Phlebotomy for Iron Overload

Not currently recruiting at 1 trial location

Overseen By Kamille A West-Mitchell, M.D.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: National Institutes of Health Clinical Center (CC)

Disqualifiers: Pregnancy, Polycythemia vera, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 9

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if a test called MCV, which measures red blood cell size, is more effective than the usual ferritin test for guiding phlebotomy therapy in individuals with hemochromatosis. Hemochromatosis causes excessive iron accumulation in the body, potentially damaging organs like the liver and heart. Participants will undergo regular blood removal sessions to reduce iron levels and may contribute to blood transfusions. The study seeks individuals aged 15 and older who have been diagnosed with hemochromatosis or have very high iron levels suggesting this condition. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that phlebotomy is safe for patients with hemochromatosis?

Research has shown that drawing blood, known as phlebotomy, safely and effectively manages high iron levels in people with hemochromatosis. Studies have found that regular blood draws lower the amount of iron in the body. This is crucial because excess iron can damage organs like the liver, heart, and pancreas.

Phlebotomy is also safe and practical for treating iron overload from other conditions. Patients generally tolerate the treatment well, and it can prevent problems caused by excess iron. Starting regular phlebotomy early in hereditary hemochromatosis can even restore normal life expectancy.

Overall, while the process involves frequent blood draws, the benefits of reducing iron levels make it a well-tolerated and effective treatment for managing iron overload. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Phlebotomy is unique because it directly removes excess iron from the body by drawing blood, which is a straightforward and natural way to manage iron overload in conditions like hereditary hemochromatosis (HH). Unlike other treatments that may involve medication to bind and eliminate iron, phlebotomy works by reducing the iron stores more directly and naturally. Researchers are excited about this treatment because it is a non-invasive procedure that can effectively lower iron levels without the need for additional drugs, minimizing potential side effects.

What evidence suggests that this trial's treatments could be effective for hemochromatosis?

Research has shown that drawing blood, known as phlebotomy, effectively treats hemochromatosis. This condition causes excessive iron accumulation in the body, and phlebotomy helps by removing the extra iron from the blood. Studies have found that phlebotomy can correct iron overload and prevent damage to organs like the liver and heart if performed before the damage becomes permanent. In one study, almost all patients successfully reduced their iron levels, demonstrating the treatment's effectiveness. Another study found that other treatments, such as medicines that bind to iron, were less effective than phlebotomy. ^{4 5 6 7 8}

Who Is on the Research Team?

Kamille A West-Mitchell, M.D.

Principal Investigator

National Institutes of Health Clinical Center (CC)

Are You a Good Fit for This Trial?

This trial is for individuals aged 15 or older with diagnosed hemochromatosis, a condition where too much iron is absorbed by the body. It's also open to those with high iron levels suggesting hemochromatosis, and family members being screened for it. People can't join if they're under 15, need phlebotomy for other conditions like polycythemia vera, have certain illnesses that increase risk, are pregnant, or have iron overload not caused by hemochromatosis.

Inclusion Criteria

My iron levels are high, but I don’t have a confirmed diagnosis of hemochromatosis.

My iron levels are high, but I don't have the specific gene changes. A liver biopsy showed high iron.

My family's health history for certain genetic conditions is unknown.

Exclusion Criteria

My iron overload is not caused by hereditary hemochromatosis.

Pregnancy

Other medical illness or condition which, in the opinion of the Investigators, may contraindicate participation due to risk to patient or to Donor Center

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Initial Phlebotomy Treatment

Participants undergo weekly phlebotomy treatments to deplete iron stores, requiring 10 to 50 treatments

10 to 50 weeks

Weekly visits (in-person)

Maintenance Phlebotomy

Blood is drawn every 8 to 12 weeks to prevent re-build up of iron

Ongoing

Every 8 to 12 weeks (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including evaluation of liver and arthritis symptoms

4 to 12 months

What Are the Treatments Tested in This Trial?

Interventions

• Phlebotomy

Trial Overview The study tests whether MCV (a measure of red blood cell size) can guide phlebotomy therapy as effectively as the ferritin test in patients with hemochromatosis. Participants will undergo regular blood drawing sessions to manage their iron levels and may contribute blood for transfusions. Some may be evaluated further for liver disease or arthritis linked to their condition.

How Is the Trial Designed?

Group I: 1/HH patients Experimental Treatment 1 Intervention

HH patients

Who Is Running the Clinical Trial?

National Institutes of Health Clinical Center (CC)

Lead Sponsor

Trials

391

Recruited

30,880,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10884378/

Health Economic Evaluations of Hemochromatosis ... Therapeutic phlebotomy aims to reduce serum iron indices and iron overload [14]. Removing the excess iron before severe tissue damage ...

2. nature.com nature.com/articles/s41598-025-01421-4

A phase 1b randomised clinical trial evaluating BBI-001, ... Up to 40% of subjects with HH may absorb in excess of 3–5 mg of iron per day and develop significant iron overload as a result of dysfunctional ...

3. jamanetwork.com jamanetwork.com/journals/jamanetworkopen/fullarticle/2810985

Testing and Management of Iron Overload After Genetic ... Second, phlebotomy is known to correct iron overload and resulting organ damage if initiated before irreversibility. Based on our study results, ...

4. cghjournal.org cghjournal.org/article/S1542-3565(24)01136-4/fulltext

Diagnosis and Treatment of Hemochromatosis Iron reduction was less efficient than with phlebotomy, and adverse effects were frequently reported. Chelators have rarely been used in ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0002927002060148

Patient compliance with phlebotomy therapy for iron ... A total of 118 patients were evaluable for iron depletion and 142 for maintenance therapy; 96.6% achieved iron depletion, and 33.1% and 43.2% followed weekly ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/15157247/

Efficacy and safety of phlebotomy to reduce transfusional ... Our study shows that phlebotomies are a safe and effective method for reducing iron over-load in multiply transfused long-term AL survivors with secondary ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT00689182

Safety and Efficacy of Iron Reduction by Phlebotomy | ... Hypothesis: The reduction of total body iron by phlebotomy will be safe and feasible in the post-HSCT setting. Iron overload is common after hematopoietic ...

8. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK526131/

Iron Overload and Toxicity - StatPearls - NCBI Bookshelf Regular phlebotomy in hereditary hemochromatosis reduces total body iron and, when initiated early, has been shown to normalize life expectancy.