Clinical Trials

Clinical trials test drug candidates for safety and effectiveness.

Clinical Trial Basics 

A clinical trial, also called an interventional study, tests new drugs and treatments. It can also test new applications for approved drugs or treatments (for example, using drugs in different combinations or for different diseases). This is different from an observational study, which records data about participants according to a predetermined schedule and process, but offers no drugs or treatments.

The process can be long, complicated, and difficult. On average, only 10% of drugs in clinical development result in an FDA-approved drug. These unique challenges require a thoughtful strategy.

Cure SMA’s Approach

Our Funding Strategy

One of the most distinctive aspects of our research program is our seed funding strategy. We fund the early stages of a drug project, with the goal of attracting increased funding from government and pharmaceutical companies as the process moves forward.

Cure SMA has invested over $20 million for early stage drug development, with another $6.5M to conduct and prepare for clinical trials. Now, because of our successes, government and industry fund the majority of SMA clinical trials.

This has freed us to focus more of our investments on basic research and drug discovery, thus increasing the number and diversity of drug candidates being prepared for clinical trials. The funding risk is shared, and the diversity of candidates minimizes the impact if a drug fails.

Our Clinical Trial Goals

Cure SMA previously funded a network that conducted five clinical trials on repurposed drugs. Today, the experience of those trial sites, and the drug testing protocols developed, are applied to novel drugs rather than repurposed drugs.

Using our connections in the family, medical, research, and industry communities, we continue our collaborative efforts to improve the efficiency of clinical trials. Current goals include:

  • Validate protocols for clinical assessment of every SMA type.
  • Implement clinical trial sites across the U.S.
  • Collaborate with the broader SMA community, including organizing a precompetitive group of industry partners to work on key aspects of clinical trial readiness.

We also continue to earmark a portion of our research funding for clinical trials, so that we can opportunistically invest where our dollars will be most effective. 

How Clinical Trials Work

The following is a summary of the clinical trial process. For a more detailed description, download our care series booklet, Learning About Clinical Trials.

Clinical Trial Phases

A drug must pass each individual phase of a clinical trial before advancing. The number of participants in each phase can vary. The numbers given below are typical of trials for an orphan disease such as SMA.

  • Phase 1 tests safety and dosage levels, usually on 10-20 individuals. Sometimes, these volunteers are healthy individuals. However, for an orphan disease like SMA, some or all may be patients with the condition being studied.
  • Phase 2 tests a slightly larger group, usually 20-40 individuals, all of whom have the condition being studied. 
  • Phase 3 increases the number of people tested—up to 100-200 including the control or placebo group. A drug that passes these three phases can be approved and marketed to the general public. 
  • Phase 4 primarily involves ongoing evaluation and monitoring, even after a drug is approved for the general public.

The First Steps

Researchers test new therapies in the laboratory and in animal studies, and those with the most promising results are moved into clinical trials.

Funding for clinical trials can come from physicians, medical institutions, foundations, voluntary groups, pharmaceutical companies, or federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran's Affairs (VA).

Regulating Clinical Trials

The clinical trial process is regulated in a number of ways, both before it begins and while it’s in progress.

  • FDA Approval: The Food and Drug Administration (FDA) must approve the drugs used in clinical trials, so that they can be distributed across state lines (clinical trials are usually conducted in multiple cities and states to ensure the most comprehensive and most accurate results). To get FDA approval, clinical trial sponsors submit an Investigational New Drug (IND) application to the FDA. Once approved, the IND allows them to distribute the drug for the duration of the trial.
  • IRB Review: Clinical trials must also be approved by an institutional review board (IRB). This independent committee ensures that the trial is conducted ethically, that the potential benefits justify any risks, and that the rights of participants are protected. The IRB continues to review the trial as it progresses.
  • Informed Consent: Trial participants must receive all key facts about a trial before it starts and sign an informed consent document. Informed consent is not a contract, and the participant can still withdraw from the trial.

Clinical Trial Protocols

A protocol is a study plan for a clinical trial. It covers important details like:

  • Who can participate. These rules, called "inclusion and exclusion criteria," typically include age, stage or type of disease, previous treatment history, and other medical conditions.
  • What procedures, medications, and dosages will be given, and how often.
  • When the participants must see a study doctor, and what tests or measurements the doctor will use to evaluate them.
  • How the control group (if any) will be set up. Participants assigned to a control group will receive a placebo—an inactive pill, liquid, or powder—or a standard treatment. The other group is given the experimental treatment. Participants are often not told which group they are in, and the researchers may not know either. This "double-blind" format keeps participant or researcher bias from affecting the results.

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