Clinical Trials
“I would like to see a world where we have a cure for mitochondrial disease, where nobody needs support and one where we all go about worrying about something else.”
Find a Clinical Trial
Simplify your search for a relevant clinical study using the Mito Clinical Trials Finder. This tool provides easy-to-understand information on all the mitochondrial disease clinical trials and observational studies listed on clinicaltrials.gov and is regularly updated to reflect active research. Know of a study that needs added to the Mito Clinical Trials Finder? Contact info@umdf.org.
How Clinical Trials Work
Clinical trials are an essential step in the development of a new treatment. Here’s an overview of how they work.
Participants are Essential
Clinical trials can’t progress without participants because all new therapeutics must be tested in enough individuals to distinguish between a chance event and an outcome that can be reproduced.
Participants with a mitochondrial disease are essential partners in the process of researching and creating treatments for their disorder. While there are always inherent risks in trying a new treatment during a clinical trial, there are also benefits, including:
- New access to emerging treatments and experts in your disease
- The knowledge that you’ve taken action to help others with your disease
Participants are Protected
- Safety Measures are Implemented Before a Clinical Trial Enrolls its First Participant.
- New treatments are subjected to years of testing in the lab and with animal models to determine if they qualify for testing in clinical trials.
- To conduct a clinical trial, sponsors present an application to the FDA with data, a proposed study design, and any documents intended for participant use.
- Clinical trials cannot proceed until a thorough review is completed by the FDA and approval is granted.
Throughout a clinical trial’s duration, monitoring is continuous to ensure researchers adhere to the study’s protocols.
Participants with a mitochondrial disease are essential partners in the process of researching and creating treatments for their disorder. While there are always inherent risks in trying a new treatment during a clinical trial, there are also benefits, including:
- New access to emerging treatments and experts in your disease
- The knowledge that you’ve taken action to help others with your disease
Participants Have Rights
- Understandable information about the study
- Being treated with respect
- Withdraw from the study at any time
View the Patient Bill of Rights
The Decision to Participate is Yours
Helpful Tips When Speaking to Your Doctor about Clinical Trials
Write down any questions in advance.
Take a family member or friend along for support.
Write down the answers & record the conversation.
Questions to Ask Before Deciding to Participate
The Clinical Trial
- What is the purpose of the trial?
- Why do researchers believe that the treatment being studied may be better than the one being used now?
- Why may it not be better?
- How long will I be in the trial?
- What kind of tests and treatments are involved?
- How will the doctor know if the treatment is working?
- How will I be told about the trial’s results?
- How long do I have to make up my mind about joining this trial?
- Who can I speak with about questions I have during and after the trial?
- Who will oversee my care?
- Is there someone I can talk to who has been in the trial?
Risks and Benefits
- What are the possible side effects or risks of the new treatment?
- What are the possible benefits?
- How do the possible risks and benefits of this trial compare to those of the standard treatment?
Your Rights
- How will my health information be kept private?
- What happens if I decide to leave the trial?
Costs
- Will I have to pay for any of the treatments or tests?
- What costs will my health insurance cover?
- Who pays if I’m injured in the trial?
- Who can help answer any questions from my insurance company?
Daily Life
- How could the trial affect my daily life?
- How often will I have to come to the hospital or clinic?
- Will I have to stay in the hospital during the clinical trial? If so, how often and for how long?
- Will I have to travel long distances?
- Will I have check-ups after the trial?
There are Both Observational Studies and Clinical Trials
There are two types of clinical studies:
Clinical trials investigate a participant’s response to medical, surgical, or behavioral therapy. This research determines whether a particular therapy safely improves a patient’s quality of life.
Clinical Trials Follow a Defined Sequence Known as Phases
Before any new treatment reaches the market, it must be tested in a series of clinical trial phases. Each phase is designed to answer a specific critical question about patient outcomes.
