About

New to Clinical Trials

Living with a disease or condition impacting your health, or the health of a loved one, can be stressful or even life-altering. But new therapies are constantly in development for hundreds of indications – many of which are being tested today through clinical trials in patients and healthy volunteers. Through these trials, and by conducting them in controlled environments, researchers and medical professionals can determine if a treatment is safe and efficacious, and how it can be used to treat a specific disease or condition.

Understanding Clinical Trials

Clinical trials are part of the final steps before an investigational intervention can be approved for general public use among patients. They can study experimental drugs, vaccines, medical devices, medical procedures, or behavioral interventions as well as existing ones that warrant further study. Both safety and efficacy are considered, and all are carried out through well-controlled, time-refined methods with highly trained researchers and medical professionals.

At Acceleron we are dedicated to developing innovative, life-changing medicines for patients with a wide range of serious and rare diseases and are motivated by our commitment to bettering the lives of the patients we serve.

The Sponsor

The person(s) or organization who initiates and funds the clinical trial.

The Site

A location where the clinical trial is being conducted, including hospitals, universities, clinics, and doctors’ offices.

The Principal Investigator

The person (healthcare professional or researcher) who is responsible for overseeing the conduct of the clinical trial at the site.

The Study Coordinator

A person who is responsible for supporting the study maintenance activities at the site, including oversight of study participants.

The Participant

A person who has volunteered to join a clinical trial and agrees to have information collected as part of their clinical trial participation.

Ethics Review Boards

A group of individuals that determine whether a clinical trial is being conducted within guidelines that demonstrate participants are treated fairly.

Phases of

Clinical Trials

Clinical Trial Phases

Clinical trials study the safety and efficacy of experimental therapies in healthy volunteers and in people with a specific illness or condition. Clinical trials typically go through a series of phases. The entire clinical trial process for an approved intervention takes an average of 10 years.

Pre-clinical

Prior to progressing to any in-human phase (Phase I through IV), these treatments have already undergone extensive study in labs – called pre-clinical development – and have demonstrated positive effects on a disease. The treatment must also be approved by health authorities to be tested in patients, and prior to moving on to the next phase.

Phases of Clinical Trials

Phase I

Phase I trials are usually the first time an experimental treatment is studied in humans. The main focus of Phase I studies is to examine an intervention’s safety and see how it interacts with the body – these studies are typically conducted in healthy volunteers. Often includes less than 100 participants

Participants are healthy volunteers or patients with a specific condition
Analyzes how the intervention interacts with the body and any potential side effects
Takes on average about 2 years to complete
To determine a safe dosage to be given
About 58% of interventions successfully advance to Phase II

Phases of Clinical Trials

Phase I

Participants are healthy volunteers or patients with a specific condition
Analyzes how the intervention interacts with the body and any potential side effects
Takes on average about 2 years to complete
To determine a safe dosage to be given
About 58% of interventions successfully advance to Phase II

Phase II

Phase II trials are done to learn if an experimental treatment helps people who have the disease or condition. Efficacy of the treatment on this patient population is evaluated, while also collecting data about safety and side effects.

Usually includes 100-500 participants
Participants have a specific condition
Participants are sometimes given a placebo (inactive version of an experimental drug)
Takes on average about 3 years to complete
About 31% of Phase II trials are successful enough to move to Phase III

Phase III

Phase III trials are in many cases the last stage of research before an intervention can be approved for general public use. Phase III clinical trials will often be placebo-controlled – you will be randomly assigned to receive either active treatment or a safe inactive version of an experimental drug – and double-blind – which means neither you nor the research team will know if you’re receiving active treatment or placebo. These requirements are necessary to learn how a treatment works compared to standard therapy.

Usually includes 300-3,000 participants
Participants have a specific condition
Takes on average about 3 years to complete
About 60% of Phase III trials are successful enough to be submitted for health authority approval, and about 90% of those receive approval (which takes around an additional 1.5 years)

Phase I

Participants are healthy volunteers or patients with a specific condition
Analyzes how the intervention interacts with the body and any potential side effects
Takes on average about 2 years to complete
To determine a safe dosage to be given
About 58% of interventions successfully advance to Phase II

Phase II

Usually includes 100-500 participants
Participants have a specific condition
Participants are sometimes given a placebo (inactive version of an experimental drug)
Takes on average about 3 years to complete
About 31% of Phase II trials are successful enough to move to Phase III

Phase III

Usually includes 300-3,000 participants
Participants have a specific condition
Takes on average about 3 years to complete
About 60% of Phase III trials are successful enough to be submitted for health authority approval, and about 90% of those receive approval (which takes around an additional 1.5 years)

Why Participate in a Clinical Trial

Clinical trials drive the advancement of experimental medicines and treatments and could not happen without people like you. Your participation gives you the opportunity to play a critical role in medical research leading to better healthcare outcomes for yourself or fellow patients in the future.

