND) become part of the NDA.
The goals of the NDA are to provide enough information to permit FDA reviewer to reach the following key decisions:
* Whether the drug is safe and effective in its proposed use(s), and whether the benefits of the drug outweigh the risks.
* Whether the drug's proposed labeling (package insert) is appropriate, and what it should contain.
* Whether the methods used in manufacturing the drug and the controls used to maintain the drug's quality are adequate to preserve the drug's identity, strength, quality, and purity.
The documentation required in an NDA is supposed to tell the drug's whole story, including what happened during the clinical tests, what the ingredients of the drug are, the results of the animal studies, how the drug behaves in the body, and how it is manufactured, processed and packaged.
From : http://www.chasingthealpha.com/p/fda-terminology.html
Any Benefit In Treating In A Trial ?
An obvious benefit of being in a clinical trial is the cost, its free. But, if you have insurance you may be billed for both blood work, and office visits.
Important Questions To Ask
The Policy On Dose Reductions
Ask the trial coordinator about the policy for dose reductions in order to manage any adverse effects. Will they let you adjust the dose if side effects become either a medical problem or intolerable.
Do they allow Rescue Drugs ?
These drugs may be needed to complete treatment ; neupogen to bring up the low white counts and epogen for red blood counts.
Ask if they will cover the cost of any rescue drugs.
What If You Relapse ?
Will you be offered a rollover trial ? A trial with another protocol ?
What is all this Placebo Arm Stuff ?
1-If you are in the "Placebo Group, or Arm" and receiving the placebo will they allow you to treat with the trial medications later ?
2-It is possible you may not receive the best dose, depending on what treatment Arm you have been placed in, remembering that often the optimum dose is often discovered after the trial is over.
Consent forms
.l They should and must have a consent form, you should receive a copy you can review before you sign it. Most likely you will sign it in the doctors office, when you do, think of me, and tell yourself you should be taking this home to read. You are not doing your homework, never sign anything before you read it.
Testing For Viral Load
Ask what PCR test will be used and at what sensitivity. They often use an in house test to save money. The viral load test should measure at least down to 50 viral copies per ml. of blood . There are ultra sensitive tests for research that can detect less than 5 copies. Find out if you are privy to the PCR results.
Criteria For Stopping Treatment
You should know the viral response or protocol of when treatment will be discontinued.
Examples : Complete early viral response (cEVR): a viral load ofless than 50 IU/mL 12 weeks into treatment.
Do they want you to be virus free by the forth week, eighth, or at week twelve ?
In Conclusion
An important factor in treating this disease is the success rate achieved
as " Treatment-Naïve ". If you enter into the wrong trial, and respond slowly, or have a break through and maybe even relapse could you be lowering your next chance at clearing this virus ? Would you have achieved SVR if the protocol were different ?
, .
If you do enter into a trial read the trial agreement and look closely at the protocol with each arm. Compare SOC or "standard of care" therapy including the newly FDA approved PIs (Telaprevir/Incivek Prescribing Information
Medication Guide / VICTRELIS™- Boceprevir: Prescribing Information and Medication Guide ) with the FDA approved protocol. ;
How much do they differ ?
How much of a risk are you taking ?
Before signing on the dotted line ask yourself if you did all your homework.
Summary of investigational Hepatitis C drugs presented at the 2011 EASL Conference
Hepatitis C New Drug Pipeline; New drugs in development for the treatment of hepatitis C
http://www.hcvdrugs.com/
Making Sense of Hepatitis C Research and Medical Literature
How to Evaluate a Clinical Trial
Guide to Understanding Clinical Trials and Medical Research in Hepatitis C
A clinical trial is a rigorously controlled test of a new drug or a new invasive medical device on human subjects. In the United States, it is conducted under the direction of the FDA before being made available for general clinical use. Typically clinical studies go through Phase I (safety), Phase II (initial efficacy and dosing), and Phase III (large scale studies assessing both safety and efficacy and may represent a pivotal study that provides the evidence to file a New Drug Application for drug approval).
What are the benefits and risks of participating in a clinical trial?
Benefits
Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:
- Play an active role in their own health care.
- Gain access to new research treatments before they are widely available.
- Obtain expert medical care at leading health care facilities during the trial.
- Help others by contributing to medical research.
Risks
There are risks to clinical trials.
- There may be unpleasant, serious or even life-threatening side effects to experimental treatment.
- The experimental treatment may not be effective for the participant.
- The protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements
Where Can I Find A Trial In My Area ?
