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

Anemia Clinical Trial

— PKD  

Official title:

Pyruvate Kinase Deficiency Global Longitudinal Registry: Patient-Reported Outcomes

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04964323
• Other study ID #: AG348-C-015
• Status: Terminated
• Start date: July 2, 2021
• Est. completion date: June 30, 2023

Study information

• Verified date: September 2023
• Source: Agios Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 15
• Est. completion date: June 30, 2023
• Est. primary completion date: June 30, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Anemia
• Anemia, Hemolytic
• Hemolysis
• Pyruvate Kinase Deficiency

Locations

Country	Name	City	State
Italy	Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico	Milan
Netherlands	Universitair Medisch Centrum Utrecht	Utrecht
Spain	Hospital Infantil Universitario Niño Jesus - PIN	Madrid
United Kingdom	Hammersmith Hospital	London
United States	Massachusetts General Hospital	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Agios Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Italy,  Netherlands,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline in Pyruvate Kinase Deficiency Diary (PKDD) Score	The PKDD is a 7-item measure of the core signs and symptoms of PK deficiency. The score range is 25 to 76, with higher scores indicating a higher disease burden.	Baseline, up to Day 672
Primary	Change from Baseline in Pyruvate Kinase Deficiency Impact Assessment (PKDIA) Score	The PKDIA is a 12-item measure of the impacts of PK deficiency on patients' HRQoL. The score range is 30 to 76, with higher scores indicating a higher disease burden.	Baseline, up to Day 672
Primary	Change from Baseline in Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI: SHP) 2.0 Score	WPAI:SHP is a 6-question participant-rated questionnaire to determine the amount of absenteeism, presenteeism, work productivity loss, and daily activity impairment attributable to PK deficiency for a period of 7 days prior to each visit. It yields 4 sub-scores: work time missed (absenteeism), impairment while working (presenteeism or reduced on-the-job effectiveness), overall work impairment (work productivity loss or absenteeism plus presenteeism) and activity impairment (daily activity impairment). These sub-scores are transformed to impairment percentages (range from 0 to 100), with higher numbers indicating greater impairment and less productivity.	Baseline, up to Day 672
Primary	Change from Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An)	FACT-An is composed of five subscales: Physical Well-Being [score range 0-28], Social/Family Well-Being [score range 0-28], Emotional Well-Being [score range 0-24], and Functional Well-Being [score range 0-28] and specific questions concerning anemia [score range 0-80]. The score at each item is summed. The sum is multiplied by the number of items in the subscale and then divided by the number of items answered. This produces the subscale score. The subscale scores are added to derive a total score [score range 0-188]. A higher score indicates better quality of life.	Baseline, up to Day 672