Haemochromatosis Clinical Trial
— Quali-SATOfficial title:
Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis
| Verified date | January 2023 |
| Source | Rennes University Hospital |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Recruiting |
| Enrollment | 240 |
| Est. completion date | January 2027 |
| Est. primary completion date | January 2027 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility | Inclusion Criteria: - - 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; - 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. Exclusion Criteria: - 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; - 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. |
| Country | Name | City | State |
|---|---|---|---|
| France | Hopital Avicenne | Bobigny | |
| France | CHU Dupuytren | Limoges | |
| France | GHBS site du Scorff | Lorient | |
| France | GHRMSA - Hôpital Emile Muller | Mulhouse | |
| France | CHR Orléans | Orléans | |
| France | Hôpital Européen Georges Pompidou | Paris | |
| France | CHU Rennes | Rennes | |
| France | CH Yves le Foll | Saint-Brieuc | |
| France | CH de St Malo | Saint-Malo | |
| France | Hôpital Rangueil | Toulouse | |
| France | Centre hospitalier Bretagne Atlantique | Vannes | |
| France | Hôpital Paul Brousse | Villejuif |
| Lead Sponsor | Collaborator |
|---|---|
| Rennes University Hospital |
France,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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. | At Month 24 | |
| Secondary | 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). | at Day 0, Month 12 and Month 24 follow-up visit | |
| Secondary | 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). | at Day 0, Month 12 and Month 24 follow-up visit | |
| Secondary | 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). | At Month 24 | |
| Secondary | 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). | at Day 0, Month 12 and Month 24 follow-up visit | |
| Secondary | 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). | at Day 0, Month 12 and Month 24 follow-up visit | |
| Secondary | 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). | at Day 0, Month 12 and Month 24 follow-up visit | |
| Secondary | 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). | Day 0, Month 6, Month 12, Month 18 and Month 24/follow-up visit | |
| Secondary | Occurrence of anaemia | Occurrence of anaemia defined as haemoglobin lower than 11g/dL at any follow-up visit. | Day 0, Month 6, Month 12, Month 18 and Month 24/follow-up visit | |
| Secondary | Occurrence of malaise | Occurrence of malaise after a bloodletting procedure | At Month 24 | |
| Secondary | Total number of phlebotomy performed | Total number of phlebotomy performed by each patient during the study period. | At Month 24 | |
| Secondary | 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. | At Month 24 |