HCV Updates:@Clinical Connection
Centerwatch CenterWatch Clinical Trials Listing Services
www.celsion.com - Oncology Drug Development
www.clinicaltrials.gov - Listing of research studies
www.ifpma.org/clinicaltrials- Links information about clinical trials worldwide
HCV Advocate- All the information in one place
Clinical studies at Rockefeller University
Clinical trials at Weill Cornell Medical Center
The Center for the Study of Hepatitis C is actively involved in both clinical research studies and clinical trials for Hepatitis C.
The aim of the clinical research studies is to learn more about the hepatitis C virus, how it replicates, what causes the damage to the liver and what role the immune system plays in the disease.
We participated in the original multicenter trial for hepatitis C treatment and conducted the largest hepatitis-C related clinical trial to date. We continue to participate in current clinical trials with novel therapies for hepatitis C.
Click here for information on Clinical Research Studies currently enrolling participants. You may also find information on our clinical studies at http://www.rucares.org/clinicalstudies/list.php?cat=11&listby=
Click here for information on Clinical Trials at the Center for the Study of Hepatitis C or call 646-962-HEPC (4372). You may also find information on our clinical trials at
In the news
Nov 2012
Hepatitis C-Avoiding Missteps When Evaluating New HCV Trial Data
Oct 2012
Missing Data May Skew Clinical Trials
Hepatitis C And Clinical Trials
Creative Marketing, Clinical Trials, and You
Of Interest
Oct 2012
Geno 1-4: Hepatitis C Drug Development Goes from Pony Ride to Rocket Launch
A few things to remember about clinical trials :
Trial Phase.
A phase 2 trial is riskier then a phase 3 trial and so on..
Why ?
There are a few reasons and one is that the dosage profile is still being adjusted early on in the trial and adverse effects are still being identified.
;Phases of Clinical Trials
In order to find treatments for chronic hepatitis C that are safe and effective, new drugs, including interferons, must undergo rigorous testing. Drug research involves many stages. Much of the knowledge about drugs is derived from initial laboratory research in animals. Animals are used during the drug development phase, primarily to confirm lack of toxicity. If laboratory tests show that a drug has potential therapeutic value without major toxicity, it may advance to the next stage.
The next step involves an extensive process of applying to the FDA for permission to proceed. After careful review of the preliminary data, if the FDA gives its approval, then drug testing – clinical trials – can begin on humans.
Clinical trials may have up to four phases. The initial introduction of an investigational new drug using humans occurs in
Phase I.
The subjects in this phase are usually healthy volunteers (usually 20 to 80 subjects). Sometimes the subjects are those with the disease that is being studied.The goals of a Phase I trial are to evaluate safety and tolerability (i.e., lack of major side effects) as well as the dosage range. This is determined by testing a range of doses (called a dose-ranging trial). Study participants initially receive a low dose of the drug; this is gradually increased as long as the drug appears to be safe.
Phase I studies may provide early indications of the drug’s effectiveness, but whether or not a drug works is the primary focus of Phase II and Phase III studies.
In Phase II trials, clinical studies are conducted using patients with the disease for which the drug is being tested. The goal of this phase is to obtain preliminary data on the effectiveness (also known as efficacy) of the test drug. This phase also allows further collection of data on the common short-term side effects and risks associated with the drug. A relatively small number of participants enroll in Phase II studies (100 to 300 subjects).
After confirming preliminary evidence of effectiveness in Phase II studies, the goal of Phase III studies is to gain additional information on effectiveness and safety. In this phase, several hundred to several thousand subjects receive the test drug.
In Phase III studies, the new drug is often compared to current standard therapy. After the drug is approved and marketed, the FDA may require acompany to obtain more information about the drug. These studies occur as Phase IV trials.
Examples are the safety and efficacy of varying doses,how the drug interacts with other drugs, or how it works in people with other diseases.
Phase IV trials may include small or large numbers o fsubjects and may reveal uncommon side effects that are too rare to show up in Phase II or III studies.
http://www.hcvadvocate.org/
Biotech terms
Fast Track Development Program
Fast Track designation is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. The designation typically enables a Company to submit a New Drug Application (NDA) on a “rolling” basis with ongoing FDA review during the submission process. NDAs with Fast Track designation are also usually granted priority review by FDA at the time of submission.
=======================================================
Special Protocol Assessment
A Special Protocol Assessment (SPA) from the FDA is an agreement that the Phase III trial protocol design, clinical endpoints, and statistical analyses are acceptable to support regulatory approval. An SPA is binding upon the FDA unless a substantial scientific issue essential to determining safety or efficacy is identified after the testing is begun.