Phase I
Learn more
Time to answer question: Months
Information gained: Therapy safety, side effects, appropriate dosing, and optimal delivery method such as by mouth or injection
Trials advancing to the next phase: 70%
Phase II
Researchers may answer these questions by comparing the outcomes of participants divided into two groups:
- A “control” group that receives the recommended treatment for disease or a placebo
- An “experimental” group that receives the new treatment
Learn more
People involved: 100s of people with the disease the therapy is intended to treat
Time to answer question: Months to years
Information gained: Safety for individuals with the target disease and insights for designing Phase III trials
Trials advancing to the next phase: 25-30%
People involved: 10-100 healthy “controls” or people without a mitochondrial disease
Time to answer question: Months
Information gained: Therapy safety, side effects, appropriate dosing, and optimal delivery method such as by mouth or injection
Trials advancing to the next phase: 70%
Phase III
Phase III trials prove whether a new treatment works better than current approaches by testing it in more people, over several years, and at many different medical centers. They always randomly assign participants into either a “control” or “experimental” group for comparison. If a Phase III trial shows that the new therapy offers a new benefit to patients and meets the standards set by regulatory authorities, it can be considered for approval.
Learn more
Time to answer question: Several years
Information gained: Proof of the safety and outcomes for individuals with the target disease
Trials advancing to regulatory approval: 33%
Phase IV
The conditions under which a treatment is normally administered may introduce variables that were not present in this setting but may affect patient outcomes. Phase IV clinical trials collect data to capture any new effects, reveal any long-term side effects, and may detect any potential for the new therapy to treat other diseases.
Learn more
Time to answer question: Several years
Information gained: Any effects that may not have been present in the clinical setting
Trial Proceeds to Completion
In general, volunteer participants cannot continue the treatment studied in a failed clinical trial.
Learn more
However, exceptions can be made under the compassionate use and expanded access programs established by the FDA if the following criteria are met:
- The sponsor allows and can provide continued access to the treatment.
- The new treatment clearly improves outcomes for the individual taking it.
- The trial failed due to reasons other than safety.
- The disease being treated threatens the participant’s life.
- No alternative treatments exist.
Even though failure isn’t the result anyone hopes for, the data collected can be used to inform future studies and makes the research valuable.
Therapeutic Development Pipeline
All new treatments or therapeutics proceed through a defined development path. This path begins with preclinical development and progresses through clinical trials all the way to FDA-approval before ending in patients’ hands.
Several treatments for mitochondrial diseases are already making their way along this path. Soon mito patients and their families will be able to see the treatment approaches being developed, where they are in the development pipeline, and what mitochondrial diseases they target.
Stay tuned for the reveal of the full pipeline!
Conditions in the Pipeline
- Barth Syndrome
- Inherited Mitochondrial Disease
- KSS-CPEO
- Leigh Syndrome
- LHON
- MELAS
- MERRF
- Mitochondrial Myopathies
- MIDD
- Pearson Syndrome
- PDCD
- Seizures
- TK2 Deficiency
Therapeutic Development Pipeline
All new treatments or therapeutics proceed through a defined development path. This path begins with preclinical development and progresses through clinical trials all the way to FDA-approval before ending in patients’ hands.
Several treatments for mitochondrial diseases are already making their way along this path. Now mito patients and their families can see the treatment approaches being developed, where they are in the development pipeline, and what mitochondrial diseases they target.
Stay tuned for the reveal of the full pipeline!
Conditions in the Pipeline
- Barth Syndrome
- KSS-CPEO
- Leigh Syndrome
- LHON
- MELAS
- MERRF
- Mitochondrial Myopathies
- MIDD
- Pearson Syndrome
- PDCD
- Seizures
- TK2 Deficiency
Glossary of Clinical Trial Terms
Adverse outcome/adverse event
Compassionate use
Crossover
Dirty drug
Double blind
Efficacy
Endpoint
Primary: The main result or endpoint measured at the end of a clinical trial to determine whether or not a new experimental treatment works.
Secondary: Results that were obtained during a trial that can be analyzed but are not necessarily controlled for.
Exclusion criteria
Inclusion criteria
Informed consent
Institutional Review Board (IRB)
Off label
Open label/open label extension
Outcome measures
Phases
Placebo
Placebo-controlled
Primary outcome
Principal investigator (PI)
Protocol
Randomized controlled trial
Recruitment status
Registry
Secondary outcome
Sponsor
Washout period
* This glossary was originally published on Rett Research Ready™ and appears here with permission from the International Rett Syndrome Foundation (IRSF).