HemochromatosisHe-ppiNCT01524757
Proton Pump Inhibitors in the Prevention of Iron Reaccumulation in Patient With Hereditary Hemochromatosis
A study testing Pantoprazole in people with Hemochromatosis, run by Maastricht University Medical Center at 3 sites in 2 countries.
Trial summary
Hereditary Hemochromatosis (HH) is a genetic disorder of iron metabolism, resulting in excessive iron overload causing damage of different important organs like heart, liver, pancreas and joints. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Hemochromatosis
- What is being tested?
- Pantoprazole
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 48 participants
- Where
- 3 sitesin 2 countries
- Study dates
- Mar 2012to Aug 2013
- Phase
- Interventional
- 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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Patients with hereditary hemochromatosis (HH), homozygous for C282Y, currently treated with phlebotomy as maintenance therapy for at least 12 months with ≥ 3 phlebotomies per year.
- Ferritin level between 50-100 μg/L at start of the inclusion.
- Age: 18 years- 60 years and weight > 50 kg.
Who cannot join
- Patients receiving other therapies such as chelating therapy or forced dietary regimen.
- Patients younger than 18 years.
- HH patients with excessive overweight (BMI > 35).
- Patients who are mentally incapacitated.
- Women being pregnant or expecting/ planning to become pregnant during the one year period of the study.
- Patients with a malignancy.
- Patients already on PPI treatment.
- Patients who experienced side effects of PPI's.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
Netherlands2 sites
Atrium MC Parkstad
Heerlen, Limburg, 6440 AG
Cees Deursen, dr, +31-45-5279639, c.vandeursen@atriummc.nl
Reggy Jaspers, drs, +31-43-3875021, r.jaspers@Mumc.nl
Maastricht university medical center
Maastricht, Limburg, 6202AZ
Reggy Jaspers, drs, +31-43-3875021, r.jaspers@mumc.nl
Ger Koek, dr-, +31-43-3875021, gh.koek@mumc.nl
Belgium1 site
University hospital Gasthuisberg
Leuven, Limburg, 3000
David Cassiman, prof. dr., +32 16344626, david.cassiman@uzleuven.be
David Cassiman, prof. dr.
About this study
Hereditary Hemochromatosis (HH) is a genetic disorder of iron metabolism, resulting in excessive iron overload causing damage of different important organs like heart, liver, pancreas and joints. Complications and symptoms can regress by intensive treatment reducing the iron overload stores.Different genes have been identified playing a role in the pathophysiology of iron overload. A clinically important HFE gene mutation is the C282Y, located on chromosome 6. Phlebotomy is currently the standard therapy which consists of removal of 500 ml whole blood weekly, representing a loss of 250 mg iron. In naive patients between 20 to 100 phlebotomies are required to reduce the serum ferritine levels to 50 μg/L. Thereafter, a lifelong maintenance therapy of 3 to 6 phlebotomies yearly is needed.
For absorption, dietary iron ( 70%) is reduced by gastric acid form the ferric (Fe3+) to the ferrous form (Fe2+). Recently, in an observational open study, Hutchinson et al. found that HH patients treated with proton pump inhibitors (PPI) needed fewer phlebotomies, resulting in a drop of 2.5 (SEM 0.25) to 0.5 (SEM 0.25) liter per year.
Read more
Research question: The primary objective is to determine the effectiveness and cost effectiveness of PPI's compared to standard phlebotomy therapy in the prevention of iron overload in HH patients.
Multi-center trial in two hospitals in the South of Limburg (Atrium medical Center, Maastricht university medical center ) and hospital in Belgium (University Hospital Gasthuisberg). The study will be conducted in randomised double blind manner. The follow up will be one year.
Patients are randomized either for the group receiving a PPI or a placebo. Every 2 month the ferritin level is measured and decided if the patient need a phlebotomy (Ferritin >100 µg/L).
- Conditions
- Hemochromatosis
- Being tested
- Pantoprazole
- Medical subject headings
- Hemochromatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PantoprazoleDrug
pantoprazole 40mg 1dd1; 12 months
| Study group | Role | What they receive |
|---|---|---|
| pantoprazol | Experimental | Not described Receives: Drug: Pantoprazole |
| placebo | Placebo comparator | Not described Receives: Drug: Pantoprazole |
What is being measured
The primary outcome is the main question the study is designed to answer.
the total number of phlebotomies for the group taking PPI treatment compared to the group taking placebo will be the primary endpoint of the study.
