- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04779593
Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis (Quali-SAT)
Study Overview
Status
Conditions
Intervention / Treatment
- 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
- Procedure: Bloodletting - control group
Study Type
Enrollment (Anticipated)
Phase
- Not Applicable
Contacts and Locations
Study Locations
-
-
-
Bobigny, France
- Recruiting
- Hôpital Avicenne
-
Contact:
- Nathalie GANNE
- Phone Number: +33 01 48 95 55 55
- Email: nathalie.ganne@aphp.fr
-
Principal Investigator:
- Nathalie GANNE
-
Limoges, France
- Recruiting
- Chu Dupuytren
-
Principal Investigator:
- Véronique LOUSTAUD-RATTI
-
Contact:
- Véronique LOUSTAUD-RATTI
- Phone Number: +33 05. 55. 05. 66. 84
- Email: veronique.loustaud-ratti@unilim.fr
-
Lorient, France
- Recruiting
- GHBS site du Scorff
-
Contact:
- Florent EHRHARD
- Phone Number: +33 02 97 06 94 98
- Email: f.ehrhard@ghbs.bzh
-
Principal Investigator:
- Florent EHRHARD
-
Mulhouse, France
- Recruiting
- GHRMSA - Hôpital Emile MULLER
-
Contact:
- Bernard DRENOU
- Phone Number: +33 03 89 64 77 55
- Email: drenoub@ghrmsa.fr
-
Orléans, France
- Recruiting
- CHR Orléans
-
Contact:
- Xavier CAUSSE
- Phone Number: 02 38 22 96 58
- Email: xavier.causse@chr-orleans.fr
-
Principal Investigator:
- Xavier CAUSSE
-
Paris, France, 75908
- Not yet recruiting
- Hopital Europeen Georges Pompidou
-
Contact:
- Prunelle GETTEN
- Phone Number: +33 01 56 09 53 41
- Email: prunelle.getten@aphp.fr
-
Contact:
- Prunelle GETTEN
-
Rennes, France
- Recruiting
- CHU Rennes
-
Contact:
- Edouard Bardou-Jacquet
- Phone Number: +33 02.99.28.53.08
- Email: edouard.bardou-jacquet@chu-rennes.fr
-
Principal Investigator:
- Edouard Bardou-Jacquet
-
Saint-Brieuc, France
- Recruiting
- CH Yves Le Foll
-
Contact:
- Antonia LE GRUYER
- Phone Number: +33 02 96 01 73 78
- Email: antonia.le-gruyer@ch-stbrieuc.fr
-
Principal Investigator:
- Antonia LE GRUYER
-
Saint-Malo, France
- Recruiting
- CH de St Malo
-
Contact:
- Christine Beusnel
- Phone Number: +33 02 99 21 27 83
- Email: c.beusnel@ch-stmalo.fr
-
Principal Investigator:
- Christine Beusnel
-
Toulouse, France
- Recruiting
- Hopital Rangueil
-
Contact:
- Christophe BUREAU
- Phone Number: +33 05 61 32 36 86
- Email: bureau.c@chu-toulouse.fr
-
Principal Investigator:
- BUREAU
-
Vannes, France
- Recruiting
- Centre Hospitalier Bretagne Atlantique
-
Contact:
- Gaëlle BILLET
- Phone Number: +33 02 97 01 99 01
- Email: gaelle.billet@ch-bretagne-atlantique.fr
-
Principal Investigator:
- Gaëlle BILLET
-
Villejuif, France
- Recruiting
- Hopital Paul Brousse
-
Contact:
- Rodolphe Sobesky
- Phone Number: +33 01 45 59 67 81
- Email: rodolphe.sobesky@aphp.fr
-
Contact:
- Rodolphe Sobesky
-
Principal Investigator:
- Rodolphe Sobesky
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: experimental group
Patients treated with bloodletting according to "transferrin saturation and serum ferritin".
|
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.
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).
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.
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.
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 count at D0, M6, M12, M18 and M24/end follow-up visit
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 Glucose at D0 and M24/end follow-up visit
Blood sample lipid panel (total cholesterol, triglycerides, HDL, LDL) at D0 and M24/end follow-up visit
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 protein at D0 and M24/end follow-up visit
Blood sample will be collected for BioBank at D0, M12 and M24.
Medico-economic data will be collected at each follow up visit.
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.
|
Active Comparator: control group
Patients treated with bloodletting according to current guidelines "ferritin alone"
|
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.
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).
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.
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.
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 count at D0, M6, M12, M18 and M24/end follow-up visit
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 Glucose at D0 and M24/end follow-up visit
Blood sample lipid panel (total cholesterol, triglycerides, HDL, LDL) at D0 and M24/end follow-up visit
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 protein at D0 and M24/end follow-up visit
Blood sample will be collected for BioBank at D0, M12 and M24.
Medico-economic data will be collected at each follow up visit.
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
|
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 Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
the AIMS2- SF (short form of the Arthritis Impact Measurement Scales 2) questionnaire
Time Frame: at Day 0, Month 12 and Month 24 follow-up visit
|
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
|
the WOMAC (Western Ontario and McMaster Universities Arthritis Index) questionnaire
Time Frame: at Day 0, Month 12 and Month 24 follow-up visit
|
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
|
Evolution of the Mental Component Score of the SF-36 questionnaire
Time Frame: At Month 24
|
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
|
Evolution of the Physical Component Score of the SF-36
Time Frame: at Day 0, Month 12 and Month 24 follow-up visit
|
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
|
Evolution of the Mental Component Score of the SF-36
Time Frame: at Day 0, Month 12 and Month 24 follow-up visit
|
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
|
Evolution of of the EQ-5D-5L score
Time Frame: at Day 0, Month 12 and Month 24 follow-up visit
|
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
|
Evolution of Serum ferritin and serum transferrin saturation
Time Frame: Day 0, Month 6, Month 12, Month 18 and Month 24/follow-up visit
|
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
|
Occurrence of malaise
Time Frame: At Month 24
|
Occurrence of malaise after a bloodletting procedure
|
At Month 24
|
Total number of phlebotomy performed
Time Frame: At Month 24
|
Total number of phlebotomy performed by each patient during the study period.
|
At Month 24
|
Incremental Cost-Effectiveness Ratio
Time Frame: At Month 24
|
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
|
Occurrence of anaemia
Time Frame: Day 0, Month 6, Month 12, Month 18 and Month 24/follow-up visit
|
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
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 35RC19_8985_Quali-SAT
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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