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HaemochromatosisQuali-SATNCT04779593

Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis

A study testing Clinical examination, SF36 questionnaire and AIMS2_SF questionnaire in people with Haemochromatosis, run by Rennes University Hospital at 12 sites in France.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

Patients in maintenance treatment for HFE hemochromatosis since at least one year will be included in a two year study period and randomized in two groups experimental and control group. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Haemochromatosis
What is being tested?
Clinical examination, SF36 questionnaire and AIMS2_SF questionnaire
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
239 participants
Where
12 sitesFrance
Study dates
2 Jul 2021to Jan 2027
Phase
Interventional
Are there results?
Not yet: the study is under way

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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • - Patients treated with iron chelators;
  • Patients treated with erythroid growth factors (erythropoietin);
  • Patient with excessive alcohol consumption (> 20g/day and > 30 g/day for women and men respectively);
  • Patients with chronic haematological condition;
  • Patients having uncontrolled chronic blood loss (of digestive or gynaecological origin);
  • Patients with chronic kidney failure;
Show all 13Show fewer
  • Patients with a diagnosis of cancer or history of cancer in the last year;
  • Pregnancy or breast feeding.
  • Patient who are included in another research protocol
  • Adults legally protected (judicial protection, guardianship, or supervision), persons deprived of their freedom.
  • with C282Y homozygous HFE hemochromatosis;
  • having finished the initial phase of HFE hemochromatosis treatment and in maintenance treatment for at least one year;
  • having signed an informed consent form.

Who cannot join

  • Patients treated with iron chelators;
  • Patients treated with erythroid growth factors (erythropoietin);
  • Patient with excessive alcohol consumption (> 20g/day and > 30 g/day for women and men respectively);
  • Patients with chronic haematological condition;
  • Patients having uncontrolled chronic blood loss (of digestive or gynaecological origin);
  • Patients with chronic kidney failure;
Show all 10Show fewer
  • Patients with a diagnosis of cancer or history of cancer in the last year;
  • Pregnancy or breast feeding.
  • Patient who are included in another research protocol
  • Adults legally protected (judicial protection, guardianship, or supervision), persons deprived of their freedom.

Trial locations and contacts

12 sites in 1 country.

12 sites in 1 country

France12 sites

  • Hopital Avicenne

    Bobigny

  • CHU Dupuytren

    Limoges

  • GHBS site du Scorff

    Lorient

  • GHRMSA - Hôpital Emile Muller

    Mulhouse

  • CHR Orléans

    Orléans

  • Hôpital Européen Georges Pompidou

    Paris, 75908

  • CHU Rennes

    Rennes

  • CH Yves le Foll

    Saint-Brieuc

  • CH de St Malo

    St-Malo

  • Hôpital Rangueil

    Toulouse

  • Centre hospitalier Bretagne Atlantique

    Vannes

  • Hôpital Paul Brousse

    Villejuif

Other trials in France

About this study

Patients in maintenance treatment for HFE hemochromatosis since at least one year will be included in a two year study period and randomized in two groups experimental and control group. Because proton pump inhibitors are widely used as chronic medication, and because they can significantly modify iron absorption, patients will be stratified according to the use of proton pump inhibitors and gender. A first bloodletting will be performed at inclusion with the same volume as usually performed by the patients. Results of the biological test performed at this visit will guide the time schedule and volume of the next bloodletting according to randomization group (patients treated with bloodletting according to current guidelines "ferritin alone" versus patients treated with bloodletting according to "transferrin saturation and serum ferritin").Blood count and iron metabolism parameters will be performed at each bloodletting and follow-up visits. Time schedule and volume of bloodletting will be adjusted to biological results after each follow-up visit. Volume and schedule for bloodlettings will be determined according to guidelines specifically designed for this study to assure harmonization of treatment management, and centrally validated through the recording of the biological tests in the electronic Case Report Form which will provide the investigator with the volume and schedule of the next bloodletting. There will be two ways of treatment modification: either change of schedule or volume of bloodletting. Patients will undergo follow-up visit every six months with clinical examination, questionnaires at J0, M12 and M24 (SF-36; AIMS2-SF, WOMAC, EQ-5D-5L), and biological test. For health economics analysis, data will be obtained thanks to a dedicated extraction from SNDS database SNDS database will allow to gather hospital stays, visits, and other healthcare-related costs as well as vital status (date (month/year) of death) and cause of death. A de-identified copy of the clinical database, restricted to the relevant variables, will be sent for semideterministic matching purpose with SNDS extraction using four key variables: gender, same date (month/year) of birth, same date (day/month/year) of visit for bloodletting; pending, of course, regulatory authorization.

Being tested
Clinical examination, SF36 questionnaire, AIMS2_SF questionnaire, WOMAC questionnaire, EQ-5D-5L questionnaire
Medical subject headings
Hemochromatosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Clinical examinationOther

    Clinical data will be recorded (general clinical examination, height, weight, blood pressure,heart beat, alcohol and tobacco consumption, antecedent) as well as concurrent medication at each follow-up visit.