All Acceleron studies are thoughtfully designed with the highest ethical and safety standards. Participants are valued and closely monitored throughout – both to ensure your health and the success of the study. Taking part in a clinical trial is voluntary and you can leave the study at any time. That being said, it is in the patient’s best interest to weigh the potential risks and benefits of a clinical trial. It’s important to discuss participation with your primary healthcare provider so you are fully aware of all considerations.

Possible Benefits of Clinical Trials

Access to experimental medications and treatments not yet available to the public

A specialized team of healthcare professionals focused on you and care for your disease or condition

Taking an active role in your healthcare and greater understanding of your disease or condition

Contribute scientific knowledge which will be used to improve medical care for other people affected by your disease or condition

Possible Risks of Clinical Trials

You may not be able choose the treatment you receive, including the dosage level or if you get a placebo (inactive version of an experimental drug)

The benefits of investigational treatments are being studied and therefore may not be better than available treatments or could have unexpected side effects

May require more time than your standard care such as more visits to the clinical trial site, more treatments, etc.

What to Expect as a Participant

End of Clinical Trial

The research team may follow up with you to see how you are doing after ending your clinical trial participation. Once the trial concludes, you’ll meet with your research team to determine the next steps for care. Some studies allow continued use of the investigational treatment following the end of the study, others may not offer this option. You can request a copy of your medical information collected during your participation in the trial and communicate the study findings to your primary healthcare provider to see if there are any other trials for which you may qualify to help guide future care.

Pre-screen

You will talk to the research team to learn about the clinical trial and you will have the option to choose whether you are interested in learning more about the trial. Based off information you share about your medical history, the research team will determine if you’re a good candidate for the trial.

Consent

After talking to your study doctor and once you fully understand all the components of the study, you can agree to participate in the trial by signing an informed consent form which explains your rights and responsibilities. This will grant your study doctor access to your medical records so your eligibility for the study can be confirmed. Study procedures can only happen after the signing of the informed consent form.

Screening

The research team will review your medical history and complete a number of screening procedures to confirm you meet all requirements for joining the trial – these screening procedures can vary from trial to trial but could include a physical exam, blood tests, review of your medication, etc. Clinical studies each have different standards that outline who can participate in a trial – these are called “inclusion criteria”. Studies also have a set of factors that can prohibit certain individuals from participating in a trial – these are called “exclusion criteria”. The research team will inform you whether you meet the eligibility criteria for the trial.

Clinical Trial Participation

If you meet all eligibility criteria and qualify for the trial, your participation can begin. The research team will review your schedule of visits, what will be required at these visits, and anything you may need to do outside of the scheduled visits. The frequency of your visits may vary from trial to trial. Your overall health and safety is continually monitored during the entire length of the study. If you choose to do so, you can leave the trial at any time.

End of Clinical Trial

Consent

Screening

Clinical Trial Participation

End of Clinical Trial

Pre-screen

Consent

Screening

Clinical Trial Participation

End of Clinical Trial

What to Expect as a Caregiver

The support that caregivers provide to clinical trial participants is similar to the support they provide to any loved one with a medical condition. Caregivers may help with day-to-day details such as providing or coordinating transportation to study visits, taking care of household duties, and offering emotional support. It can also be helpful for caregivers to attend study visits with their loved one, ask questions to the research team, and take notes.

To support a loved one in a clinical trial, it may be helpful for a caregiver to review the informed consent document and speak with the research team to learn more about the details of the study. This can help with things like planning for study activities and knowing how to report any side effects that the participant may experience.

Please refer to the Resources section to learn more.

What to Expect When a Clinical Trial is Over

After a clinical trial is completed, the researchers will carefully analyze the information collected during the study. Researchers can then determine whether the results support the investigational treatment should continue to the next phase of development, stop treatment development, or possibly seek approval by the relevant authorities. After an investigational treatment has been proven to be safe and effective, it may be approved by the US Food and Drug Administration, European Medical Agency, or other health authorities; and become part of standard treatment for a disease.

Clinical trial results may be published in a peer-reviewed scientific or medical journal and made available to the public. Study findings of medical importance may also be shared by the media, patient advocacy groups, or at scientific meetings. If you have taken part in a trial, you can ask the research team if the study results have been or will be published.

About

New to Clinical Trials

Understanding Clinical Trials

At Acceleron we are dedicated to developing innovative, life-changing medicines for patients with a wide range of serious and rare diseases and are motivated by our commitment to bettering the lives of the patients we serve.

Phases of

Clinical Trials

Clinical Trial Phases

Pre-clinical

Phase I

Phase I

Why Participate in a Clinical Trial

Possible Benefits of Clinical Trials

Possible Risks of Clinical Trials

What to Expect as a Participant

Pre-screen

Pre-screen

Consent

Screening

Clinical Trial Participation

End of Clinical Trial

What to Expect as a Caregiver

What to Expect When a Clinical Trial is Over

For clinical trial side effects or product inquiry reporting, please contact and talk with your study doctor or study coordinator to follow the reporting requirements and timelines specified in the applicable clinical trial protocol.