NCT04246060

Brief Summary

Most of the real world evidence data related to efficacy of cysteamine therapy is retrospective. This study is a ambispective study to investigate the impact of cystine depletion therapy on the quality of life of patients and their parents.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
31

participants targeted

Target at below P25 for all trials

Timeline
2mo left

Started Jul 2020

Longer than P75 for all trials

Geographic Reach
1 country

7 active sites

Status
enrolling by invitation

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress97%
Jul 2020Jul 2026

First Submitted

Initial submission to the registry

January 27, 2020

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 29, 2020

Completed
6 months until next milestone

Study Start

First participant enrolled

July 31, 2020

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2022

Completed
4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2026

Expected
Last Updated

November 29, 2021

Status Verified

November 1, 2021

Enrollment Period

1.9 years

First QC Date

January 27, 2020

Last Update Submit

November 25, 2021

Conditions

Nephropathic Cystinosis

Outcome Measures

Primary Outcomes (1)

  • to descriptively assess the change from baseline and percent change from baseline in the quality of life overall summary sores at 12 months in switch cohort

    12 months

Study Arms (3)

Cohort 1

Patients on extended release cysteamine treatment at study enrollment

Drug: Cysteamine Bitartrate

Cohort 2

Patients switching from immediate release cysteamine to extended release cysteamine during the study

Drug: Cysteamine Bitartrate

Cohort 3

Patients remaining on immediate release cysteamine treatment

Drug: Cysteamine Bitartrate

Interventions

Cysteamine BitartrateDRUG

ERT

Cohort 1Cohort 2Cohort 3

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

All Belgian nephropathic cystinosis patients.

You may qualify if:

  • Confirmed diagnosis of nephropathic cystinosis
  • Undergoing cystine depletion therapy with oral cysteamine
  • Signature of informed concent.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

UZA

Antwerp, Belgium

Location

HUDERF

Brussels, Belgium

Location

UCL Saint-Luc

Brussels, Belgium

Location

UZ Gent

Ghent, Belgium

Location

UZ Leuven

Leuven, Belgium

Location

CHC

Liège, Belgium

Location

CHU

Liège, Belgium

Location

MeSH Terms

Conditions

Cystinosis

Interventions

Cysteamine

Condition Hierarchy (Ancestors)

Lysosomal Storage DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

MercaptoethylaminesEthylaminesAminesOrganic ChemicalsSulfhydryl CompoundsSulfur Compounds

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
OTHER
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 27, 2020

First Posted

January 29, 2020

Study Start

July 31, 2020

Primary Completion

July 1, 2022

Study Completion (Estimated)

July 1, 2026

Last Updated

November 29, 2021

Record last verified: 2021-11

Locations

BE(7)