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Frequently Asked Questions

Frequently Asked Questions About Clinical Trials

What Is a Clinical Trial?

It is important to understand that clinical studies are experiments. It typically involves conducting research on human volunteers/participants with the intent to determine if a specific drug or therapy is effective.  The two main types of clinical trials are: a) interventional studies, and b) observational studies.

In a clinical trial, participants receive specific interventions, such as a drug, according to the protocol created by the investigators that guides the trial. The intent is to determine the safety and efficacy of the intervention/observation by monitoring/measuring certain outcomes in the participants.

Clinical trials are sometimes described by phases as described below:

  • Phase 1: The goal in a phase I trial is to determine whether the drug is safe and to find out the effective dose.
  • Phase 2: In Phase 2, the drug is given to a large group of people to see if it is effective and to further evaluate its safety.
  • Phase 3: The drug is given to larger groups of people to confirm its effectiveness, monitor side effects, compare the effects of newer drugs with current treatments, and collect information that will allow the drug or treatment to be used safely.
  • Phase 4: Studies are done after the drug or treatment has been approved and marketed to gather information on the drug’s effect in various populations and any side effects associated with long-term use.

Who Sponsors/Conducts a Clinical Trial?

A clinical trial is typically sponsored or funded by a pharmaceutical company or an institution such as a medical research institution, the National Institutes of Health (NIH) or the European Commission (EC).

A clinical trial is typically conducted by a Principal Investigator (PI) along with a research team who assists with the study. The research team includes doctors, nurses and other health care professionals.

Who Can Participate in a Clinical Trial?

The protocol, which guides the study, lists the eligibility requirements to participate in the study. These are listed under the inclusion criteria and exclusion criteria. Examples of eligibility requirements would typically include age of the participant, stage of disease, previous treatment and other medical conditions.

To get the latest information on the protocol and the eligibility requirements for current trials, follow the link below.


What is Informed Consent (IC)?

IC is the process in which researchers provide interested participants with information about the clinical study including the risks and potential benefits of the clinical study. The idea is to provide as much information as needed in order for the participant to decide whether he or she wants to enroll into the clinical trial. Note that providing consent is not a contract. A participant can withdraw from the study at any time.

Additional Questions to Ask/Consider

It is important to learn as much as possible about any clinical trial so that you can make an informed decision on whether to participate or not. Below is a list of questions to consider when doing research or to ask the research team about the study.

  • What is being studied?
  • Why do researchers believe the intervention being tested might be effective? In other words, what are the primary and secondary outcomes?
  • How do the possible risks, side effects, and benefits of this trial compare with those of my current treatment?
  • What will I be required to do if I decide to participate?
  • What tests and procedures are involved?
  • How often will I have to visit the hospital or clinic?
  • Will hospitalization be required?
  • How long will the study last?
  • Who will pay for my participation/treatment?
  • Will I be reimbursed for other expenses?
  • What type of long-term follow-up care is part of this trial?
  • If I benefit from the intervention, will I be allowed to continue receiving it after the trial ends? If so, who will pay for it, what will they pay for just the drug or the delivery as well and for how long?
  • Will results of the study be provided to me?
  • Who will oversee my medical care while I am participating in the trial?
  • What kind of prior research has been done to prove the efficacy of the drug?
  • Has this drug been used previously to treat other diseases?  For example, prior to its approval to treat NPC, Zavesca had been approved for Gaucher disease?
  • What are the expected primary and secondary outcomes? e.g. improved swallowing, better gait, increase in life expectancy?
  • What is the dosage?
  • What are the side effects?
  • How will the drug be administered?
  • Are the risks of using this drug known? Are the potential risks understood?
  • Is the proposed treatment part of a formal study (clinical trial)? (Note: please check to determine if it is a formal clinical trial.)
  • Has a plan for follow-up monitoring been defined for participants after the treatment?
  • At which locations will the study be conducted?
  • What is the current phase of the clinical trial? (Note: The further along the clinical trial, the more information is available about dosage and efficacy.)
  • What assurance will I have that the trial results will be published promptly, regardless of trial outcome?
  • Is the involved pharmaceutical company/medical research institution experienced in running a clinical trial?
  • Has the treatment been studied in several different animal models, and especially in one that is thought to mirror how it will act in humans?

This is an exciting and enviable time for the NPC community with several clinical trials currently being discussed. It can be a confusing time too as families try to decipher all of the available information to understand what the trials are about and whether their loved ones a) are eligible, b) whether they want to be involved and c) whether it will be available in their country/location.

Below is a document that is divided into two sections; the section “Clinical Trials” explains what a clinical trial is and provides a list of potential questions to ask as part of your research into the trials, and the section “Clinical Trials Comparison” provides a table comparing the current trials in Niemann Pick Type C.

Current Clinical Trial Comparisons Developed by Parent/Patient NPC Trial Recruitment Team


The “51 and Done” is a committee composed strictly of parents dedicated to providing information and knowledge to the Niemann Pick Type C community on ALL upcoming and future clinical trials. The intent is to ensure parents have objective and relevant information and knowledge to help them decide on the path to take for their loved ones affected by NPC. Please note that the “51 and Done” committee does not engage in the practice of medicine or claim to have medical knowledge. The main purpose of this committee is to provide information and knowledge from publicly available sources.

All material contained in above is for educational and informative purposes only.  It is not intended to substitute medical or professional services nor replace the relationship that exists between patients, their physicians and other health care providers. No action, treatment, independent entities or persons are endorsed and all information is provided without warranty.  Any questions should be referred to your physicians and in particular, any changes in treatment or care should be made in conjunction with your medical team.