Finding Clinical Trials

For up-to-date information about Pompe disease research studies throughout the world that are actively recruiting volunteer patients, visit www.clinicaltrials.gov, the US government clinical trial Website run by the National Library of Medicine (NLM). Enter “Pompe” in the search area. You can also call NLM at 1-888-346-3656 (toll free in the United States) or 1-301-594-5983 (from outside the United States).

You may also contact the Medical Information department at Genzyme if you have questions about enrolling in clinical trials of ERT for Pompe disease. The contact information for Genzyme services is listed below. The Pompe Patient Program offers more information about ERT clinical trials.

Learn more about Genzyme’s Pompe Program >

Genzyme Services

As a partner in the effort to manage the challenges of living with Pompe disease, Genzyme offers a wide range of support services to patients and their families.

1-800-745-4447 (toll free in the United States) 1-617-768-9000

Monday–Friday, 8 am to 6 pm, US Eastern Time

Drug Development Process

The process of taking a new drug from discovery to manufacturing and finally to approved use in humans is long, costly, and complex. In the United States, new drugs can only be approved after going through a comprehensive review by the Food and Drug Administration (FDA) to confirm their safety and effectiveness. On average, it takes 12 to 15 years for an investigational treatment to pass through the drug development process, obtain marketing approval, and reach patients. To learn more about this process, click the links provided below.

Clinical Trial Process >

Manufacturing Process >

About Clinical Trials

Very few drugs meet the FDA's rigorous standards to be tested in humans. In fact, an estimated five out of 5,000 drugs that enter preclinical testing make it to clinical trials.

A clinical trial is a research study that is performed in humans to determine if a new drug or therapy is both safe and effective for treating a disease or condition. Clinical trials leading up to drug approval are usually staged in multiple phases, each with a different purpose and a different guideline for the number of patient volunteers that should be included.

Patient volunteers must meet the criteria for enrollment in the trial, which can include factors such as age, gender, and the type or stage of the disease. In some cases, each phase of study may involve multiple trials.

Clinical Trials in Rare Diseases

In testing drugs for rare or “orphan” disorders, the clinical trial process may be different. Because there are so few people with the disease, the number of patients per trial may be smaller. Despite this difference, the same standards apply for demonstrating safety and effectiveness.

Steps in the Traditional Clinical Trial Approval Process

Preclinical

These tests are intended to evaluate the drug's safety and potential efficacy in animal models prior to being tested in humans.

Investigational New Drug Application (IND)

An Investigational New Drug Application (IND) must be filed with the FDA in order to test an investigational product in humans.

Phase 1

Phase 1 typically involves fewer than 100 patient or healthy volunteers. Initial human trials are designed to determine the safety of the drug and to note side effects.

Phase 2

Phase 2 typically involves up to several hundred patient volunteers. Secondary trials are designed to evaluate the effectiveness of the drug and further study its safety.

Phase 3

Phase 3 typically involves several hundred to several thousand patient volunteers. Often the final phase, these trials seek to confirm the effectiveness of the drug, monitor adverse reactions, and gather information as to how to safely use the drug.

New Drug Application (NDA)/ Biologics License Application (BLA)

A New Drug Application (NDA) or Biologics License Application (BLA) is filed with the FDA. The applications contain all the relevant scientific information from the clinical trials needed to evaluate the drug for approval.

Note: If the drug is approved by the FDA, there may also be Phase 4 trials after the drug becomes available in order to collect additional information on risks, benefits, and optimal use.

Clinical trials may be sponsored by a variety of organizations, including:

Pharmaceutical, biotechnology, or medical device manufacturers
Medical institutions
The National Institutes of Health (NIH)
Foundations
Voluntary groups

Information about many upcoming trials that are actively recruiting patient volunteers can be found by visiting the NIH online database of clinical trials: www.clinicaltrials.gov.

Studies listed are conducted in the United States and in more than 100 countries. It also provides information to help you understand the clinical trial process, including considerations for potential participation.

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