  • SF36 questionnaireOther

    At D0, M12 and M24. This 36 item patient reported survey of patient's health is the most commonly used and validated health survey instrument for appraising quality of life.

    Items are grouped in 8 scaled scores exploring multiple dimension of global health (vitality, physical functioning, bodily pain, general health, perceptions, physical role functioning, emotional role functioning, social role functioning, mental health).

    Scoring will be performed as recommended by the SF-36 instruction manual to create the eight scale scores. Furthermore, these subscales sum to obtain the total SF-36 score and will be summarized into two composite scores (physical and mental quality of life).

  • AIMS2_SF questionnaireOther

    At D0, M12 and M24. The Arthritis Impact Measurement Scales 2 Short Form (AIMS2-SF) is a specific tool to measure changes in global health, pain, mobility and social function in patients with arthritis. It was described in 1992 in patients with rheumatoid arthritis and osteoarthritis and include 26 items that are summarized in scales according to a predefined scoring system: mobility, physical activity (walking, bending, lifting), dexterity, household activity (managing money and medications, housekeeping), social activities, activities of daily living, pain, depression, and anxiety. The French translation has been validated and this questionnaires has been widely used in the rheumatology field to assess quality of life of patients with arthritis. Because HFE related arthropathy is very similar to osteoarthritis this questionnaire is ought to be adequate in this setting.

  • WOMAC questionnaireOther

    At D0, M12 and M24. The Western Ontario and McMaster Universities Arthritis Index (WOMAC) is a widely used questionnaires specifically assessing lower limb (hips and knee) osteoarthritis. It measures five items for pain, two for stiffness and 17 for functional limitation and had been translated in French.

  • EQ-5D-5L questionnaireOther

    At D0, M12 and M24. The EQ-5D is a standardized instrument which evaluates the generic quality of life (http://www.euroqol.org/). It is a preference-based health-related quality of life measure with one question for each of the five dimensions that include mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The answers given to EQ-5D permit to find 243 health states that can be converted into utility score anchored at 0 for death and 1 for perfect health. EQ-5D is an instrument developed in Europe, widely used in cost-utility analysis. It has been validated in a representative sample of French population.

  • Blood Sample Complete blood countBiological

    Blood Sample Complete blood count at D0, M6, M12, M18 and M24/end follow-up visit

  • Blood Sample Iron panelBiological

    Blood Sample Iron panel (serum ferritin, serum iron and serum transferrin to determine transferrin saturation according to randomization group (at M6, M12 and M18) at D0, M6, M12, M18 and M24/end follow-up visit and at each bloodletting.

  • Blood Sample Fasting GlucoseBiological

    Blood Sample Fasting Glucose at D0 and M24/end follow-up visit

  • Blood sample lipid panelBiological

    Blood sample lipid panel (total cholesterol, triglycerides, HDL, LDL) at D0 and M24/end follow-up visit

  • Blood sample liver panelBiological

    Blood sample liver panel (total bilirubin, Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Gamma-Glutamyl Transferase) at D0 and M24/end follow-up visit

  • Blood sample C reactive proteinBiological

    Blood sample C reactive protein at D0 and M24/end follow-up visit

  • Bloodletting - control groupProcedure or surgery

    Patients treated with bloodletting according to current guidelines (ferritin alone). Patient will undergo bloodletting with a goal of maintaining a serum ferritin equal or lower than 50 g/L according to current clinical practice guidelines (French and European).A first bloodletting will be performed at inclusion with the same volume as usually performed by the patients.Results of the biological test performed at this visit will guide the time schedule and volume of the next bloodletting according to randomization group. Time schedule and volume of bloodletting will be adjusted to biological results after each follow-up visit.

  • BioBankBiological

    Blood sample will be collected for BioBank at D0, M12 and M24.

  • Medico-economicalOther

    Medico-economic data will be collected at each follow up visit.

  • Bloodletting - experimental groupProcedure or surgery

    Patients treated with bloodletting according to "transferrin saturation and serum ferritin". Patients will undergo bloodletting with a goal of maintaining a serum transferrin saturation equal or lower than 50 % and a serum ferritin lower than the upper limit of the normal range (300 g/L for men and 200 g/L for women).A first bloodletting will be performed at inclusion with the same volume as usually performed by the patients.Results of the biological test performed at this visit will guide the time schedule and volume of the next bloodletting according to randomization group. Time schedule and volume of bloodletting will be adjusted to biological results after each follow-up visit.

