About the PH1 Study

The PH1 clinical research study is currently evaluating Alnylam’s investigational medication ALN-GO1 for the treatment of PH1, using a new approach called RNAi therapeutics. Adults and children may be able to take part if they:

  • Are 6–64 years old
  • Have been diagnosed with PH1
  • Have not had a liver and/or kidney transplant
  • Other criteria may apply

Interested individuals would first be assessed to determine if the study is right for them.

Find out about taking part in our current and future PH1 clinical research studies.

Clinicaltrials.gov reference: NCT02706886

What would taking part involve?

  • All participants in the PH1 Study will receive the investigational medication for three months during the study.
  • Before receiving the investigational medication, 25% (1 in 4) of study participants will be randomly assigned to receive placebo for three months.
  • A study nurse will administer the study medication as a monthly injection using a small needle under the skin.
  • Participation in the study lasts up to 18 months, with monthly visits for most of that time.
  • At study visits, participants may receive the study medication, undergo a range of health examinations, and provide blood and urine samples. The study team will aim to make appointments as convenient as possible for participants and their families.
  • All study-related medication, medical assessments, and tests will be provided at no cost to study participants. In addition, study-related travel and accommodation (if required) will also be provided for participants.
  • At the end of the study, participants may be able to receive the investigational medication as part of an extension study.

It is important to note that the decision to take part is completely voluntary. A study participant is free to change his or her mind and leave the study at any time for any reason.

Find out where the study is taking place.