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uniQure Announces First Two Patients Treated in Clinical Trial of AMT-130 for the Treatment of HD

According to a press release dated June 19, 2020, uniQure N.V.(NASDAQ: QURE), a leading gene therapy company advancing trans-formative therapies for patients with severe medical needs, today announced that the first two patients in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease have been treated. The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States, with now one patient treated with AMT-130, and one patient who received the imitation surgery.

It is important to note that uniQure has been very transparent in regards to making sure patients are informed about the procedures and risks involved in this study.

What is involved in this study?

According to uniQure's website:

It is important to reiterate that AMT-130 is administered only once. The study will test two dose levels of AMT-130 (low dose and high dose). The safety of the low dose will be assessed before testing the high dose. A total of 26 patients will be enrolled. Of the 26 patients enrolled, 16 will receive treatment with AMT-130 (“Treated Group’) and 10 will not receive any study treatment (“Imitation Group”). Patients assigned to the Treated Group will receive the dose of AMT-130 during a neurosurgical visit. AMT-130 will be infused into two specific brain regions (caudate and striatum) under general anesthesia. This is done by drilling two to six small holes in the skull and administering AMT-130 by a micro-catheter. For patients assigned to the Imitation Group, small, superficial holes will be drilled into the surface of the skull under general anesthesia, but they will NOT receive a dose of AMT-130. The main part of the study lasts for 18 months, with additional annual visits out to 5 years for continued safety follow up. Procedures will include clinic visits, assessments of physical and neurological health, a neurosurgical procedure, lumbar punctures (LP), brain scans, and samples from body fluids. The study is “double blinded” meaning neither the patient, the investigator or clinical staff will know if the patient is in the Treated Group or the Imitation Group.

What are the risks involved for patients in this study?

According to their website:

This is the first study in humans testing AMT-130 gene therapy. Currently, there is no information on the long-term side effects of AMT-130 in humans. AMT-130 has been studied in animals. The doses in this study are based on doses given to animals and were found to be generally safe. Some AAV vectors have been approved for clinical use and are being used in several gene therapy clinical trials. In general, AAV vectors are considered safe. Risks that should be considered are the following:
  • Treatment with gene therapy is permanent and cannot be removed once administered. (May include adverse reactions)

  • AMT-130 lowers both normal and mutant huntingtin protein in animals. The effects of lowering normal and mutant huntingtin protein levels in adults are currently unknown.

  • Patients may be ineligible to receive another gene therapy treatment after receiving AMT-130.

  • Because AMT-130 is investigational, there may be risks or side effects that are not known at this time. There may be rare and unknown side effects, including reactions that may be life threatening.

  • AMT-130 is delivered directly to the brain and will include risks associated with general anesthesia and micro-catheter placement in the brain.



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