Treatment of Refractory Hemochromatosis Rheumatism by Anakinra: a Preliminary Phase II Study

NCT02263638 | Terminated Phase 2

• 2 other identifiers: 2014-000609-1035RC13_8811_THERA
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 3

Brief Summary

Treatment of refractory hemochromatosis rheumatism by Anakinra. Prospective, multicenter, non-randomised, single-arm, open-label, phase II trial.

Conditions

Refractory Hemochromatosis Rheumatism

Keywords

hereditary haemochromatosis; rheumatism; anakinra

Outcome Measures

Primary Outcomes (1)

• 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)

  Day 15

Secondary Outcomes (22)

• Assessment of the disease activity

  Day 0, day 15, day 30, day 60, day 90

• Assessment of the number of painful joints

  Day 0, day 15, day 30, day 60, day 90

• Assessment of the number of swollen joints

  Day 0, day 15, day 30, day 60, day 90

• Assessment of analgesics consumption

  Day 0, day 15, day 30, day 60, day 90

• Assessment of non-steroidal anti-inflammatory drugs (NSAID) consumption

  Day 0, day 15, day 30, day 60, day 90

Study Arms (1)

Anakinra

EXPERIMENTAL

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

Interventions

Anakinra DRUG

One daily subcutaneous injection of a fixed dose of 100 mg will be administered at a fixed time during a five day period

Anakinra

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with age equal to or over 18 years old,
• Patients with proved hereditary hemochromatosis with homozygosity for the C282Y mutation of the HFE gene,
• Patients with rheumatism related to hemochromatosis, considered by the rheumatologist refractory to usual treatment defined by a persistent painful symptomatology despite a treatment of at least one month with level 2 analgesics (weak opioids) at maximal dose, NSAID, colchicine, steroid injection or a combination of these treatments,
• Patients with pain \> 40/100mm measured by VAS (pain of the last 48 hours),
• Effective contraception to be used during treatment and until 48h after the last administration for women of reproductive age,
• Patients who have given written informed consent.

You may not qualify if:

• Other cause of inflammatory rheumatisms such as Rheumatoid Arthritis, Spondyloarthropathies, psoriatic arthritis, or systemic diseases,
• Ongoing treatment with Methotrexate, Hydroxychloroquine, biologic or immunosuppressive drugs
• Malignant pathology, monoclonal gammopathy,
• Intolerance to anakinra,
• Contraindications to the use of anakinra: pregnancy or breastfeeding, hypersensitivity to any of the excipients or to proteins from E. coli, severe renal impairment (creatinine clearance \<30 ml / minute), neutropenia (neutrophil count \<1.5 x 109 / l), ongoing infection
• Patients that cannot follow the protocol,
• Persons subject to major legal protection (safeguarding justice, guardianship, trusteeship), persons deprived of liberty.

Sponsors & Collaborators

• Rennes University Hospital: lead

Study Sites (3)

CHRU de Lille

Lille, France

Location

Groupe Hospitalier Lariboisière

Paris, France

Location

Rennes University Hospital

Rennes, 35033, France

Location

MeSH Terms

Conditions

Hemochromatosis; Rheumatic Diseases

Interventions

Interleukin 1 Receptor Antagonist Protein

Condition Hierarchy (Ancestors)

Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Iron Overload; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Musculoskeletal Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Pascal Richette

  Groupe Hospitalier Lariboisière - Paris

  PRINCIPAL INVESTIGATOR

• Pascal Guggenbuhl

  Rennes University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2014
• First Posted: October 13, 2014
• Study Start: February 4, 2015
• Primary Completion: September 4, 2018
• Study Completion: September 4, 2018
• Last Updated: March 12, 2019

Record last verified: 2019-03

Locations

FR (3)