========================================================
Abbreviated New Drug Application
An Abbreviated New Drug Application (ANDA) is an application for a U.S. generic drug approval for an existing licensed medication or approved drug. The ANDA contains data which when submitted to FDA's Center for Drug Evaluation and Research, Office of Generic Drugs, provides for the review and ultimate approval of a generic drug product. Once approved, an applicant may manufacture and market the generic drug product to provide a safe, effective, low cost alternative to the American public.
=========================================================
Investigational New Drug
The United States Food and Drug Administration's Investigational New Drug (IND) program is the means by which a pharmaceutical company obtains permission to ship an experimental drug across state lines (usually to clinical investigators) before a marketing application for the drug has been approved. The FDA reviews the IND application for safety to assure that research subjects will not be subjected to unreasonable risk. If the application is approved, the candidate drug usually enters a Phase 1 clinical trial..
==========================================================
New Drug Application (NDA)
Since 1938, every new drug has been the subject of an approved NDA before U.S. commercialization. The NDA application is the vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the U.S. The data gathered during the animal studies and human clinical trials of an Investigational New Drug (IND) become part of the NDA.
The goals of the NDA are to provide enough information to permit FDA reviewer to reach the following key decisions:
* Whether the drug is safe and effective in its proposed use(s), and whether the benefits of the drug outweigh the risks.
* Whether the drug's proposed labeling (package insert) is appropriate, and what it should contain.
* Whether the methods used in manufacturing the drug and the controls used to maintain the drug's quality are adequate to preserve the drug's identity, strength, quality, and purity.
The documentation required in an NDA is supposed to tell the drug's whole story, including what happened during the clinical tests, what the ingredients of the drug are, the results of the animal studies, how the drug behaves in the body, and how it is manufactured, processed and packaged.
From : http://www.chasingthealpha.com/p/fda-terminology.html
Any Benefit In Treating In A Trial ?
An obvious benefit of being in a clinical trial is the cost, its free. But, if you have insurance you may be billed for both blood work, and office visits.
Important Questions To Ask
The Policy On Dose Reductions
Ask the trial coordinator about the policy for dose reductions in order to manage any adverse effects. Will they let you adjust the dose if side effects become either a medical problem or intolerable.
Do they allow Rescue Drugs ?
These drugs may be needed to complete treatment ; neupogen to bring up the low white counts and epogen for red blood counts.
Ask if they will cover the cost of any rescue drugs.
What If You Relapse ?
Will you be offered a rollover trial ? A trial with another protocol ?
What is all this Placebo Arm Stuff ?
1-If you are in the "Placebo Group, or Arm" and receiving the placebo will they allow you to treat with the trial medications later ?
2-It is possible you may not receive the best dose, depending on what treatment Arm you have been placed in, remembering that often the optimum dose is often discovered after the trial is over.
Consent forms
.l They should and must have a consent form, you should receive a copy you can review before you sign it. Most likely you will sign it in the doctors office, when you do, think of me, and tell yourself you should be taking this home to read. You are not doing your homework, never sign anything before you read it.
Testing For Viral Load
Ask what PCR test will be used and at what sensitivity. They often use an in house test to save money. The viral load test should measure at least down to 50 viral copies per ml. of blood . There are ultra sensitive tests for research that can detect less than 5 copies. Find out if you are privy to the PCR results.
Criteria For Stopping Treatment
You should know the viral response or protocol of when treatment will be discontinued.
Examples : Complete early viral response (cEVR): a viral load ofless than 50 IU/mL 12 weeks into treatment.
Do they want you to be virus free by the forth week, eighth, or at week twelve ?
In Conclusion
An important factor in treating this disease is the success rate achieved
as " Treatment-Naïve ". If you enter into the wrong trial, and respond slowly, or have a break through and maybe even relapse could you be lowering your next chance at clearing this virus ? Would you have achieved SVR if the protocol were different ?
, .
If you do enter into a trial read the trial agreement and look closely at the protocol with each arm. Compare SOC or "standard of care" therapy including the newly FDA approved PIs (Telaprevir/Incivek Prescribing Information
Medication Guide / VICTRELIS™- Boceprevir: Prescribing Information and Medication Guide ) with the FDA approved protocol. ;
How much do they differ ?
How much of a risk are you taking ?
Before signing on the dotted line ask yourself if you did all your homework.
Summary of investigational Hepatitis C drugs presented at the 2011 EASL Conference
Hepatitis C New Drug Pipeline; New drugs in development for the treatment of hepatitis C
http://www.hcvdrugs.com/
Making Sense of Hepatitis C Research and Medical Literature
How to Evaluate a Clinical Trial
Guide to Understanding Clinical Trials and Medical Research in Hepatitis C