Measured: 12 months
Secondary outcomes (1)
number of participants with side effects
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 posted2 Feb 2012 (estimated)
- Study startMar 2012
- Primary completionAug 2013 (estimated)
- Study completionAug 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (27)Papers the sponsor has linked to this study.
- Other trials for HemochromatosisStudies of the same condition, open and under way.
- More trials run by Maastricht University Medical CenterThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Maastricht University Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Annadal stichting
- Responsible party
- Sponsor
- Investigators
- G Koek, Dr, Principal investigator, Maastricht University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01524757
- Sponsor's protocol ID
- NL3364409612
- Acronym
- He-ppi
- Status last verified
- Jan 2012
- First submitted
- 31 Jan 2012
- Last updated
- 2 Feb 2012
- Results posted
- No
- Data monitoring committee
- No
Publications
Hutchinson C, Geissler CA, Powell JJ, Bomford A. Proton pump inhibitors suppress absorption of dietary non-haem iron in hereditary haemochromatosis. Gut. 2007 Sep;56(9):1291-5. doi: 10.1136/gut.2006.108613. Epub 2007 Mar 7.
PubMed 17344278Adams P, Brissot P, Powell LW. EASL International Consensus Conference on Haemochromatosis. J Hepatol. 2000 Sep;33(3):485-504. doi: 10.1016/s0168-8278(01)80874-6. No abstract available.
PubMed 11020008Pietrangelo A. Hereditary hemochromatosis. Biochim Biophys Acta. 2006 Jul;1763(7):700-10. doi: 10.1016/j.bbamcr.2006.05.013. Epub 2006 May 27.
PubMed 16891003van der Plas SM, Hansen BE, de Boer JB, Stijnen T, Passchier J, de Man RA, Schalm SW. Generic and disease-specific health related quality of life of liver patients with various aetiologies: a survey. Qual Life Res. 2007 Apr;16(3):375-88. doi: 10.1007/s11136-006-9131-y. Epub 2006 Nov 25.
PubMed 17334830European Association For The Study Of The Liver. EASL clinical practice guidelines for HFE hemochromatosis. J Hepatol. 2010 Jul;53(1):3-22. doi: 10.1016/j.jhep.2010.03.001. Epub 2010 Apr 18.
PubMed 20471131Jacobs EM, Meulendijks CF, Elving L, van der Wilt GJ, Swinkels DW. Impact of the introduction of a guideline on the targeted detection of hereditary haemochromatosis. Neth J Med. 2005 Jun;63(6):205-14.
PubMed 16011012Swinkels DW, Janssen MC, Bergmans J, Marx JJ. Hereditary hemochromatosis: genetic complexity and new diagnostic approaches. Clin Chem. 2006 Jun;52(6):950-68. doi: 10.1373/clinchem.2006.068684. Epub 2006 Apr 20.
PubMed 16627556Rombout-Sestrienkova E, Nieman FH, Essers BA, van Noord PA, Janssen MC, van Deursen CT, Bos LP, Rombout F, van den Braak R, de Leeuw PW, Koek GH. Erythrocytapheresis versus phlebotomy in the initial treatment of HFE hemochromatosis patients: results from a randomized trial. Transfusion. 2012 Mar;52(3):470-7. doi: 10.1111/j.1537-2995.2011.03292.x. Epub 2011 Aug 16.
PubMed 21848963Hungin AP, Rubin G, O'Flanagan H. Factors influencing compliance in long-term proton pump inhibitor therapy in general practice. Br J Gen Pract. 1999 Jun;49(443):463-4.
PubMed 10562747Hicken BL, Tucker DC, Barton JC. Patient compliance with phlebotomy therapy for iron overload associated with hemochromatosis. Am J Gastroenterol. 2003 Sep;98(9):2072-7. doi: 10.1111/j.1572-0241.2003.07292.x.
PubMed 14499790Bonapace ES, Fisher RS, Parkman HP. Does fasting serum gastrin predict gastric acid suppression in patients on proton-pump inhibitors? Dig Dis Sci. 2000 Jan;45(1):34-9. doi: 10.1023/a:1005496907686.
PubMed 10695611Newman BH. Blood donor complications after whole-blood donation. Curr Opin Hematol. 2004 Sep;11(5):339-45. doi: 10.1097/01.moh.0000142105.21058.96.
