Erythrocytapheresis Versus Phlebotomy as Maintenance Therapy in Hereditary Hemochromatosis (HH) Patients

NCT01398644 | Unknown Phase 3

• 1 other identifier: 07-2-104
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

Hereditary hemochromatosis (HH) is a genetic disorder of iron metabolism, resulting in excessive iron overload. Phlebotomy is currently the standard therapy. More recently Therapeutic Erythrocytapheresis (TE) has become a new therapeutic modality, which potentially offers a more efficient method to remove iron overload with fewer procedures.In the proposed clinical trial the investigators will examine whether TE can keep the ferritin levels in patients requiring maintenance therapy below 50 microg/L, with minimally half the number of treatment procedures when compared to current standard therapy by P.

Conditions

Hereditary Hemochromatosis

Keywords

Phlebotomy; Therapeutic erythrocytapheresis; Hereditary Hemochromatosis; maintenance therapy

Outcome Measures

Primary Outcomes (1)

• The difference in number of required treatments and the interval between treatments per year to keep the serum ferritin levels between 30-50 microg/L

  one year after first phlebotomy treatment and one year after first erythrocytapheresis treatment

Study Arms (2)

Phlebotomy -intervention phlebotomy

ACTIVE COMPARATOR

Patients are treated with phlebotomy if ferritin level \>50 ug/l

Other: Phlebotomy and erythrocytapheresis

Erythrocytapheresis

EXPERIMENTAL

Patients are treated with erythrocytapheresis if serum ferritin level \>50ug/l

Other: Phlebotomy and erythrocytapheresis

Interventions

Phlebotomy and erythrocytapheresis OTHER

Phlebotomy- removal of 500 ml whole blood Erythrocytapheresis- removal of 300-800 ml erythrocytes

Erythrocytapheresis; Phlebotomy -intervention phlebotomy

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• homozygous for C282Y
• currently treated with phlebotomy as maintenance therapy for at least 6 month
• ferritin level between 30-50 micog/L
• age 18 years an older
• weight more than 50 kg
• signed informed consent
• willingness to fill out additional questionnaires at three points in time

You may not qualify if:

• chelating therapy
• forced dietary regime
• aged below 18 years
• excessive overweight ( BMI more than 35)
• pregnancy

Sponsors & Collaborators

• Sanquin Research & Blood Bank Divisions: lead
• Maastricht University Medical Center: collaborator
• Atrium Medical Center: collaborator
• Radboud University Medical Center: collaborator
• Orbis Medical Centre: collaborator

Study Sites (1)

University Hospital Maastricht

Maastricht, 6300, Netherlands

Location

MeSH Terms

Conditions

Hemochromatosis

Interventions

Phlebotomy

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

Intervention Hierarchy (Ancestors)

Blood Specimen Collection; Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Therapeutics; Surgical Procedures, Operative; Investigative Techniques

Study Officials

• Eva Rombout, MD

  Sanquin Blood Supply

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Transfusion specialist

Study Record Dates

• First Submitted: July 19, 2011
• First Posted: July 20, 2011
• Study Start: May 1, 2008
• Primary Completion: December 1, 2013
• Study Completion: December 1, 2013
• Last Updated: December 4, 2013

Record last verified: 2013-12

Locations

NL (1)