1. Late-onset Observational Study (LOPOS)
Genzyme is currently enrolling a prospective observational study in approximately 60 patients with mild to intermediate late-onset Pompe disease. Details of this study are available on the website: clinicaltrials.gov, and there are sites in the United States and Europe. This 12-month study does not involve investigational treatment drug. However, it is anticipated that the data from this study will help design a future placebo-controlled clinical trial in late-onset Pompe patients.
This observational study (also called LOPOS study) will provide information on the clinical presentation of late-onset Pompe disease and the variability of the disease over time and between patients. This will be valuable in determining appropriate assessments and clinical outcomes for future studies.
A subset of patients involved in the observational study, who meet the inclusion criteria, are anticipated to be enrolled in a placebo-controlled, dose ranging clinical trial for late onset Pompe disease patients. This study is expected to begin in late 2004 and will be a twelve month study. Participation in the clinical trial is not guaranteed to all observational study participants nor is participation in the clinical trial required for participation in the observational study. Patients who do not participate in the observational study will likely not participate in the clinical trial.
Study sites and contact information will be posted at www.clinicaltrials.gov (search Pompe). Additional sites will be listed after they received ethics/ institutional (IRB) approval.
The criteria to participate:
The late-onset observational study includes patients eight-years-old and older in the US and Canada and 18 years and older in Europe, with mild to intermediate late-onset Pompe disease. For the purposes of this study, mild to intermediate late-onset Pompe disease is defined as people who are able to walk (may be with assistive device) and who do not require ventilator support while awake. One reason for the specific criteria is the importance of having several testable muscle groups. Participants have to perform multiple assessments, for example pulmonary and muscle testing, to help understand progression of the disease.
Detailed eligibility criteria are also listed on http://www.clinicaltrials.gov.
How to participate?
Your physician may contact a study site to learn more about participation in the study. Information regarding the LOPOS study is posted on Clinicaltrials.gov, and includes contact information for the sites.
Participation from other countries then the study sites
While there will be 5 study sites in 3 countries, patients from other countries may still be screened for inclusion in the observational study. As planned, it is a 12 month study with multiple assessments requiring travel. The principal investigator will need to determine that travel to and from the study site will not compromise the health and safety of the patient or the outcome of the study.
Genzyme will reimburse patients for travel within the North American continent but any travel or relocation over greater distances will be at the expense of the patient. At the European sites, the primary focus will be to screen patients within the site’s country.
All patients will need to verify that either their health insurance will cover them at the study site or that they will pay for their own medical costs (unrelated to the trial).
2. Late-Onset Expanded Access Program
Genzyme provides an expanded access program for late-onset Pompe disease. This program (LO-EAP) will be open to a very limited number of severely affected late-onset patients who will likely be ineligible for participation in the placebo-controlled late-onset clinical trial because of the advanced severity of their disease.
Regulatory requirements differ for the various countries and regions of the world, so the availability and timing of LO-EAP may vary based on geography. Because investigational enzyme for this program is very limited at this time, Genzyme will allocate investigational enzyme to 3 regions (US, Europe, and International Countries). Patients requesting LO-EAP will be evaluated for enrollment within their geographic region to determine whether they meet the inclusion criteria. In the US, expanded access to rhGAA will occur via a clinical trial where investigators will be required to collect data on EAP patients. In areas outside the US, investigators will be asked to collect data on EAP patients.
The medical enrollment criteria for the Late-onset Expanded Access program is posted on clinicaltrials.gov. The physician may contact Genzyme Medical Information, to get more information.
Eligibility is based primarily on meeting specific medical criteria as outlined in the clinical protocol and as found on clinicaltrials.gov. These LO-EAP eligibility criteria include, but are not limited to, dependence on a wheelchair and use of invasive ventilation. Additional considerations will include the patient’s and treating physician’s ability to comply with the program. Qualification for enrollment will be based on regulatory requirements and other factors particular to each region and may vary from region to region. If someone meets the eligibility criteria as well as any other requirements and considerations, they will be considered qualified for enrollment. Genzyme will work closely with the physician to determine qualification for enrollment in LO-EAP.
Genzyme has begun calling all physicians who have requested LO-EAP and has begun the qualification process. This process is expected to take several weeks, since the physician will be asked to return specific medical information to Genzyme.
Given the high likelihood of more patients qualifying in each region (US, EU, Int’l) than there is investigational enzyme available, there will be random enrollment of a limited number of patients.
If you are qualified, but not enrolled in LO-EAP at this time, you will be considered for future random enrollment as more investigational enzyme becomes available.
How to enroll/ refer for enrollment in an Expanded Access Program?
If you have already submitted a patient authorization form (US only, due to regulations) or your physician has already contacted Genzyme and requested consideration for Expanded Access, your physician will be contacted.
If you or your physician have not yet contacted Genzyme regarding Expanded Access please read the following:
US/Int’l: If you are interested in participating in an Expanded Access Program, you may contact Genzyme’s Medical Information department. The email address in the United States is email@example.com and the telephone number is 800-745-4447 (press 2).
EU: If you are interested in participating in an Expanded Access Program, please ask your physician to contact Genzyme’s Medical Information department to learn more about the Expanded Access Program. In Europe, the email address is firstname.lastname@example.org and the telephone number is 31-35-699-1499, or contact your local Genzyme office.
Genzyme will be happy to share the eligibility criteria with your treating physician to determine if you/ your child are eligible to participate in this program and to provide general information about the Expanded Access Program with your physician.
Do I have to pay to participate in the program? Will my insurance cover the cost of the program? Do I need insurance to participate in the program?
Genzyme will supply the investigational drug free of charge for the period in which patients are being treated under the expanded access protocol. Any other health care related costs are the responsibility of the patient.