Nephropathic CystinosisPROREALNCT04246060
Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients
An observational study of Nephropathic Cystinosis, run by Chiesi SA/NV at 7 sites in Belgium.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Nephropathic Cystinosis
- What is being tested?
- Cysteamine Bitartrate
- How many people
- 31 participants
- Where
- 7 sitesBelgium
- Study dates
- 31 Jul 2020to 1 Jul 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All Belgian nephropathic cystinosis patients.
Who may be able to join
- Confirmed diagnosis of nephropathic cystinosis
- Undergoing cystine depletion therapy with oral cysteamine
- Signature of informed concent.
Who cannot join
None listed.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
Belgium7 sites
UZA
Antwerp
HUDERF
Brussels
UCL Saint-Luc
Brussels
UZ Gent
Ghent
UZ Leuven
Leuven
CHC
Liège
CHU
Liège
About this study
Official title: Multicentre, Ambispective, Observational, Real Life Study to Assess the Quality of Life Effectiveness of Extended Release (ER) Oral Cysteamine Therapy (Procysbi) in Belgian Patients Suffering From Nephropathic Cystinosis
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.
- Conditions
- Nephropathic Cystinosis
- Being tested
- Cysteamine Bitartrate
- Medical subject headings
- Cystinosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cysteamine BitartrateDrug
ERT
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Patients on extended release cysteamine treatment at study enrollment Receives: Drug: Cysteamine Bitartrate | |
| Cohort 2 | Patients switching from immediate release cysteamine to extended release cysteamine during the study Receives: Drug: Cysteamine Bitartrate | |
| Cohort 3 | Patients remaining on immediate release cysteamine treatment Receives: Drug: Cysteamine Bitartrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2020
- Study start31 Jul 2020
- Primary completion1 Jul 2022 (estimated)
- Study completion1 Jul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nephropathic CystinosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 31 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chiesi SA/NV
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04246060
- Sponsor's protocol ID
- CHIESI-NIS-003
- Acronym
- PROREAL
- Status last verified
- Nov 2021
- First submitted
- 27 Jan 2020
- Last updated
- 29 Nov 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicentre, Ambispective, Observational, Real Life Study to Assess the Quality of Life Effectiveness of Extended Release (ER) Oral Cysteamine Therapy (Procysbi) in Belgian Patients Suffering From Nephropathic Cystinosis
- ClinicalTrials.gov ID
- NCT04246060
- Acronym / sponsor ID
- PROREAL / CHIESI-NIS-003
- Conditions
- Nephropathic Cystinosis
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 31 participants (estimated)
- Lead sponsor
- Chiesi SA/NVUniversity, hospital or other
- Study start
- 31 Jul 2020 (actual)
- Primary completion
- 1 Jul 2022 (estimated)
- Study completion
- 1 Jul 2026 (estimated)
- First posted
- 29 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | Drug: Cysteamine BitartratePatients on extended release cysteamine treatment at study enrollment |
| Cohort 2 | – | Drug: Cysteamine BitartratePatients switching from immediate release cysteamine to extended release cysteamine during the study |
| Cohort 3 | – | Drug: Cysteamine BitartratePatients remaining on immediate release cysteamine treatment |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All Belgian nephropathic cystinosis patients.
Key inclusion criteria (3 listed)
- Confirmed diagnosis of nephropathic cystinosis
- Undergoing cystine depletion therapy with oral cysteamine
- Signature of informed concent.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cysteamine BitartrateERT | Drug | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
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
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Nov 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Nephropathic Cystinosis, run by Chiesi SA/NV at 7 sites in Belgium.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 29 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Nov 2021.