search  
 


Home

Who are we?

Why
register?


Signup for
registration


Online registration

Log in to register
your trial


Search a trial

NRT en CCMO

Contact

NEDERLANDS





MetaRegister
van CCT (UK)


ISRCTN-Register
van CCT (UK)


Congenital hemolytic anemia: causes, symptoms and consequences


- candidate number22518
- NTR NumberNTR5337
- ISRCTNISRCTN no longer applicable
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR30-jul-2015
- Secondary IDsNL53609.041.15 
- Public TitleCongenital hemolytic anemia: causes, symptoms and consequences
- Scientific TitleClinical sequelae and pathophysiology of rare congenital hemolytic anemias
- ACRONYMZEbRA-study
- hypothesisObservational study. We aim to analyze the clinical consequences of congenital hemolytic anemia in order to treat and monitor patients optimally. Secondary, we aim to gain understanding in the pathophysiology of congenital hemolytic anemia
- Healt Condition(s) or Problem(s) studiedCongenital hemolytic anemia
- Inclusion criteriaAdult patients who meet the criteria of non-immune mediated hemolytic anemia in whom acquired causes have been excluded in the diagnostic track. Such patients can be subdivided into 4 main categories:
1. red cell membrane disorders, e.g. hereditary spherocytosis
2. disorders of hemoglobin, e.g. thalassemia
3. metabolic disorders, e.g. pyruvate kinase deficiency
4. hemolytic anemia e.c.i.
- Exclusion criteriaInability to give informed consent
- mec approval receivedyes
- multicenter trialno
- randomisedno
- group[default]
- TypeSingle arm
- Studytypeobservational
- planned startdate 1-sep-2015
- planned closingdate1-sep-2020
- Target number of participants100
- Interventionsnot applicable
- Primary outcomeTo create insight in current disease burden by creating a descriptive cohort of patients, diagnosed with rare congenital hemolytic anemia.
Points of interest are:
- Prevalence and incidence of disease
- Quality of life
- Prevalence and incidence of iron overload
- Prevalence and incidence of comorbidities and related silent organ damage
- Prevalence and incidence of splenectomy and complications
- Secondary outcomeTo further analyze the pathophysiology of congenital hemolytic anemia: to perform a case control study comparing patient parameters and healthy control parameters.
Parameters of interest are:
1. The tolerability of low hemoglobin levels in rare congenital hemolytic anemia patients.
2. Patterns in laboratory parameters: pro-inflammatory profile, Red blood cell characteristics, microparticle analysis, and markers of coagulation activation.
3. RNA seq parameters for peripheral blood mononuclear cell transcriptome mapping using blood sample analysis and then compare and relate outcome to other results of the study.
- TimepointsEnrollment, 1 year after enrollment, 2 years after enrollment
- Trial web sitenot applicable
- statusopen: patient inclusion
- CONTACT FOR PUBLIC QUERIESMD, PhD-Candidate H.A.S. van Straaten
- CONTACT for SCIENTIFIC QUERIESMD, PhD-Candidate H.A.S. van Straaten
- Sponsor/Initiator University Medical Center Utrecht (UMCU)
- Funding
(Source(s) of Monetary or Material Support)
University Medical Center Utrecht (UMCU)
- Publications
- Brief summaryRationale: Rare congenital hemolytic anemias share a common clinical picture and common pathophysiologic pathways such as iron overload, severe anemia and hemolysis. These patients develop comparable organ damage to patients with more common and more studied congenital hemoglobinopathies such as thalassemia and sickle cell disease. Treatment nowadays is mainly supportive. Research is necessary in order to find the best monitoring- and treatment regimens. Objective: To create insight in current disease burden by creating a descriptive cohort of patients, diagnosed with rare congenital hemolytic anemia. To further analyze the pathophysiology of congenital hemolytic anemia by performing a case control study comparing patient parameters and healthy control parameters. Study design: longitudinal observational descriptive cohort study and case-control study
Study population: All patients diagnosed with rare congenital hemolytic anemia. The majority of this patient group will be composed of patients with hereditary red blood cell membranopathies and red blood cell enzyme disorders.

Main study parameters/endpoints:
Prevalence and incidence of disease
Quality of life
Prevalence and incidence of iron overload
Prevalence and incidence of comorbidities and related silent organ damage
Prevalence and incidence of splenectomy and complications
The study consist of medical chart review, yearly two short quality of live questionnaires, a 6 minute walking test and one additional venipuncture.
- Main changes (audit trail)
- RECORD30-jul-2015 - 3-mei-2017


  • Indien u gegevens wilt toevoegen of veranderen, kunt u een mail sturen naar nederlands@trialregister.nl