Today, we will explore the latest research and clinical trials focused on the management of hemochromatosis. The following list has been carefully curated by evaluating ongoing trials related to hemochromatosis in the United States. Please note that the dates provided are approximate and may be subject to change. The list primarily includes studies with results published within the last five years.

This series is intended to provide a preview of what new innovations are to come on a given topic, and how might the results of those studies ultimately impact clinical guidelines on the topic(s). 

So now let’s jump into the list of Hemochromatosis Clinical Trials!

Quick View Table of Hemochromatosis Clinical Trials

Study TitlePhasesEnrollmentStart DateLast Update Posted
Treatment of HemochromatosisPHASE 26141/1/20017/2/2024
PTG-300 in Subjects With Hereditary HemochromatosisPHASE 2163/19/20206/15/2023
A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary HemochromatosisPHASE 27011/29/20176/9/2022
Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to FoodPHASE 4655/2/20097/20/2021
Pilot Study for Patients With Poor Response to DeferasiroxPHASE 4153/7/20081/12/2024

HemochromatosisClinical Trials:

Treatment of Hemochromatosis

Study Details | Source

  • Sponsor: National Institutes of Health Clinical Center (CC)
  • This study will evaluate the effectiveness of a test called MCV in guiding phlebotomy (blood drawing) therapy in patients with hemochromatosis, an inherited disorder that causes too much iron to be absorbed by the intestine. The excess damages body tissues, most severely in the liver, heart, pancreas and joints. Because iron is carried in the hemoglobin of red blood cells, removing blood can effectively lower the body’s iron stores.
  • Interventions: PROCEDURE: Phlebotomy
  • Primary Outcomes Measures: MCV drops 1-3% below baseline, Response to phlebotomy therapy in HH patients, as evidenced by iron-depletion, 4 to 12 months after starting phlebotomy therapy

PTG-300 in Subjects With Hereditary Hemochromatosis

Study Details | Source

  • Sponsor: Protagonist Therapeutics, Inc.
  • This study will be conducted at multiple sites and every patient will get treated with PTG-300. The objective of the study is to assess the effect of PTG-300 in treating adult hereditary hemochromatosis patients.
  • Interventions: DRUG: PTG-300
  • Primary Outcomes Measures: Effect of PTG-300 on Transferrin Saturation, Change from Baseline to Week 24 (or End of Treatment) in transferrin saturation (TSAT) as measured by blood laboratory tests., Week 24 (or End of Treatment if treated for less than 24 weeks)|Effect of PTG-300 on Serum Iron, Change from Baseline to Week 24 (or End of Treatment) in serum iron as measured by blood laboratory tests., Week 24 (or End of Treatment if treated for less than 24 weeks)

A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis

Study Details | Source

  • Sponsor: La Jolla Pharmaceutical Company
  • This study is a Phase 2 multicenter, randomized, placebo controlled, single-blind study. The primary objective of the study is to compare the effect of weekly dosing of LJPC-401 (synthetic human hepcidin) versus placebo on transferrin saturation (TSAT) in an adult hereditary hemochromatosis patient population.
  • Interventions: DRUG: LJPC-401, DRUG: Placebo
  • Primary Outcomes Measures: Effect of LJPC-401 Versus Placebo on Blood Iron Levels, Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests., 16 Weeks

Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food

Study Details | Source

  • Sponsor: Novartis Pharmaceuticals
  • This single-arm, open-label, multi-center study enrolled 65 patients from approximately 20 centers. All patients who met the study criteria and were taking, beginning or resuming treatment with Deferasirox were allowed. The study will begin with a one month run-in phase, where all patients were instructed to take Deferasirox according to their physician’s prescribing information.
  • Interventions: DRUG: deferasirox
  • Primary Outcomes Measures: Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12, Palatability was assessed by participants based on a five-point Facial Hedonic scale defined as: dislike extremely; somewhat dislike; neither like or dislike; somewhat like; like extremely for the meal and method of administration. For participants under 5 years of age, the scale was completed by parent or caregiver., Week 8 and Week 12

Pilot Study for Patients With Poor Response to Deferasirox

Study Details | Source

  • Sponsor: Boston Children’s Hospital
  • “This purpose of this study is to understand the differences between people who have a good response to deferasirox (exjade) compared to people who have a poor response to this medication when used for transfusion-dependent iron overload.
  • The hypothesis is that patients with poor responses have physiologic barriers to deferasirox that may include absorption, pharmacokinetics of drug metabolism, hepatic clearance and/or genetic factors.”
  • Interventions: DRUG: Deferoxamine|DRUG: Deferasirox|RADIATION: HIDA
  • Primary Outcomes Measures: Area Under the Curve of Deferasirox After a Dose of 35 mg/kg, Area Under the Curve (AUC) 0 to 24 hours post dose, 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose|Half-Life of Deferasirox, All patients received the same interventions of deferoxamine challenge, deferasirox dose with pharmacokinetic monitoring. Then we compared responses between patients who were known to be slow responders to deferasirox and those who were known to be rapid responders (chelated well).
  • Deferoxamine: After a 3-day washout period from all chelation, all patients have a 12 hour infusion of 50mg/kg of deferoxamine with urine collection and pre and post blood sampling to assess iron and Total Iron Binding Capacity (TIBC) by atomic absorption., 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose.|Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg, Volume of distribution/bioavailability (Vd/F), 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose|Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg, Volume of distribution/bioavailability (Vd/F), adjusted per kilogram body weight, 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose|Clearance/Bioavailability of Deferasirox in Patients With Poor Response to Deferasirox Compared to Patients With Good Response After a Dose of 35 mg/kg, Clearance/bioavailability (CL/F), 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose.

Potential Guideline That May Be Affected Includes:

There you have it – a list of Clinical Trials for Hemochromatosis as of July 2024. Stay tuned, for our next Guidelines+ Trials Rundown. Sign up for alerts and stay informed on the latest published guidelines and articles.

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