A Phase 2 interventional study of Anakinra in Refractory Hemochromatosis Rheumatism, sponsored by Rennes University Hospital. Terminated at 3 sites in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-03-12.
Sponsored by Rennes University Hospital · Phase 2, Interventional, and Treatment
Treatment of refractory hemochromatosis rheumatism by Anakinra. Prospective, multicenter, non-randomised, single-arm, open-label, phase II trial.
Hereditary hemochromatosis (HH) is a genetic disease characterized by tissue iron overload. The most common genotype is homozygosity for the p.Cys282Tyr mutation of the HFE gene (MIM 235200). It is a serious disease which can lead to life threatening complications such as cirrhosis, liver carcinoma, heart failure or diabetes mellitus. Currently, these complications can be prevented by phlebotomies. Two-thirds of patients complain of joint symptoms which represent a major cause of impaired quality of life. Phlebotomies are ineffective on HH rheumatism and patients' quality of life is very often altered while life threatening complications are prevented. Furthermore, there is a significant higher risk for joint replacement surgery in these patients compared to controls (X 9). There is currently no approved treatment for hemochromatosis rheumatism. As it looks like severe osteoarthritis, calcium pyrophosphate deposition disease (CPDD) or chondrocalcinosis, symptomatic treatments are employed such as analgesics (type I or II), non-steroidal anti-inflammatory drugs or colchicine in case of acute joint flare, corticosteroids intra-articular injections or occasionally oral glucocorticoids. However in some cases these treatments remain ineffective leading to a true disability.
Frequently, there are local inflammatory symptoms. Interleukin 1ß (IL1ß) plays a key role in the pathogenesis of crystal arthropathies (CPDD or gout).
Anakinra (IL-1Ra), a drug approved in France for rheumatoid arthritis, has been tested in short series or case controls in refractory gout, CPDD and only in two patients with HH rheumatism. The aim of this phase II study is to test the efficacy of anakinra in patients with hemochromatosis and refractory joint pain. It is also to evaluate the opportunity to perform a phase III trial. In the absence of available data on the evolution of this rheumatism treated by anakinra in this population of patients resistant to standard therapy, the investigators consider that a phase III trial would not be justifiable if the rate of success is insufficient.
377 studies on the registry are indexed under Rheumatic Diseases; 79 are open to participants now.
This study's enrollment of 17 is below the median of 74 across 210 interventional studies indexed under Rheumatic Diseases.
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Exclusion Criteria:
One daily subcutaneous injection of a fixed dose of 100 mg will be administered at a fixed time by a nurse during a five day period
Drug: Anakinra
One daily subcutaneous injection of a fixed dose of 100 mg will be administered at a fixed time during a five day period
Rate of patients with improvement of joint pain
Improvement is defined as the minimal clinically important improvement of joint pain and is assessed on a 0-100 mm visual analogue scale (VAS)
Time frame: Day 15
Assessment of the disease activity
Assessment of the disease activity by Visual analog scale (VAS)
Time frame: Day 0, day 15, day 30, day 60, day 90
Assessment of the number of painful joints
Assessment of the number of painful joints by a clinical exam
Time frame: Day 0, day 15, day 30, day 60, day 90
Assessment of the number of swollen joints
Assessment of the number of swollen joints by a clinical exam
Time frame: Day 0, day 15, day 30, day 60, day 90
Assessment of analgesics consumption
Time frame: Day 0, day 15, day 30, day 60, day 90
Assessment of non-steroidal anti-inflammatory drugs (NSAID) consumption
Time frame: Day 0, day 15, day 30, day 60, day 90
Assessment of colchicine consumption
Time frame: Day 0, day 15, day 30, day 60, day 90
Assessment of steroids injections consumption
Time frame: Day 0, day 15, day 30, day 60, day 90
Assessment of the quality of life
Assessment of the quality of life by the SF36 questionnaire
Time frame: Day 0, day 15, day 30, day 90
Assessment of the quality of life
Assessment of the quality of life by the HAQ questionnaire
Time frame: Day 0, day 15, day 30, day 90
Functional evaluation
Functional evaluation by WOMAC index for hip and knee
Time frame: Day 0, day 15, day 30, day 90
Functional evaluation
Functional evaluation by Dreiser index for hands
Time frame: Day 0, day 15, day 30, day 90
Assessment of joint damage
Assessment of joint damage by X-rays and Doppler ultrasound
Time frame: Day 0, day 90
Synovial fluid analysis
Puncture if acute joint effusion : cells count
Time frame: 3 months
Synovial fluid analysis
Puncture if acute joint effusion : search for crystals presence
Time frame: 3 months
Synovial fluid analysis
Puncture if acute joint effusion : iron parameters markers
Time frame: 3 months
Biological effects on inflammation and iron metabolism
Biological/Vaccine : iron and inflammatory markers
Time frame: Day 0, day 15, day 30, day 60, day 90
Time at which Cmax of anakinra was observed (Tmax)
Pharmacokinetics study
Time frame: Predose, 0.25, 0.5, 0.75, 1, 2, 3, 4, 6, 8, 10, 12, 15, 18, 21, 24 hours post-dose
Maximum observed concentration (Cmax) of anakinra
Pharmacokinetics study
Time frame: Predose, 0.25, 0.5, 0.75, 1, 2, 3, 4, 6, 8, 10, 12, 15, 18, 21, 24 hours post-dose
Half-life (T1/2) of anakinra
Pharmacokinetics study
Time frame: Predose, 0.25, 0.5, 0.75, 1, 2, 3, 4, 6, 8, 10, 12, 15, 18, 21, 24 hours post-dose
Area under the concentration-time curve of time 0 to the last detectable concentration (AUC0-last) of anakinra
Pharmacokinetics study
Time frame: Predose, 0.25, 0.5, 0.75, 1, 2, 3, 4, 6, 8, 10, 12, 15, 18, 21, 24 hours post-dose
Area under the concentration-time curve of time 0 to infinity (AUC0-∞) of anakinra
Pharmacokinetics study
Time frame: Predose, 0.25, 0.5, 0.75, 1, 2, 3, 4, 6, 8, 10, 12, 15, 18, 21, 24 hours post-dose
Plasma clearance after administration (CL/F) of anakinra
Pharmacokinetics study
Time frame: Predose, 0.25, 0.5, 0.75, 1, 2, 3, 4, 6, 8, 10, 12, 15, 18, 21, 24 hours post-dose
This study is terminated, as verified in Mar 2019. You cannot join it, but the record below documents what was studied.
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Rennes University Hospital