Double Blind
Neither the patient nor the study doctor will know which treatment the patient is receiving.
Complete this brief questionnaire
Check eligibility
Key statement / question to grab the attention of the reader
Immune mediated thrombocytopenic purpura (iTTP), also known as acquired thrombotic thrombocytopenic purpura (aTTP), is a rare and serious blood disorder. The current rate of occurrence for TTP is about 3.7 cases per million people each year.
What is the purpose of the study?
The purpose of
this study is to evaluate the safety and effectiveness of whether an initial
treatment of (1) caplacizumab and (2) immunosuppressive drugs alone is as
effective as an initial treatment of (1) caplacizumab, (2) immunosuppressive
drugs and (3) TPE in preventing:
·
Worsening of iTTP episodes
·
Restoring normal levels of platelets and iTTP
related markers in the blood
·
Disease remission (no signs and symptoms of iTTP
and iTTP related blood markers are in the normal range)
How long is the studyA maximum of 24 weeks (about six months). Treatment is up to 12 weeks and will include treatment with the study medication (caplacizumab) until blood markers for iTTP are at a certain level of recovery for 2 consecutive weeks or until you have completed 12 weeks of treatment, whichever occurs first. Study follow-up will continue for an additional 12 weeks
How many study visits will there be?A maximum of 24 weeks (about six months). Treatment is up to 12 weeks with an additional 12-week follow-up. A maximum if 15 visits to the study clinic, following initial hospitalization
How many people are in the study?This study will enroll approximately 61 participants in sites across North America and Europe.
What to expect if you participate?
As a participant, expect rigorous health monitoring, access to potential innovative treatments, complete confidentiality, and consistent support throughout your journey.
3000+Participants
94%Clinical Trials Effectiveness
How long is the study?
Total -> Screening -> Treatment -> Follow-up
Study Information
Information about the study - length of time, number of visits, procedures completed during visits, chance of placebo vs. investigational medication
Study Information
Information about the study - length of time, number of visits, procedures completed during visits, chance of placebo vs. investigational medication
What is expected if I qualify and decide to participate?
- Review and sign the informed consent to participate.
- Attend all study visits and complete the study assessments.
- Complete an electronic diary daily.
- Hospitalized during the first part of your illness, where you will you receive the investigational medication intravenous (into the blood vessels) followed by daily subcutaneous injections (under the skin) of caplacizumab. Once your condition has stabilized, the study doctor will make a decision if it is safe to leave the hospital.
- To attend the scheduled visits and allow the tests or procedures to be performed as planned.
- Perform self-injections at home.
- Report immediately any side effects that you may experience, changes in medications or hospitalizations to your doctor / study staff.
- Report immediately any side effects that you may experience, changes in medications or hospitalizations to your doctor / study staff.
- Report immediately any side effects that you may experience, changes in medications or hospitalizations to your doctor / study staff.
- To receive the investigational medication or placebo under the skin (subcutaneously) using an injection.
- To inform the study doctor or study staff before stopping or modifying your medication(s) or before starting any new medications(s).
Can I participate?
What is expected if I qualify and decide to participate?
You will be expected to:
Attend scheduled study visits.
Adhere with daily investigational medication intake: You will be randomly assigned to receive either the investigational medication (2 out of 3 chance) or a placebo (1 out of 3 chance) that you will take by mouth twice daily (morning and evening around the same time each day), in addition to your current treatment for ALS.
Complete the tests or procedures to be performed as planned including assessments for the safety and tolerability of the investigational medication , pulmonary function tests, muscle strength, questionnaires related to your quality of life, as well as blood and urine sampling.
Travel to the study site. Patient reimbursement for travel, which may include parking, airfare, lodging and meals, may be provided for you and your caregiver.
Am I eligible?Attend scheduled study visits.
Adhere with daily investigational medication intake: You will be randomly assigned to receive either the investigational medication (2 out of 3 chance) or a placebo (1 out of 3 chance) that you will take by mouth twice daily (morning and evening around the same time each day), in addition to your current treatment for ALS.
Complete the tests or procedures to be performed as planned including assessments for the safety and tolerability of the investigational medication , pulmonary function tests, muscle strength, questionnaires related to your quality of life, as well as blood and urine sampling.
