The PMD Phase I safety study has completed it's enrollment and no more applicants will be accepted.
- How many children will take part in this study?
- Four male children with the “connatal” (most severe) form of PMD will be enrolled in this study. This study is not open to patients with less severe forms of PMD. The study is only open to United States or Canadian citizens living in North America.
- Who are the different doctors that will be involved in the study and my child’s care?
- UCSF doctors with many different specialties will be involved in this study. They are available for any questions or concerns you may have. The team of doctors involved will be Dr Jonathan Strober from Neurology, Dr. Nalin Gupta from Neurosurgery, and Dr. David Rowitch from Pediatrics.
- What are the costs of taking part in the study?
- All procedures required by the study will be paid for by the study sponsor, StemCells, Inc., at no cost to you. Travel, lodging and meal expenses associated with participation in the study while at UCSF Children’s Hospital will also be covered. However, costs of your child’s routine medical care will be your responsibility.
- Are there benefits to taking part in this study?
- There may be no benefit to your child from participating in this study. If there is a benefit, it is uncertain when the effect may occur and how long it may last. However, this study may provide important information to help doctors learn more about the transplantation of HuCNS-SC® cells and it is hoped that this information will help in the treatment of future patients with PMD.
- Where will we stay while in San Francisco?
- If your child is screened and eligible to be evaluated at UCSF for study, a reservation for hotel accommodations will be made for your family near the hospital. If your child is enrolled in the study and undergoes the stem cell transplant, your stay during the time of the operation will be one month long. You will stay at a local hospital housing facility called “Family House” which is very close to the hospital. When you return to UCSF for your child’s follow-up visits your family will again stay at the Family House.
- How will our travel to San Francisco and lodging be arranged?
- You will not be required to make your own travel arrangements. Plane reservations, airport transfers and lodging will be arranged through a travel agent. The study nurses will work with your family to coordinate your travel plans.
- How long will my child be in the study?
- The time commitment for this study is approximately thirteen months. Your child will take a drug to suppress his/her immune system, an antibiotic, and other medications before, during and after the transplantation surgery for up to nine months after the surgery. The follow-up visits to UCSF after the surgery will continue for eleven additional months.
- What are the side effects or risks I can expect for my child from being in this study?
- The doctors will be closely monitoring your child for side effects. All medications and treatments have side effects and, with an investigational product – HuCNS-SC® cells, these can sometimes not be predicted. The primary purpose of this study is safety. You will be given a consent form to read that will explain all of the possible side effects your child may experience.
- What happens to my child when the follow-up studies are completed at one year post-transplant?
- At the completion of the study, you will be asked to have your child participate in a separate long-term follow-up study. It may require annual examinations by your study doctors for an additional four years. Keeping in touch with you and checking on your child’s condition every year will help us look at the long-term effects of the study.
- How can I get more information about the study?
If you are interested obtaining more information on this trial please call us at 1-800-793-3887 to speak to our clinical trial coordinator or email PMD@ucsfmedicalcenter.org.
We also recommend that you download our Pelizaeus-Merzbacher Disease (PMD) Brochure [PDF 4.7MB]