This trial is active, not recruiting.

Condition hereditary hemochromatosis
Treatment phlebotomy and erythrocytapheresis
Phase phase 3
Sponsor Sanquin Research & Blood Bank Divisions
Collaborator Maastricht University Medical Center
Start date May 2008
End date December 2013
Trial size 40 participants
Trial identifier NCT01398644, 07-2-104


Hereditary hemochromatosis (HH) is a genetic disorder of iron metabolism, resulting in excessive iron overload. Phlebotomy is currently the standard therapy. More recently Therapeutic Erythrocytapheresis (TE) has become a new therapeutic modality, which potentially offers a more efficient method to remove iron overload with fewer procedures.In the proposed clinical trial the investigators will examine whether TE can keep the ferritin levels in patients requiring maintenance therapy below 50 microg/L, with minimally half the number of treatment procedures when compared to current standard therapy by P.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification efficacy study
Intervention model crossover assignment
Masking single blind (outcomes assessor)
Primary purpose treatment
(Active Comparator)
Patients are treated with phlebotomy if ferritin level >50 ug/l
phlebotomy and erythrocytapheresis
Phlebotomy- removal of 500 ml whole blood Erythrocytapheresis- removal of 300-800 ml erythrocytes
Patients are treated with erythrocytapheresis if serum ferritin level >50ug/l
phlebotomy and erythrocytapheresis
Phlebotomy- removal of 500 ml whole blood Erythrocytapheresis- removal of 300-800 ml erythrocytes

Primary Outcomes

The difference in number of required treatments and the interval between treatments per year to keep the serum ferritin levels between 30-50 microg/L
time frame: one year after first phlebotomy treatment and one year after first erythrocytapheresis treatment

Eligibility Criteria

Male or female participants from 18 years up to 90 years old.

Inclusion Criteria: - homozygous for C282Y - currently treated with phlebotomy as maintenance therapy for at least 6 month - ferritin level between 30-50 micog/L - age 18 years an older - weight more than 50 kg - signed informed consent - willingness to fill out additional questionnaires at three points in time Exclusion Criteria: - chelating therapy - forced dietary regime - aged below 18 years - excessive overweight ( BMI more than 35) - pregnancy

Additional Information

Official title Erythrocytapheresis Versus Phlebotomy as Maintenance Therapy in Patients With Hereditary Hemochromatosis; a Randomised, Single Blinded Sequential, Cross-over Trial
Principal investigator Eva Rombout, MD
Description The research population exists of patients with HH ( by genetic analysis confirmed as homozygous for C282Y) living in south-east of the Netherlands and currently treated with phlebotomy as maintenance treatment to keep their serum ferritin levels < 50 ug/l. Ferritin level at start of the inclusion between 30-50ug/l. Exclusion criteria are: patient receiving other therapies such as chelating therapy or forced dietary regimen, further patients with excessive overweight (BMI>35). After enrollment the patients will be randomized to start either with TE or continue with P. After a year of treatment and being at a serum ferritin level <50ug/l, patients will continue the study but then being treated with the other of the two treatments. Randomization will be done by blocked randomization.
Trial information was received from ClinicalTrials.gov and was last updated in December 2013.
Information provided to ClinicalTrials.gov by Sanquin Research & Blood Bank Divisions.