Hereditary HemochromatosisPhase 3NCT01398644
Erythrocytapheresis Versus Phlebotomy as Maintenance Therapy in Hereditary Hemochromatosis (HH) Patients
A Phase 3 study testing Phlebotomy and erythrocytapheresis in people with Hereditary Hemochromatosis, run by Sanquin Research & Blood Bank Divisions at 1 site in Netherlands.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 to 90 yearsHealthy volunteers can take part
- Conditions the study is for
- Hereditary Hemochromatosis
- What is being tested?
- Phlebotomy and erythrocytapheresis
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteNetherlands
- Study dates
- May 2008to Dec 2013
- Phase
- Phase 3
- 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 18 to 90 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- chelating therapy
- forced dietary regime
- aged below 18 years
- excessive overweight ( BMI more than 35)
- pregnancy
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Erythrocytapheresis Versus Phlebotomy as Maintenance Therapy in Patients With Hereditary Hemochromatosis; a Randomised, Single Blinded Sequential, Cross-over Trial
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.
Read the detailed 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.
- Conditions
- Hereditary Hemochromatosis
- Being tested
- Phlebotomy and erythrocytapheresis
- Keywords
- Phlebotomy, Therapeutic erythrocytapheresis, Hereditary Hemochromatosis, maintenance therapy
- Medical subject headings
- Hemochromatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Phlebotomy and erythrocytapheresisOther
Phlebotomy- removal of 500 ml whole blood Erythrocytapheresis- removal of 300-800 ml erythrocytes
| Study group | Role | What they receive |
|---|---|---|
| Phlebotomy -intervention phlebotomy | Active comparator | Patients are treated with phlebotomy if ferritin level >50 ug/l Receives: Other: Phlebotomy and erythrocytapheresis |
| Erythrocytapheresis | Experimental | Patients are treated with erythrocytapheresis if serum ferritin level >50ug/l Receives: Other: Phlebotomy and erythrocytapheresis |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: one year after first phlebotomy treatment and one year after first erythrocytapheresis treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2011 (estimated)
- Study startMay 2008
- Primary completionDec 2013 (estimated)
- Study completionDec 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary HemochromatosisStudies 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sanquin Research & Blood Bank Divisions
- Sponsor type
- University, hospital or other
- Collaborators
- Maastricht University Medical Center, Atrium Medical Center, Radboud University Medical Center, Orbis Medical Centre
- Responsible party
- Principal investigator, Eva Rombout, Sanquin Research & Blood Bank Divisions
- Investigators
- Eva Rombout, MD, Principal investigator, Sanquin Blood Supply
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01398644
- Sponsor's protocol ID
- 07-2-104
- Status last verified
- Dec 2013
- First submitted
- 19 Jul 2011
- Last updated
- 4 Dec 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Erythrocytapheresis Versus Phlebotomy as Maintenance Therapy in Patients With Hereditary Hemochromatosis; a Randomised, Single Blinded Sequential, Cross-over Trial
- ClinicalTrials.gov ID
- NCT01398644
- Acronym / sponsor ID
- 07-2-104
- Conditions
- Hereditary Hemochromatosis
- Keywords
- Phlebotomy, Therapeutic erythrocytapheresis, Hereditary Hemochromatosis, maintenance therapy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Sanquin Research & Blood Bank DivisionsUniversity, hospital or other
- Collaborators
- Maastricht University Medical Center, Atrium Medical Center, Radboud University Medical Center, Orbis Medical Centre
- Study start
- May 2008
- Primary completion
- Dec 2013 (estimated)
- Study completion
- Dec 2013 (estimated)
- First posted
- 20 Jul 2011 (estimated)
- 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.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Phlebotomy -intervention phlebotomy | Active comparator | Other: Phlebotomy and erythrocytapheresisPatients are treated with phlebotomy if ferritin level >50 ug/l |
| Erythrocytapheresis | Experimental | Other: Phlebotomy and erythrocytapheresisPatients are treated with erythrocytapheresis if serum ferritin level >50ug/l |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (5 listed)
- chelating therapy
- forced dietary regime
- aged below 18 years
- excessive overweight ( BMI more than 35)
- pregnancy
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 |
|---|---|---|
| Phlebotomy and erythrocytapheresisPhlebotomy- removal of 500 ml whole blood Erythrocytapheresis- removal of 300-800 ml erythrocytes | Other | Erythrocytapheresis, Phlebotomy -intervention phlebotomy |
Primary outcome
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2013.
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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.
Eva Rombout, MD principal investigator
Sanquin Blood Supply
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Phlebotomy and erythrocytapheresis in people with Hereditary Hemochromatosis, run by Sanquin Research & Blood Bank Divisions at 1 site in Netherlands.
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 7 inclusion and 5 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 4 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Netherlands. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
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). 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 4 Dec 2013.