PubMed 15666658HOFSTETTER JR. [Risks in blood-letting in therapy of hemochromatosis]. Gastroenterologia. 1957;87(3-4):186-91. No abstract available. French.
PubMed 13448264Klinkenberg-Knol EC, Festen HP, Jansen JB, Lamers CB, Nelis F, Snel P, Luckers A, Dekkers CP, Havu N, Meuwissen SG. Long-term treatment with omeprazole for refractory reflux esophagitis: efficacy and safety. Ann Intern Med. 1994 Aug 1;121(3):161-7. doi: 10.7326/0003-4819-121-3-199408010-00001.
PubMed 8017742Klinkenberg-Knol EC, Nelis F, Dent J, Snel P, Mitchell B, Prichard P, Lloyd D, Havu N, Frame MH, Roman J, Walan A; Long-Term Study Group. Long-term omeprazole treatment in resistant gastroesophageal reflux disease: efficacy, safety, and influence on gastric mucosa. Gastroenterology. 2000 Apr;118(4):661-9. doi: 10.1016/s0016-5085(00)70135-1.
PubMed 10734017
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Proton Pump Inhibitors in the Prevention of Iron Reaccumulation in Patient With Hereditary Hemochromatosis
- ClinicalTrials.gov ID
- NCT01524757
- Acronym / sponsor ID
- He-ppi / NL3364409612
- Conditions
- Hemochromatosis
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Maastricht University Medical CenterUniversity, hospital or other
- Collaborators
- Annadal stichting
- Study start
- Mar 2012
- Primary completion
- Aug 2013 (estimated)
- Study completion
- Aug 2013 (estimated)
- First posted
- 2 Feb 2012 (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 causing damage of different important organs like heart, liver, pancreas and joints. Complications and symptoms can regress by intensive treatment reducing the iron overload stores.Different genes have been identified playing a role in the pathophysiology of iron overload. A clinically important HFE gene mutation is the C282Y, located on chromosome 6. Phlebotomy is currently the standard therapy which consists of removal of 500 ml whole blood weekly, representing a loss of 250 mg iron. In naive patients between 20 to 100 phlebotomies are required to reduce the serum ferritine levels to 50 μg/L. Thereafter, a lifelong maintenance therapy of 3 to 6 phlebotomies yearly is needed.
For absorption, dietary iron ( 70%) is reduced by gastric acid form the ferric (Fe3+) to the ferrous form (Fe2+). Recently, in an observational open study, Hutchinson et al. found that HH patients treated with proton pump inhibitors (PPI) needed fewer phlebotomies, resulting in a drop of 2.5 (SEM 0.25) to 0.5 (SEM 0.25) liter per year.
Research question: The primary objective is to determine the effectiveness and cost effectiveness of PPI's compared to standard phlebotomy therapy in the prevention of iron overload in HH patients.
Multi-center trial in two hospitals in the South of Limburg (Atrium medical Center, Maastricht university medical center ) and hospital in Belgium (University Hospital Gasthuisberg). The study will be conducted in randomised double blind manner. The follow up will be one year.
Patients are randomized either for the group receiving a PPI or a placebo. Every 2 month the ferritin level is measured and decided if the patient need a phlebotomy (Ferritin >100 µg/L).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| pantoprazol | Experimental | Drug: Pantoprazole |
| placebo | Placebo comparator | Drug: Pantoprazole |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Patients with hereditary hemochromatosis (HH), homozygous for C282Y, currently treated with phlebotomy as maintenance therapy for at least 12 months with ≥ 3 phlebotomies per year.
- Ferritin level between 50-100 μg/L at start of the inclusion.
- Age: 18 years- 60 years and weight > 50 kg.
Key exclusion criteria (8 listed)
- Patients receiving other therapies such as chelating therapy or forced dietary regimen.
- Patients younger than 18 years.
- HH patients with excessive overweight (BMI > 35).
- Patients who are mentally incapacitated.
- Women being pregnant or expecting/ planning to become pregnant during the one year period of the study.
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 |
|---|---|---|
| Pantoprazolepantoprazole 40mg 1dd1; 12 months | Drug | pantoprazol, placebo |
Primary outcome
the total number of phlebotomies for the group taking PPI treatment compared to the group taking placebo will be the primary endpoint of the study.