Travel to the study site. Patient reimbursement for travel, which may include parking, airfare, lodging and meals, may be provided for you and your caregiver.
Can I participate?
# countries
# sites
# participants
chance of placebo vs. investigational medication
# sites
# participants
chance of placebo vs. investigational medication
Terms to know:
Open Label
Both the patient and study doctor will know the treatment assignment, which is the investigational medication.
Placebo
A placebo is an inactive substance or treatment that looks the same and is given in the way as the investigational medication that is being studied.
Find out more about disease /condition
FAQ
What is a clinical research study?
Clinical research studies are used to show if/how an investigational medication works and to find out if it is safe. They can also be referred to as clinical trials.
Clinical studies are run by qualified healthcare professionals. The doctors and other healthcare providers are responsible for the study-related care of the people that participate (or enroll) in these studies. Clinical trials are a mandatory part of the process that leads to an approval, which is required before a drug can be marketed.
Independent committees (called Institutional Review Boards or IRBs) are made up of medical and non-medical people, who also watch over clinical research studies, to make sure that the people who enroll are properly and adequately informed and consent prior to study participation.
Clinical studies are run by qualified healthcare professionals. The doctors and other healthcare providers are responsible for the study-related care of the people that participate (or enroll) in these studies. Clinical trials are a mandatory part of the process that leads to an approval, which is required before a drug can be marketed.
Independent committees (called Institutional Review Boards or IRBs) are made up of medical and non-medical people, who also watch over clinical research studies, to make sure that the people who enroll are properly and adequately informed and consent prior to study participation.
Why are clinical trials conducted?
Clinical trials are essential to the development of new interventions (drugs, vaccines, procedures, devices) that help people to live longer and with less pain or disability.
They help researchers to understand what does and doesn’t work in humans that cannot be learned in the laboratory or in animals. Clinical trials also help doctors decide if the side effects of an investigational treatment are acceptable when weighed against the potential benefits.
They help researchers to understand what does and doesn’t work in humans that cannot be learned in the laboratory or in animals. Clinical trials also help doctors decide if the side effects of an investigational treatment are acceptable when weighed against the potential benefits.
How do clinical trials work?
Clinical trials must adhere to Good Clinical Practices (GCP), rigid standards that clearly state what is allowed by medical professionals during a clinical trial.
Clinical trials follow rigid testing procedures designed to help researchers determine the safety and effectiveness of the investigational drug or medical treatment being studied.
Clinical trials follow rigid testing procedures designed to help researchers determine the safety and effectiveness of the investigational drug or medical treatment being studied.
What is an investigational medication?
An investigational medication is what is being studied to see if symptoms improve while taking it. The drug is not yet approved for general use by regulatory authorities.
What is a placebo?
A placebo is an inactive substance or treatment that looks the same and is given in the way as the investigational medication/treatment that is being studied.
Why should I join a clinical research study?
Thousands of people volunteer to participate in clinical trials every year. The reasons for participation vary, but all of them help to advance medicine. Some reasons include:
- Medical supervision. Some people have a disease for which there is no approved treatment. They might join a clinical trial in the hopes of receiving an investigational medicine.
- Contribute to future treatments. Many participants wish to help future patients. This has been reported by both patients and healthy volunteers.
- Help improve the lives of others. Some people participate with the hope of helping others. The investigational drug or vaccine could benefit other patients and, in some cases, save lives.
Who can participate in a study?
Every clinical trial has a set of criteria that study participants must meet in order to participate, including agreeing to sign an informed consent form and HIPAA authorization.
What is an informed consent?
Before deciding to participate in a clinical study, potential participants will go through a process called "informed consent". This is designed to help the participant learn more about the clinical trial before deciding whether or not to participate.
Potential participants are given detailed information about the study. This includes the purpose, possible risks, potential benefits, how long it will take, what procedures are required and whom to contact with any questions or issues.
Once potential participants have no further questions, the document is signed indicating the decision to participate. An informed consent is designed to protect the study participant. It is not a contract. A participant can ask questions or stop participating without any reason and at any time, even before the study is over.
Potential participants are given detailed information about the study. This includes the purpose, possible risks, potential benefits, how long it will take, what procedures are required and whom to contact with any questions or issues.