Study groups in NCT04779593
Study groupRoleWhat they receive
experimental groupExperimentalPatients treated with bloodletting according to "transferrin saturation and serum ferritin".
Receives: Other: Clinical examination; Other: SF36 questionnaire; Other: AIMS2_SF questionnaire; Other: WOMAC questionnaire; Other: EQ-5D-5L questionnaire; Biological: Blood Sample Complete blood count; Biological: Blood Sample Iron panel; Biological: Blood Sample Fasting Glucose; Biological: Blood sample lipid panel; Biological: Blood sample liver panel; Biological: Blood sample C reactive protein; Biological: BioBank; Other: Medico-economical; Procedure: Bloodletting - experimental group
control groupActive comparatorPatients treated with bloodletting according to current guidelines "ferritin alone"
Receives: Other: Clinical examination; Other: SF36 questionnaire; Other: AIMS2_SF questionnaire; Other: WOMAC questionnaire; Other: EQ-5D-5L questionnaire; Biological: Blood Sample Complete blood count; Biological: Blood Sample Iron panel; Biological: Blood Sample Fasting Glucose; Biological: Blood sample lipid panel; Biological: Blood sample liver panel; Biological: Blood sample C reactive protein; Procedure: Bloodletting - control group; Biological: BioBank; Other: Medico-economical

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The primary evaluation criteria is the Physical Component Score of the SF-36 questionnaire at the end of the study period (two years)

    The primary evaluation criteria is the Physical Component Score of the SF-36 questionnaire at the end of the study period (two years). Physical Component score of the SF-36 has been chosen because SF-36 it is a widely validated and reproducible questionnaire, and this component is best susceptible to reflect both fatigue and joint involvement.

    Measured: At Month 24

Secondary outcomes (11)

  1. the AIMS2- SF (short form of the Arthritis Impact Measurement Scales 2) questionnaire

    Assessment of Arthropathy and joint pain related quality of life as assessed by the AIMS2- SF (short form of the Arthritis Impact Measurement Scales 2) questionnaire throughout the study, as performed at J0, M12 and M24 follow-up visit (every 12 months).

    Measured: at Day 0, Month 12 and Month 24 follow-up visit

  2. the WOMAC (Western Ontario and McMaster Universities Arthritis Index) questionnaire

    Assessment of Arthropathy and joint pain related quality of life as assessed by the WOMAC (Western Ontario and McMaster Universities Arthritis Index) questionnaire throughout the study, as performed at J0, M12 and M24 follow-up visit (every 12 months).

    Measured: at Day 0, Month 12 and Month 24 follow-up visit

  3. Evolution of the Mental Component Score of the SF-36 questionnaire

    Evolution of the Mental Component Score of the SF-36 questionnaire between inclusion and the end of the study period (two years).

    Measured: At Month 24

  4. Evolution of the Physical Component Score of the SF-36

    Evolution of the Physical Component Score of the SF-36 throughout the study, as performed at J0, M12 and M24 follow-up visit (every 12 months).

    Measured: at Day 0, Month 12 and Month 24 follow-up visit

  5. Evolution of the Mental Component Score of the SF-36

    Evolution of the Mental Component Score of the SF-36 throughout the study, as performed at J0, M12 and M24 follow-up visit (every 12 months).

    Measured: at Day 0, Month 12 and Month 24 follow-up visit

  6. Evolution of of the EQ-5D-5L score

    Evolution of of the EQ-5D-5L score throughout the study, as performed at J0, M12 and M24 follow-up visit (every 12 months).

    Measured: at Day 0, Month 12 and Month 24 follow-up visit

  7. Evolution of Serum ferritin and serum transferrin saturation

    Evolution of Serum ferritin and serum transferrin saturation determined at each follow-up visit (every 6 months).

    Measured: Day 0, Month 6, Month 12, Month 18 and Month 24/follow-up visit

  8. Occurrence of anaemia

    Occurrence of anaemia defined as haemoglobin lower than 11g/dL at any follow-up visit.

    Measured: Day 0, Month 6, Month 12, Month 18 and Month 24/follow-up visit

  9. Occurrence of malaise

    Occurrence of malaise after a bloodletting procedure

    Measured: At Month 24

  10. Total number of phlebotomy performed

    Total number of phlebotomy performed by each patient during the study period.

    Measured: At Month 24

  11. Incremental Cost-Effectiveness Ratio

    Incremental Cost-Effectiveness Ratio (ICER) defined as the cost for QALY gained in the "transferrin saturation + ferritin" strategy versus "ferritin alone" strategy.

    Measured: At Month 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Mar 2021
  2. Study start2 Jul 2021
  3. Primary completionJan 2027 (estimated)
  4. Study completionJan 2027 (estimated)

Resources

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
None (open label)
Enrolment
239 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04779593
Sponsor's protocol ID
35RC19_8985_Quali-SAT
Acronym
Quali-SAT
Status last verified
Jun 2026
First submitted
2 Feb 2021
Last updated
12 Jun 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A study testing Clinical examination, SF36 questionnaire and AIMS2_SF questionnaire in people with Haemochromatosis, run by Rennes University Hospital at 12 sites in France.

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 13 inclusion and 10 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: under way, not looking for participants, as of the sponsor's last update on 12 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in France. 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.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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 primary evaluation criteria is the Physical Component Score of the SF-36 questionnaire at the end of the study period (two years) (time frame: At Month 24). 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 NCT04779593.

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 12 Jun 2026.

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