A type of gene silencing treatment in which specially designed DNA molecules are used to switch off a gene
You do not know if you carry the genetic mutation for HD gene
Brain-derived neurotrophic factor: a growth factor that may be able to protect neurons in HD.
A biomarker, or biological marker is a measurable indicator of some biological state or condition. It can be a level of something in your blood or hormon levels for instance. Biomarkers are often measured and evaluated to examine normal biological processes – like measuring the level of hemoglobin in your blood or your blood pressure. In reference to research on Huntingtons disease, a biomarker is an indicator of disease progression. Examples are changes within the cells related to so-called metabolism (energy processes) or levels of mutant Huntingtin in nervecells. It can also be changes in organs – like loss of muscle or brain volume. We use biomarkers to measure how the disease progress and potentially how a treatment influence the disease progression.
Direct stimulation of the brain using electrical impulses through tiny wires
The stretch of DNA at the beginning of the HD gene, which contains the sequence CAG repeated many times, and is abnormally long in people who will develop HD
The word can have different meaning in different context but here it is someone who is not from a HD family
A clear fluid produced by the brain, which surrounds and supports the brain and spinal cord.
A research study involving human volunteers (also called participants) that is intended to add to medical knowledge. There are two types of clinical studies: interventional studies (also called clinical trials) and observational studies.
Medical research involving people.
An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
A measure of whether a treatment works or not.
The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.
A type of eligibility criteria. These are reasons that a person is not allowed to participate in a clinical study.
A type of eligibility criteria. These are the reasons that a person is allowed to participate in a clinical study.
A process used by researchers to communicate to potential and enrolled participants the risks and potential benefits of participating in a clinical study.
A study in which an experimental drug or treatment is given to volunteers.
A type of clinical study in which participants are assigned to groups that receive one or more intervention/treatment (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. The assignments are determined by the study’s protocol. Participants may receive diagnostic, therapeutic, or other types of interventions.
A clinical trial design strategy in which one or more parties involved in the trial, such as the investigator or participants, do not know which participants have been assigned which interventions. Types of masking include: open label, single blind masking, and double-blind masking.
Mutant huntingtin protein. The protein produced by the faulty HD gene.
A study in which measurements are made in human volunteers but no experimental drug or treatment is given.
A type of clinical study in which participants are identified as belonging to study groups and are assessed for biomedical or health outcomes. Participants may receive diagnostic, therapeutic, or other types of interventions, but the investigator does not assign participants to a specific interventions/treatment.
A patient registry is a type of observational study.
Positron emission tomography which produces detailed 3-dimensional images of the inside of the body.
The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study’s objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
An inactive substance or treatment that looks the same as, and is given in the same way as, an active drug or intervention/treatment being studied.
The placebo effect is a psychological effect that causes people to feel better even if they’re taking a pill that doesn’t work.
Liquid component of the blood.
Prior to onset or diagnosis of movement symptoms.
The main reason for the clinical trial. The types of primary purpose are: treatment, prevention, diagnostic, supportive care, screening, health services research, basic science, and other.
The person who is responsible for the scientific and technical direction of the entire clinical study.
Protein builds, maintains, and replaces the tissues in your body. The building blocks of life.
The written description of a clinical study. It includes the study’s objectives, design, and methods. It may also include relevant scientific background and statistical information.
A type of allocation strategy in which participants are assigned to the arms of a clinical trial by chance.
- Not yet recruiting: The study has not started recruiting participants.
- Recruiting: The study is currently recruiting participants.
- Enrolling by invitation: The study is selecting its participants from a population, or group of people, decided on by the researchers in advance. These studies are not open to everyone who meets the eligibility criteria but only to people in that particular population, who are specifically invited to participate.
- Active, not recruiting: The study is ongoing, and participants are receiving an intervention or being examined, but potential participants are not currently being recruited or enrolled.
- Suspended: The study has stopped early but may start again.
- Terminated: The study has stopped early and will not start again. Participants are no longer being examined or treated.
- Completed: The study has ended normally, and participants are no longer being examined or treated (that is, the last participant’s last visit has occurred).
- Withdrawn: The study stopped early, before enrolling its first participant.
The chemical, similar to DNA, that makes up the ‘message’ molecules that cells use as working copies of genes, when manufacturing proteins.
The organization or person who initiates the study and who has authority and control over the study.
The acronym or initials used to identify a clinical study (not all studies have one). For example, the title acronym for the Women’s Health Initiative is “WHI.”
The name of ASO drug developed by Ionis/Roche.
A figure estimating how many people there are in a particular population who have a certain medical condition.