Clinical trials

How does a clinical trial work?

Clinical trials operate in at least three phases and are mandated by strict government guidelines. The purpose of a clinical trial is to determine which recently developed methods are the safest and most effective for treating individuals with cerebral palsy. Clinical trials are aimed at gathering as much research and results as possible to improve the various ways that CP is diagnosed, treated, and prevented.

Clinical trials for cerebral palsy typically involve:

• Testing the effectiveness of a recently developed therapy method
• Testing the effectiveness of a diagnostic procedure or device
• Testing a new treatment method for a particular type of CP
• Conducting further research on the potential causes of CP

In order to ensure patients’ safety, clinical trials follow very strict medical standards. This includes a series of 3-4 phases. The purpose of these phases is to gradually establish the risks and benefits associated with various methods by utilizing different groups of participants over time.

Benefits of participating in a clinical trial

There are many advantages to engaging in a clinical trial for cerebral palsy.

Additional potential benefits of participating in a clinical trial include:

• The opportunity to have health closely monitored by a research team of doctors and medical professionals
• Gaining a more complex understanding of cerebral palsy and the various forms of treatment, therapy, and prevention
• Assisting the overall population affected by CP, as the results of a clinical trial can help generate an entirely new segment of treatment methods

While there are benefits associated with clinical trials, there are also risks that should be taken into account. As a result of various tests, health risks for a clinical trial range from temporarily unpleasant side effects to potentially life-threatening conditions.

The process of a clinical trial

There are at least three phases that clinical trial participants may be asked to take part in. The fourth phase is reserved for future long-term testing if the trial method is approved by the FDA.

Phase I

The first phase typically consists of a test on a small group of people, usually 20-100 participants, to determine if the method is safe and to identify any immediate side effects. It will also test dose-ranging, where various doses are administered to establish which is the least harmful while still producing the best results.

Phase II

The objective of the second phase is to determine whether the method being tested is better than the existing treatment procedure. Groups of participants usually consist of around 100-300 people. Researchers will be looking to find out if patients are responding to the treatment method and if the same results can be replicated in other patients.

Phase III

The third phase of a clinical trial utilizes a larger group of patients, generally around 1,000-2,000 participants. Some patients will be administered the treatment method while others will receive a placebo instead. Using a placebo serves as a control group against which doctors can use to measure the true therapeutic effects of a specific trial.

Phase IV

Once a treatment method has proven to be safe and effective, it is submitted for approval by the FDA. After a trial method receives FDA approval, it will be available to the public through primary care physicians. Researchers will continue to test the effectiveness and safety of the method being administered to the public for any long-term side effects or complications.

Qualifications for clinical trials

Individuals are able to receive information about clinical trials for CP from their doctor. At that time, various aspects of an individual’s identity and condition will be taken into account to determine eligibility.

Each clinical trial is unique, but there are n.d.a few characteristics that will be evaluated for every clinical trial. These include:

• Age — Certain clinical trials seek out individuals of a specific age group for research purposes, which may limit a patient’s participation.
• Gender — There are different hormones present in both men and women. Certain clinical trials may be aiming to test the effects a method has on both genders individually.
• Current status of health — Some clinical trials have a preference regarding current health, with some preferring individuals only in optimal health. Others may not be as selective about past or present health conditions.
• The type or severity of CP — Clinical trials may aim to test the effects of various methods on specific types or stages of cerebral palsy. Others are less selective and can be tested on any form of CP.

Participants in a clinical trial will be considered based on the above qualifications to determine eligibility. Clinical trials vary in many ways. If an individual with CP does not qualify for one specific trial, there are many other options available that cover a range of demographics and health conditions.

Accepted participants will be asked to sign a waiver agreeing to take part in the trial. All participants are able to leave a clinical trial at any point if they decide they would no longer like to continue.

Current clinical trials recruiting individuals affected by CP

There are many current options available if you are interested in participating in a clinical trial for individuals with cerebral palsy. Various types of CP are consistently being tested and researched by doctors to generate the most beneficial methods of CP prevention and treatment.

Current types of clinical trials being conducted for individuals with CP are:

Cord blood stem cell therapy

Efforts are underway to enroll children between the ages of 1 and 12 to participate in cord blood stem cell transplants. All levels and severities of cerebral palsy are accepted. This FDA-regulated clinical trial is being conducted to evaluate the effectiveness of using cord blood stem cell infusion to treat cerebral palsy in children.

Autologous stem cell transplants are the safest form of stem cell therapy. “Autologous” refers to the transplant being taken from the same individual, so the recipient of the transplant is also the stem cell donor. As a result, the stem cell transplant carries virtually no threat of being rejected by the immune system. This will be the primary method used in stem cell therapy testing.

Bone marrow transplants

While bone marrow stem cell transplant is already recognized as an effective therapy for CP, researchers are now testing the effects of performing multiple bone marrow transplants over an extended period of time. Doctors will evaluate the safety and effectiveness of performing multiple procedures of deriving stem cells from bone marrow to replace any faulty bone marrow that currently exists.

Children between the ages of 4 and 12 years old are asked to participate in testing that includes undergoing multiple bone marrow transplants. If no allergic reactions or abnormal symptoms are present following the first transplant, patients will be asked to return in 6 months to receive a second injection of bone marrow.

Testing educational programs for caregivers of children with CP

Researchers are now testing the effects of incorporating parents or caregivers into physical therapy sessions as a way to improve overall mobility in children. The objective of this clinical trial is to determine if incorporating caregivers into physical therapy sessions can have a positive effect on a child’s development and functioning.

The parent’s programs will generally be held one day a week for 12 weeks. Meetings will be oriented around motivating your child to complete everyday activities, such as bathing, dressing, or eating. Parents will also be given a diary to record the child’s performance of activities in the home to see how caregivers actively encouraged independence. The child’s performance in daily activities and overall mobility will be tested at the end of the 12 weeks.

Finding a clinical trial for cerebral palsy

Participating in a clinical trial is often a great option for people who feel they have exhausted the current treatment methods available and are looking for an alternative that offers the potential for more favorable results.

There are a number of clinical trials being performed for individuals with CP in addition to the ones listed above. If you are interested in taking part in a clinical trial, talk to your doctor to find one that is the right fit for you or your child.

For more information on CP treatment, therapy, and clinical trials, download our free Cerebral Palsy Guide here.