Time frame: 12 months
1 secondary outcome
number of participants with side effects
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 2 Feb 2012.
- Background: PubMed 17344278Hutchinson C, Geissler CA, Powell JJ, Bomford A. Proton pump inhibitors suppress absorption of dietary non-haem iron in hereditary haemochromatosis. Gut. 2007 Sep;56(9):1291-5. doi: 10.1136/gut.2006.108613. Epub 2007 Mar 7.
- Background: PubMed 11020008Adams P, Brissot P, Powell LW. EASL International Consensus Conference on Haemochromatosis. J Hepatol. 2000 Sep;33(3):485-504. doi: 10.1016/s0168-8278(01)80874-6. No abstract available.
- Background: PubMed 16891003Pietrangelo A. Hereditary hemochromatosis. Biochim Biophys Acta. 2006 Jul;1763(7):700-10. doi: 10.1016/j.bbamcr.2006.05.013. Epub 2006 May 27.
- Background: PubMed 17334830van der Plas SM, Hansen BE, de Boer JB, Stijnen T, Passchier J, de Man RA, Schalm SW. Generic and disease-specific health related quality of life of liver patients with various aetiologies: a survey. Qual Life Res. 2007 Apr;16(3):375-88. doi: 10.1007/s11136-006-9131-y. Epub 2006 Nov 25.
- Background: PubMed 20471131European Association For The Study Of The Liver. EASL clinical practice guidelines for HFE hemochromatosis. J Hepatol. 2010 Jul;53(1):3-22. doi: 10.1016/j.jhep.2010.03.001. Epub 2010 Apr 18.
- Background: PubMed 16011012Jacobs EM, Meulendijks CF, Elving L, van der Wilt GJ, Swinkels DW. Impact of the introduction of a guideline on the targeted detection of hereditary haemochromatosis. Neth J Med. 2005 Jun;63(6):205-14.
- Background: PubMed 16627556Swinkels DW, Janssen MC, Bergmans J, Marx JJ. Hereditary hemochromatosis: genetic complexity and new diagnostic approaches. Clin Chem. 2006 Jun;52(6):950-68. doi: 10.1373/clinchem.2006.068684. Epub 2006 Apr 20.
- Background: PubMed 21848963Rombout-Sestrienkova E, Nieman FH, Essers BA, van Noord PA, Janssen MC, van Deursen CT, Bos LP, Rombout F, van den Braak R, de Leeuw PW, Koek GH. Erythrocytapheresis versus phlebotomy in the initial treatment of HFE hemochromatosis patients: results from a randomized trial. Transfusion. 2012 Mar;52(3):470-7. doi: 10.1111/j.1537-2995.2011.03292.x. Epub 2011 Aug 16.
- Background: PubMed 10562747Hungin AP, Rubin G, O'Flanagan H. Factors influencing compliance in long-term proton pump inhibitor therapy in general practice. Br J Gen Pract. 1999 Jun;49(443):463-4.
- Background: PubMed 14499790Hicken BL, Tucker DC, Barton JC. Patient compliance with phlebotomy therapy for iron overload associated with hemochromatosis. Am J Gastroenterol. 2003 Sep;98(9):2072-7. doi: 10.1111/j.1572-0241.2003.07292.x.
- Background: PubMed 10695611Bonapace ES, Fisher RS, Parkman HP. Does fasting serum gastrin predict gastric acid suppression in patients on proton-pump inhibitors? Dig Dis Sci. 2000 Jan;45(1):34-9. doi: 10.1023/a:1005496907686.
- Background: PubMed 15666658Newman BH. Blood donor complications after whole-blood donation. Curr Opin Hematol. 2004 Sep;11(5):339-45. doi: 10.1097/01.moh.0000142105.21058.96.
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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.
G Koek, Dr principal investigator
Maastricht University Medical Center
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Pantoprazole in people with Hemochromatosis, run by Maastricht University Medical Center at 3 sites in 2 countries.
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 8 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 2 Feb 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Netherlands, 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. Some participants in this study may receive a placebo. 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?
Contact G Koek, dr (+31-43-3875021, gh.koek@mumc.nl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
the total number of phlebotomies for the group taking PPI treatment compared to the group taking placebo will be the primary endpoint of the study. (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01524757, which also lists 27 linked publications.
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 2 Feb 2012.