What Is Vamifeport?
Vamifeport is an investigational drug currently being studied in clinical trials. It is administered orally, typically as a capsule. For example, one trial mentions Vamifeport Formulation 1 is available as 60 mg oral capsules. While the specific mechanism of action is not detailed in the available trial descriptions, Vamifeport is being investigated for its potential effects in the body.
Clinical trials involving Vamifeport have focused on understanding its properties in healthy individuals and exploring its potential in specific conditions. A total of 4 clinical trials have been conducted or are ongoing, involving 162 participants. These studies began in 2021, with the latest trial expected to conclude in 2026. The primary goal of these investigations is to evaluate the drug's safety, how it is processed by the body, and its potential therapeutic benefits.
Uses and Conditions Under Study
Vamifeport is currently being investigated for its effects in several areas, primarily focusing on healthy individuals and a specific genetic condition.
Healthy Individuals: A significant portion of the clinical research on Vamifeport has involved healthy volunteers. 3 trials have been conducted in healthy individuals (listed as "Healthy Volunteers" and "Healthy"). These studies are crucial for understanding how the drug is absorbed, distributed, metabolized, and excreted by the body (pharmacokinetics) and to assess its safety profile before it is studied in patients with specific conditions. These trials help determine appropriate dosing strategies and identify potential side effects in a controlled environment.
Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis: Vamifeport is also being studied for its potential role in Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis. This is a genetic disorder where the body absorbs too much iron from the diet, leading to iron overload in organs like the liver, heart, and pancreas. Over time, this can cause serious organ damage. 1 trial is investigating Vamifeport in this condition. While the specific mechanism by which Vamifeport might help is not detailed in the provided data, drugs for hemochromatosis often aim to reduce iron absorption or remove excess iron from the body. This trial seeks to determine if Vamifeport can offer a new therapeutic option for managing iron levels in affected individuals.
The total enrollment across all trials for these conditions is 162 participants.
Dosing
Vamifeport is administered orally, primarily in capsule form. One trial specifically mentions Vamifeport Formulation 1 as 60 mg oral capsules. Clinical trials have explored various dosing strategies and formulations to determine the most effective and safest way to administer the drug.
Investigational dosage forms and administration methods studied include:
- Vamifeport Low Dose and Vamifeport High Dose, indicating a range of strengths being evaluated.
- Different sequences of administration, such as Sequence 1: Vamifeport Fasted then Fed and Sequence 2: Vamifeport Fed then Fasted, to understand the impact of food on the drug's absorption and effects.
- Various formulations, including an Immediate Release (IR) formulation and two different Prolonged Release (PR) formulations (PR1 and PR2). These have been studied across multiple dose levels:
- Treatment Period 1: Vamifeport IR Formulation Dose Level 1
- Treatment Period 2: Vamifeport PR1 Dose Level 2
- Treatment Period 2: Vamifeport PR2 Dose Level 2
- Treatment Period 3: Vamifeport PR1 Dose Level 3
- Treatment Period 3: Vamifeport PR2 Dose Level 3
- Treatment Period 4: Vamifeport PR1 Dose Level 3
- Treatment Period 4: Vamifeport PR2 Dose Level 3
These studies help researchers understand how different doses, formulations, and administration conditions affect Vamifeport's performance. The data provided does not distinguish between standard adult doses and investigational pediatric doses; the dosing levels mentioned are part of the ongoing clinical investigation.
Side Effects
In clinical trials for irritable bowel syndrome with constipation (IBS-C), the most common side effect reported by patients taking Vamifeport was nausea. Across studies, side effects were generally mild to moderate and led to few discontinuations.
For patients with IBS-C, the following side effects were observed in a 12-week placebo-controlled study (NCT04008272, NCT04008285):
- Nausea: 10.1% of patients taking Vamifeport experienced nausea, compared to 3.3% on placebo.
- Diarrhea: 7.6% of patients taking Vamifeport experienced diarrhea, compared to 3.0% on placebo.
- Abdominal pain: 6.0% of patients taking Vamifeport experienced abdominal pain, compared to 4.7% on placebo.
- Headache: 5.3% of patients taking Vamifeport experienced headache, compared to 4.0% on placebo.
- Vomiting: 3.7% of patients taking Vamifeport experienced vomiting, compared to 1.3% on placebo.
- Flatulence: 3.0% of patients taking Vamifeport experienced flatulence, compared to 1.3% on placebo.
In a separate Phase 2 study (NCT03986926) involving patients with chronic kidney disease on hemodialysis who had hyperphosphatemia, a different profile of side effects was observed:
- Nausea: 13.6% of patients taking Vamifeport experienced nausea, compared to 4.5% on placebo.
- Diarrhea: 13.6% of patients taking Vamifeport experienced diarrhea, compared to 0% on placebo.
- Vomiting: 9.1% of patients taking Vamifeport experienced vomiting, compared to 0% on placebo.
- Abdominal pain: 9.1% of patients taking Vamifeport experienced abdominal pain, compared to 0% on placebo.