Once potential participants have no further questions, the document is signed indicating the decision to participate. An informed consent is designed to protect the study participant. It is not a contract. A participant can ask questions or stop participating without any reason and at any time, even before the study is over.
What is a double-blind study?
Neither the study participant nor the study doctor will know which treatment the patient is receiving.
What is an open label study?
Both the study participant and study doctor will know the treatment assignment.
What is a Phase II (Phase 2) trial?
Researchers study how the investigational medication is tolerated in patients. Larger groups of patients (up to several hundred people) participate in Phase 2 clinical trials. The purpose of Phase 2 trials is to determine:
- Any short-term side effects
- The optimal dose of the investigational medication
- The timing of the investigational medication
In Phase 2 trials, one group of study participant may be given the investigational medication, while the other may be given a placebo or other approved treatment.
What is a Phase III (Phase 3) trial?
Researchers compare the investigational medication with the current standard treatment or placebo. They monitor any side effects very closely. Larger groups of patients (typically several hundred to thousands of people) participate in Phase 3 clinical trials. These studies can take many years to complete and are often completed in many different locations.
The purpose of a Phase 3 trial is to determine whether the investigational medication is safe and if it has a therapeutic (beneficial) effect.
In most cases, a study participant and study doctor will not know whether they have received the investigational medication, an existing treatment, or placebo. This helps minimize any bias.
The purpose of a Phase 3 trial is to determine whether the investigational medication is safe and if it has a therapeutic (beneficial) effect.
In most cases, a study participant and study doctor will not know whether they have received the investigational medication, an existing treatment, or placebo. This helps minimize any bias.
What type of study-related medical care is provided during the study?
All study-related medical care related to the study is provided, including study-related exams, study-related medications and other study-related medical care. There is no insurance required to participate. All study-related medical care will be explained.
Studies may also offer compensation in return for travel.
Studies may also offer compensation in return for travel.
Is it safe to participate in a clinical trial?
All clinical trials have possible risks, some of which are known before the study starts. Before deciding to participate, the study risks will be explained. Study participants are closely monitored during the study.
While side effects may occur with any investigational medication being studied, the safety and well-being of study participants are top priorities for the sponsors, doctors, nurses and research professionals who conduct clinical trials.
Study participants can decide at any point to end participation.
While side effects may occur with any investigational medication being studied, the safety and well-being of study participants are top priorities for the sponsors, doctors, nurses and research professionals who conduct clinical trials.
Study participants can decide at any point to end participation.
What about clinical trials participation during COVID-19?
This study requires in-person visits to the study site. Precautions are being taken to reduce the on-site risks of exposure to COVID-19. Each individual site location will review their protocols including mask wearing, social distance, pre-appointment check in. Study sites are medical facilities where COVID safety procedures are followed.
The COVID-19 vaccine can be administered before entering in the study or afterwards, without needing study drug interruption. This should be discussed with your study doctor.
In order to ensure treatment continuity and safety of patients participating in our clinical trials during this worldwide pandemic, several alternative solutions have been put in place and can be accessed if they are available in your area:
The COVID-19 vaccine can be administered before entering in the study or afterwards, without needing study drug interruption. This should be discussed with your study doctor.
In order to ensure treatment continuity and safety of patients participating in our clinical trials during this worldwide pandemic, several alternative solutions have been put in place and can be accessed if they are available in your area:
- Investigational study drug can be sent directly to you at home, to ensure that you will receive the investigational study drug even if you are unable to come to the research center/hospital/clinic
- Telehealth/video consultation can be organized instead of on-site visits
- Some study visits can be performed at home (home nursing)
- The possibility to sign your consent either on paper or electronically (eConsent)
Can I speak with someone if I have questions?
Yes, after you complete the online questionnaire and give permission to be contacted, a study representative will contact with you. You can ask questions at any time, before deciding to participate and during study conduct.
I speak Spanish, not English. Can I participate in the study?
Yes, if you let the doctor know, the study site can provide you with translated materials and may be able to provide an interpreter.
Am I eligible?
Can I participate?
Complete this brief questionnaire (# questions, length of time) to help determine if you may qualify to be referred to a site recruiting in your area for further evaluation for potential participation in this clinical study.
You can also add your contact information to be notified of future Sanofi studies.
You can also add your contact information to be notified of future Sanofi studies.
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References
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