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Ionis-HTT rx Study

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Sponsored by Ionis Pharmaceuticals (formerly ISIS Pharmaceuticals)

Click here for the latest update from IONIS/Roche. 

The Ionis-HTTRx  trial is a small safety study that aims to test a ‘huntingtin lowering’ therapy, called an antisense drug. The antisense drug specifically targets the huntingtin mRNA that inhibits and prevents the formation of huntingtin protein directly. This is the first human trial to target the underlying cause of HD, and has shown great promise in animal models. The drug will be administered via a needle inserted into the fluid-filled space below the lower spinal cord.


This study enrollment is now closed.

Formal Name
A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered Ionis-HTTRx in Patients With Early Manifest Huntington’s Disease

Study Type

This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending doses of Ionis-HTTRx administered intrathecally to adult patients with early manifest Huntington disease.

Canadian Locations and Contact Information:

British Columbia
Location: Centre for HD, UBC Hospital, Vancouver
Status: Not Recruiting
Coordinator: Joji Decolongon
Phone: 604-875-3801

Exclusion Criteria  (eligibility that would exclude you from this study)

  • Clinically significant medical condition, such as severe chorea, active suicidal ideation or any other conditions which would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study
  • Recent treatment with another investigational drug, biological agent, or device
  • Prior treatment with an antisense oligonucleotide (including siRNA)
  • Any history of gene therapy or cell transplantation or any other experimental brain surgery
  • Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter
  • History of post-lumbar-puncture headache of moderate or severe intensity and/or blood patch
  • Malignancy within 5 years of Screening, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated
  • Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of Screening or planned during the study

Source A service of the U.S. National Institutes of Health Identifier: NCT02519036
This study is not currently recruiting participants.
Verified August 5, 2015 by Ionis Pharmaceuticals
Information provided by (Responsible Party): Ionis Pharmaceuticals

For more detailed information on this study and others and for the most recent updates, please go to is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world.

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