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National observational study to monitor the new guideline concerning treatment of atypical hemolytic uremic syndrome


- candidate number24587
- NTR NumberNTR5988
- ISRCTNISRCTN no longer applicable
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR8-jul-2016
- Secondary IDsNL52817.091.15 CMO Arnhem-Nijmegen
- Public TitleNational observational study to monitor the new guideline concerning treatment of atypical hemolytic uremic syndrome
- Scientific TitleNational observational study to monitor the new guideline concerning treatment of atypical hemolytic uremic syndrome
- ACRONYMCUREiHUS
- hypothesis
- Healt Condition(s) or Problem(s) studiedAtypical hemolytic uremic syndrome, Complement, Orphan drug
- Inclusion criteria1. Patients of all ages, suspected of or diagnosed with aHUS
2. Treated conform the new Dutch guideline for aHUS.
3. Subject and/or his parents is able and willing to sign the Informed Consent
- Exclusion criteria1, Subject and/or his parents is not able or willing to sign the Informed Consent before start of the study.
2. Patients with other etiological forms of HUS than aHUS
- mec approval receivedyes
- multicenter trialyes
- randomisedno
- group[default]
- TypeSingle arm
- Studytypeobservational
- planned startdate 1-aug-2016
- planned closingdate1-aug-2018
- Target number of participants50
- InterventionsNone
- Primary outcomeMonitoring and evaluation of the Dutch guideline for treatment of aHUS in children and adults during two years.
- Secondary outcomeTwo add-on studies are submitted with this research protocol.
1. To gather more inside in the pharmacokinetics and dynamics of eculizumab.
2. To test the psychometric properties of the questionnaire: medication-related patient-reported experience measures (M-PREM) and the expectations measure that are aimed to better understand patient expectations, experiences and satisfaction with medications.
- TimepointsObservational study for two years
- Trial web sitewww.CUREiHUS.nl
- statusplanned
- CONTACT FOR PUBLIC QUERIESMD Kioa Wijnsma
- CONTACT for SCIENTIFIC QUERIESMD Kioa Wijnsma
- Sponsor/Initiator Radboud University Medical Center Nijmegen
- Funding
(Source(s) of Monetary or Material Support)
ZON-MW, The Netherlands Organization for Health Research and Development
- Publications
- Brief summaryAtypical hemolytic uremic syndrome (aHUS) is a rare, but severe form of thrombotic microangiopathy, and is considered the consequence of complement dysregulation. Atypical HUS has a poor outcome with mortality up to 10% and over 50% of patients developing end stage renal disease. Since the end of 2012, these outcomes have greatly improved with the introduction of eculizumab. The European Medicines Agency has approved eculizumab for the treatment of aHUS patients. The guideline advocates lifelong treatment. However, there is no hard evidence to support this advice. Historically, a substantial number of aHUS patients were weaned of plasma therapy, often without disease recurrence. Moreover, the long-term consequences of eculizumab treatment are unknown. Recently, a new guideline concerning therapy in aHUS patients and hereby addressing therapy adjustment and/or discontinuation in aHUS patients is implemented in the Netherlands. In this national, multicenter, observational study we will include all aHUS, both pediatric and adults, patients who are treated conform this new guideline. During four years this guideline will be monitored and evaluated.
- Main changes (audit trail)
- RECORD8-jul-2016 - 26-sep-2016


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