SHP-GCB-402-effect of Velaglucerase alfa on Bones in Gaucher Disease

• Research type

  Research Study

• Full title

  An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment with Velaglucerase alfa on Bone-related Pathology in Treatment-naïve Patients with Type 1 Gaucher Disease

• IRAS ID

  191038

• Contact name

  Derralynn Hughes

• Contact email

  rmgvdah@ucl.ac.uk

• Sponsor organisation

  Shire Human Genetic Therapies, Inc.

• Eudract number

  2015-001578-17

• Duration of Study in the UK

  3 years, 4 months, 22 days

• Research summary

  Research Summary

  Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is caused by a hereditary deficiency of the enzyme glucocerebrosidase. Symptoms may begin early in life or in adulthood and include enlarged liver and grossly enlarged spleen, which can rupture and cause additional complications. Bone-related problems may be extensive. Bruising, fatigue, anaemia and low blood platelet count are also common.
  Most research effort to date has focused on strategies for augmenting enzyme levels to compensate for the underlying defect; enzyme replacement therapy (ERT) has been the cornerstone of treatment for Gaucher disease since the early 1990s and is highly effective in improving many of the clinical manifestations.

  Since bone-related problems of the disease remain a significant unmet medical need, this phase 4 study will focus on VPRIV on this. The effects of velaglucerase alfa (VPRIV) on BMD (bone mineral density) have been shown to be positive in phase 1/2 and 3 studies.

  Up to 40 people (≥16 and ≤65 years of age) will take part in this study for approximately 110 weeks.

  Patients who provide written consent and meet all eligibility criteria will be given VPRIV for up to 102 weeks. During this 102 week treatment period patients will return to the clinic for visits every 2 weeks. Patients also have the option for home infusions starting at week 7. Patients will need to return to the clinic for visits that require medical imaging and the completion of patient questions.

  The study involves procedures including: Physical exams, ECG (which measures the electrical activity of your heart), Vital signs, Blood draws, urine tests, DXA Scans (Dual energy X-ray absorptiometry which measures bone mineral density), MRI scans (Magnetic Resonance Imaging is a test that uses magnets and radio waves to produce pictures of the inside of the body) and patient questionnaires.

  Summary of results

  : As published on the Takeda clinical trials website: https://eur03.safelinks.protection.outlook.com/?url=http%3A%2F%2Furl6570.hra.nhs.uk%2Fls%2Fclick%3Fupn%3DXv3JSvJ-2B3M71ppf7N9agbbpZE2vbNZdOHfLVGU7cQCgRUViyEzaOnvGS0AriJBpc-2FRjOSCylozYJ6f-2B87gkFfHcvQm2uDqw-2F3diaJvcixYYDFQ7tL51CObg0Pod8PxoOFkMKHv-2FJod-2FBuCK6Wcsp7fCrOYrPzMkHptkLPRAtCDE-3DAwQC_E1aO2-2BZlVOSJJV-2FajQqskegTd6IRomHYTi-2Fbt8SH3YIwekxQaZV0w2kd-2F1v0z5vW1z143ezcBto8DJF1iiWkRpSgTs5tv6qNCVinuVCTOqkpZpT3NjGHLXyjysdk5IQ59MK0gjJXBNL2mLsXJJE7jXueAWZoeUXL32dplhB-2FYSeFqrWikJyO0G6K1L3FCB7rz-2B0pWzaBWpIJsN-2BNLKPxLw-3D-3D&data=04%7C01%7Capprovals%40hra.nhs.uk%7C5ae1c5cead90446f300408da1d25f39c%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C637854346078596768%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000&sdata=OCfZ1KM%2Fymj04qTCUIdYViyFw7aSiheJeua2TCB9AVE%3D&reserved=0

  Researchers wanted to learn how well VPRIV worked to repair bone damage. The results include only the 16 participants who completed the study. The other 5 participants received at least 1 VPRIV infusion but left the study early. The question researchers wanted to answer was:
  What was the average change in the bone mineral density of the lower back after 24 months of treatment?
  To answer this question, researchers compared DXA scan results of the lower back. The DXA scan is a high-precision type of X-ray that measures the bone mineral density.
  Both Z-scores and bone mineral density measurements were compared from before treatment and after 24 months of receiving VPRIV.
  • The results showed that the average Z-score increased by 0.17 Z-scores units after 24 months of treatment with VPRIV.
  • The results showed the average bone mineral density measurement increased by about 1% after 24 months of treatment with VPRIV.
  However, these results may be to chance and not to treatment with VPRIV.
  For more information on study results, please refer to the website: https://eur03.safelinks.protection.outlook.com/?url=http%3A%2F%2Furl6570.hra.nhs.uk%2Fls%2Fclick%3Fupn%3DXv3JSvJ-2B3M71ppf7N9agba4yu73OCS9U-2BkKS40W1kfatiYvtk1-2B2gi1K6vKImualqfjI4EWGRCq5YsNji0bHvTAkwPaQ9nnKXGAieLbmmPkZKERnbEhQ9yH-2B0TnLHfetZuS1_E1aO2-2BZlVOSJJV-2FajQqskegTd6IRomHYTi-2Fbt8SH3YIwekxQaZV0w2kd-2F1v0z5vW56ZWijdQk9Ybx2xcnbL4bYZtdFKSV9q-2B6LjX7TXw5La-2FSdg7n2XjpbLmQe7OwEbn1lbZpvrQYw10jxZ81OjPfGKXcxu0X0O1AQOonVGUm9uzGf4m-2F7vInMptw-2BPJs-2BZfIbB-2BtJw7DtzPICGkEdZhkw-3D-3D&data=04%7C01%7Capprovals%40hra.nhs.uk%7C5ae1c5cead90446f300408da1d25f39c%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C637854346078596768%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000&sdata=tkMjiAZeoLW84r4XB%2B1MTSVlZgpUj%2B7pu4cI9gHnjyI%3D&reserved=0

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  16/LO/0144

• Date of REC Opinion

  17 Feb 2016

• REC opinion

  Favourable Opinion