Extended BMN 701 treatment in patients with late-onset Pompe disease

• Research type

  Research Study

• Full title

  A Long-Term Study for Extended BMN 701 Treatment of Patients with Pompe Disease who have participated in a BMN 701 study

• IRAS ID

  103018

• Contact name

  Derralynn Hughes

• Sponsor organisation

  BioMarin Pharmaceutical Inc.

• Eudract number

  2011-001805-28

• Research summary

  EXTENDED BMN 701 TREATMENT IN PATIENTS WITH LATE ONSET POMPE DISEASE This is a phase 2 research study that is being conducted to learn more about an experimental treatment (BMN 701) for people with Pompe disease after they complete an earlier research study with BMN 701. BMN 701 is intended to help replace one of the enzymes (GAA) that there isn??t enough of in the body of Pompe disease patients. The main purpose of this study is to test the safety and effects of up to 5 years of treatment of BMN 701 at the same or lower dose than the one the participants were receiving in the last study. The study will see if BMN 701 antibodies are being produced. It will also look at how the medication is absorbed, distributed in the body, how the chemical components change forms and how it is broken-down and gotten rid of by the body. Another purpose of the study is to learn whether replacing the missing enzyme in this way can help improve, slow or stop the disease. The study will also look at potentially good and bad effects the study medication may have. This study will take place at approximately 15 hospitals in Europe, USA, Australia and Taiwan. About 30-45 patients will take part. In the UK the study will be conducted at NHS Hospitals. The participants will undergo screening tests. During treatment, the participant will return to the hospital every 2 weeks for up to 24 weeks (a treatment cycle). Treatment will be repeated in 24 week cycles if it is safe to do so, for up to 5 years. There will also be a final follow-up visit about 1 month the last infusion. The study visits will last about 6 - 16 hours depending on what tests and procedures are being done. Participants will undergo a series of tests including blood and urine tests, physical examinations, muscle tests, X-rays and ECGs.

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  12/NW/0321

• Date of REC Opinion

  3 Jul 2012

• REC opinion

  Further Information Favourable Opinion