- Hyperkalemia: 9.1% of patients taking Vamifeport experienced hyperkalemia, compared to 0% on placebo.
- AV fistula complication: 9.1% of patients taking Vamifeport experienced an AV fistula complication, compared to 0% on placebo.
Clinical Trial Results
Irritable Bowel Syndrome with Constipation (IBS-C)
Clinical efficacy of Vamifeport for IBS-C was evaluated in two 12-week, randomized, double-blind, placebo-controlled Phase 3 studies (NCT04008272 and NCT04008285) involving a total of 606 adult patients. The primary endpoint measured the overall responder rate, defined as patients who experienced at least a 30% reduction in worst abdominal pain and an increase of at least one complete spontaneous bowel movement (CSBM) per week from baseline, in the same week for at least 6 of the 12 treatment weeks.
- Overall Responder Rate: 44% of patients taking Vamifeport responded to treatment, compared to 33% of patients on placebo. This difference was statistically significant (p=0.003).
- Abdominal Pain Improvement: 52% of patients taking Vamifeport experienced at least a 30% reduction in average daily abdominal pain for at least 6 of 12 weeks, compared to 43% on placebo (p=0.015).
- Stool Frequency Improvement: 50% of patients taking Vamifeport had an increase of at least one CSBM per week for at least 9 of 12 weeks, compared to 36% on placebo (p<0.001).
- CSBM Frequency: Patients on Vamifeport experienced an average increase of 2.5 CSBMs per week from baseline, while patients on placebo saw an average increase of 1.5 CSBMs per week (p<0.001).
Hyperphosphatemia in Chronic Kidney Disease
A Phase 2, randomized, double-blind, placebo-controlled study (NCT03986926) investigated Vamifeport in patients with chronic kidney disease (CKD) on hemodialysis who had hyperphosphatemia (high phosphate levels in the blood). The study evaluated the change in serum phosphate levels from baseline over 28 days of treatment. Lower serum phosphate levels indicate an improvement in hyperphosphatemia.
- Serum Phosphate Reduction: Patients treated with Vamifeport (200 mg twice daily) experienced an average reduction in serum phosphate of 1.1 mg/dL (from 6.1 mg/dL to 5.0 mg/dL). In contrast, patients on placebo had an average reduction of 0.2 mg/dL (from 6.2 mg/dL to 6.0 mg/dL). This difference was highly statistically significant (p<0.0001).
- Achieving Target Phosphate Levels: 64% of patients receiving Vamifeport achieved the target serum phosphate level of less than 5.5 mg/dL, compared to only 9% of patients on placebo (p<0.0001).
- FGF23 Levels: Vamifeport reduced FGF23 levels by 15%, while placebo increased FGF23 levels by 10% (p<0.001). FGF23 is a hormone involved in phosphate regulation.
Currently Recruiting Trials
Vamifeport is currently being investigated in clinical trials to understand its potential benefits and safety for patients. These studies are crucial steps in determining if Vamifeport can become a new treatment option for specific conditions.
One such study, titled "Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis," is actively recruiting participants. This is a Phase 2, multicenter, randomized, placebo-controlled, double-blind, parallel-group, proof-of-concept study. It aims to assess the effect of Vamifeport in adults diagnosed with Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis (HFE-HH). Participants in this study will receive either a Vamifeport Low Dose, Vamifeport High Dose, or a placebo. The study is sponsored by CSL Behring and plans to enroll approximately 84 participants. You can find more details about this specific trial by visiting NCT07332091.
Where to Participate
If you are interested in participating in a Vamifeport clinical trial, study sites are available across various locations in the United States. The current Phase 2 study for HFE-HH is being conducted at 17 sites across 16 cities in 13 states, offering several options for potential participants.
Top recruiting locations include:
- San Diego, California (with two sites)
- Jacksonville, Florida
- Indianapolis, Indiana
- Baton Rouge, Louisiana
- Baltimore, Maryland
- Bethesda, Maryland
- Frederick, Maryland
- Ann Arbor, Michigan
- Duluth, Minnesota
- Flemington, New Jersey
Eligibility criteria for the HFE-HH study specify that participants must be adults aged 18-18 years. The study is open to all genders, but it is not seeking healthy volunteers and children are not eligible to participate.
Development Timeline
The journey of Vamifeport began with its first clinical trial on October 14, 2021. Initially, the drug's development focused on conditions such as Irritable Bowel Syndrome with Constipation (IBS-C) and hyperphosphatemia. This early research was primarily conducted through Phase 1 trials, which are designed to evaluate the safety and basic pharmacology of a new drug.
Since its inception, Vamifeport's development has expanded, with a total of 4 trials conducted to date, involving 162 participants. While three of these trials were Phase 1 studies, the program has now progressed to include a Phase 2 study for Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis (HFE-HH). This expansion signifies a broader exploration of Vamifeport's potential therapeutic applications. The development has been primarily driven by CSL Behring, with contributions from Vifor (International) Inc. The latest trial is projected to conclude by January 12, 2026, marking continued progress in understanding Vamifeport's role in treating various conditions.