• Skip to main content
  • Skip to footer

PHYOX Clinical Trial Engagement Website | Dicerna Pharmaceuticals

  • About Primary Hyperoxaluria
  • Our Clinical Trials
    • PHYOX7
    • PHYOX8
  • Resources
  • FAQs
  • English
    • Français (French)
    • Deutsch (German)
    • Polski (Polish)
    • Español (Spanish)
    • Italiano (Italian)
    • Română (Romanian)

Glossary of Terms

Glossary of Terms

Clinical Trial

A clinical trial is a research study that explores whether a medical strategy, investigational treatment, or device is safe and effective for humans.

Double-blind

A blinded experiment is an experiment in which information about the test is hidden from the participant, to reduce or eliminate bias, until after a trial outcome is known. Double-blind means that the investigator does not know the information either.

Genes

Genes are strings of DNA that serve as the functional and physical unit of heredity passed from parent to offspring. Most genes contain the information for making a specific protein. When mutated or inappropriately activated, certain genes can drive diseases, including cancer.

Informed Consent

Informed consent is when the patient/caregiver/legal guardian is provided clear, easy-to-understand information about a clinical trial as he/she is deciding whether to participate in a clinical trial.

NEDOSIRAN (formerly DCR-PHXC)

Nedosiran is an investigational therapy that Dicerna is developing for the treatment of primary hyperoxaluria (PH).

Phase 1

Phase 1 tests safety in humans by giving the treatment or drug to healthy volunteers.

Phase 2

Phase 2 tests safety and effectiveness in patients living with the disease being studied.

Phase 3

Phase 3 tests safety and confirms effectiveness in patients with the disease and studies a larger group of people than the Phase II trial.

Phase 4

Phase 4 tests the drug’s effect in various populations and any side effects associated with long-term use after it is approved.

PHYOXTM

PHYOXTM is the name of Dicerna’s clinical development program for nedosiran, which includes all phases of development.

Pivotal trial

A pivotal trial is a clinical trial intended to provide evidence for a drug marketing approval, e.g. by the United States Food and Drug Administration.

Primary hyperoxaluria

Primary hyperoxaluria (PH) is a family of severe, ultra-rare, genetic liver disorders characterized by overproduction of oxalate, a natural chemical in the body that is normally eliminated as waste through the kidneys. In patients with PH, the kidneys are unable to eliminate the large amount of oxalate that is produced, and the accumulation of oxalate can result in severe damage to the kidneys and other organs.

Randomized trial

A randomized controlled trial is a type of study that aims to reduce bias when testing a new treatment. The people participating in the trial are randomly allocated to either the group receiving the treatment under investigation or to a group receiving standard treatment as the control.

RNA

RNA, which stands for ribonucleic acid, is a genetic material with diverse uses within the cell. Most notably, when a gene is being used, a copy of its DNA sequence is made using RNA. This RNA copy is known as messenger RNA, or mRNA. The RNAi machinery targets mRNA.

RNA interference (RNAi)

RNAi is a biologic process in which certain double-stranded RNA molecules inhibit the expression of disease-causing genes by destroying the messenger RNAs (mRNAs) of those genes.

Subcutaneous delivery

Subcutaneous means “under the skin.” So, a therapy that is delivered subcutaneously is delivered with a needle under the skin before it enters the bloodstream.

Footer

Safety Information

Nedosiran, formerly DCR-PHXC, is an investigational drug intended for the treatment of patients with PH. Investigational drugs are medicines that have not yet been approved by appropriate regulatory agencies. The safety and efficacy for nedosiran has not been established yet. Current and future studies are evaluating the safety and efficacy for nedosiran.

©2021 Dicerna Pharmaceuticals, Inc. / Lexington, MA

MED-ALL—000011 v2.0

  • About PH
  • Our Clinical Trials
    • PHYOX7
    • PHYOX8
  • Resources
  • FAQ
  • Glossary of Terms
  • Privacy Policy
  • Cookies Policy

Select your language:

  • English
  • Français
  • Deutsch
  • Italiano
  • Polski
  • Română
  • Español
This website uses strictly necessary cookies to enable the website to function.

If you agree we would also like to use non-essential cookies to enable us to observe traffic levels to the website, to improve the quality of your experience when visiting our website, and to show you relevant advertisements. For more information, including on the third parties who use cookies on our website, please see our Cookie Policy
Cookie settingsACCEPT
Manage consent

Privacy Overview

This website uses cookies to improve your experience while you navigate through the website. Out of these cookies, the cookies that are categorized as necessary are stored on your browser as they are essential for the working of basic functionalities of the website. We also use third-party cookies that help us analyze and understand how you use this website. These cookies will be stored in your browser only with your consent. You also have the option to opt-out of these cookies. But opting out of some of these cookies may have an effect on your browsing experience.
Necessary
Always Enabled
Necessary cookies are absolutely essential for the website to function properly. These cookies ensure basic functionalities and security features of the website, anonymously.
CookieDurationDescription
cookielawinfo-checbox-analytics1 yearThis cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Analytics".
cookielawinfo-checbox-functional1 yearThe cookie is set by GDPR cookie consent to record the user consent for the cookies in the category "Functional".
cookielawinfo-checbox-others1 yearThis cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Other.
cookielawinfo-checkbox-advertisement1 yearThe cookie is set by GDPR cookie consent to record the user consent for the cookies in the category "Advertisement".
cookielawinfo-checkbox-necessary1 yearThis cookie is set by GDPR Cookie Consent plugin. The cookies is used to store the user consent for the cookies in the category "Necessary".
cookielawinfo-checkbox-performance1 yearThis cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Performance".
viewed_cookie_policy6 monthsThe cookie is set by the GDPR Cookie Consent plugin and is used to store whether or not user has consented to the use of cookies. It does not store any personal data.
Functional
Functional cookies help to perform certain functionalities like sharing the content of the website on social media platforms, collect feedbacks, and other third-party features.
CookieDurationDescription
DRIVE_STREAMsessionThis cookie is set by Google Drive. This cookie allows Google Drive video player to obtain correct file from its cloud storage.
Performance
Performance cookies are used to understand and analyze the key performance indexes of the website which helps in delivering a better user experience for the visitors.
CookieDurationDescription
_gat1 minuteThis cookies is installed by Google Universal Analytics to throttle the request rate to limit the colllection of data on high traffic sites.
Analytics
Analytical cookies are used to understand how visitors interact with the website. These cookies help provide information on metrics the number of visitors, bounce rate, traffic source, etc.
CookieDurationDescription
_ga1 yearsThis cookie is installed by Google Analytics. The cookie is used to calculate visitor, session, campaign data and keep track of site usage for the site's analytics report. The cookies store information anonymously and assign a randomly generated number to identify unique visitors.
_gid1 dayThis cookie is installed by Google Analytics. The cookie is used to store information of how visitors use a website and helps in creating an analytics report of how the website is doing. The data collected including the number visitors, the source where they have come from, and the pages visted in an anonymous form.
Advertisement
Advertisement cookies are used to provide visitors with relevant ads and marketing campaigns. These cookies track visitors across websites and collect information to provide customized ads.
CookieDurationDescription
NID6 monthsThis cookie is used to a profile based on user's interest and display personalized ads to the users.
Others
Other uncategorized cookies are those that are being analyzed and have not been classified into a category as yet.
Save & Accept