Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)

  • STATUS
    Recruiting
  • End date
    Aug 1, 2025
  • participants needed
    75
  • sponsor
    Agios Pharmaceuticals, Inc.
Updated on 19 September 2021
anemia

Summary

The primary objective of this study is to understand and characterize the health-related quality of life (HRQoL) and disease burden of adult participants with PK deficiency receiving routine clinical care. This study is an observational (i.e., noninterventional), longitudinal, multicenter, global registry for participants with PK deficiency, a rare nonspherocytic hemolytic anemia.

This study will be open for enrollment for 2 years and all enrolled participants will be followed prospectively for up to 96 weeks.

Data will be collected from participants who have provided informed consent and authorization pursuant to applicable laws and regulations.

Description

Participants aged 18 years and previously enrolled in Study AG348-C-008 (NCT03481738) will be eligible to participate in this study. Data will be submitted via electronic patient-reported outcomes (ePRO) application.

All data collection efforts will abide by this protocol and be prospectively disclosed in the study informed consent. This study, with the appropriate participant (and or parent/guardian) consent/assent, may incorporate retrospective data from other properly consented studies done for the purpose of examining the longitudinal natural history of PK deficiency.

Details
Condition Blood disorder, Anemia, Hemolytic anemia, Pyruvate Kinase Deficiency, anaemia, Pyruvate Kinase Deficiency Anemia
Clinical Study IdentifierNCT04964323
SponsorAgios Pharmaceuticals, Inc.
Last Modified on19 September 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Participant must be greater than or equal to ()18 years of age
Participant must be enrolled in Study AG348-C-008

Exclusion Criteria

Participant is enrolled in an Agios-sponsored clinical study involving treatment with a pyruvate kinase activator
Clear my responses

How to participate?

Step 1 Connect with a site
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar
Name

Primary Contact

site
Name

0/250
Preferred Language
Other Language
Please verify that you are not a bot.

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Loading...

Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note