A Trial to Evaluate the Safety and Tolerability of a Novel Medical Food for Management of Iron Deficiency Anemia

Study Overview
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Brief Summary
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This is a Phase 1 clinical trial designed to evaluate the safety and tolerability of a novel medical food utilizing a nutritional strain of yeast for management of Iron Deficiency Anemia.

Detailed Description
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The investigational product is a medical food and is the result of discoveries following extensive research on two preclinical models carried out by a team of researchers at Penn State University. This study will utilize the standard 3+3 rule-based dose-escalation schemes, which use predetermined dose levels and cohorts of three patients. The total time commitment for each subject is approximately three months.

Official Title
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A Trial to Evaluate the Safety and Tolerability of a Novel Medical Food for Management of Iron Deficiency Anemia

Conditions
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Iron Deficiency Anemia

Intervention / Treatment
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* Other: Novel Medical Food


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Hemoglobin, (Hgb) 7-10 g/dL; transferrin saturation <20%; and serum ferritin <20ng/mL Exclusion Criteria: Pregnant or sexually-active female subjects who are of childbearing potential and who are not willing to use an acceptable form of contraception (tubal ligation or otherwise be incapable of pregnancy, hormonal contraceptives, spermicide plus barrier or intrauterine device). Present consumption of iron supplements or multivitamins must be switched to vitamins not containing iron, such as the multivitamin Centrum Silver. No washout period is necessary since it will be apparent from the ongoing anemia that any current supplements are ineffective. Current anemia not attributed to immune deficiency, (ID) (e.g. other microcytic anemia or hemolytic, macrocytic, sideroblastic or myelosuppression or chemotherapy or radiotherapy induced anemia). Active malignancy within 1 year. Basal or squamous cell skin cancer is not exclusionary. Aspartate Aminotransferase, (AST) or Alanine Aminotransferase, (ALT) at screening greater than 1.5 times the upper limit of normal. Known positive hepatitis B with evidence of active hepatitis. Known positive HIV-1/HIV-2 antibodies (anti-HIV). Patient has a current diagnosis of asthma and is actively using an anti-asthmatic therapy. Received an investigational drug within 30 days of screening. Hemochromatosis or other iron storage disorders. Unregulated hypertension Chronic kidney disease. Chronic inflammatory condition including but not limited to Lupus and Rheumatoid Arthritis. Significant cardiovascular disease, including but not limited to myocardial infarction or unstable angina within 6 months prior to study inclusion or current history of New York Heart Association, (NYHA) Class III or IV congestive heart failure. Smoking Irritable Bowel Syndrome Any other laboratory abnormality, medical condition or psychiatric disorder which in the opinion of the investigator puts the subject's disease management at risk or may result in the subject being unable to comply with study requirements. Breastfeeding planned on or after enrolling in the study. Known allergy to yeast Currently on Monoamine oxidase inhibitors, (MAOI's) or Demerol

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Allocation: Non-Randomized
Intervention Model: Crossover Assignment
Interventional Model Description: Individuals will receive 300 mg capsules of the novel medical food. The iron content of these capsules is 7.4mg. Our initial dose is set at 22.2 mg of iron. This dose was chosen as the initial starting dose because it is within the range of the RDA for iron for adult women (18mg). Thus this dose is considered the minimum that would be utilized in any setting for maintenance of a healthy iron status. The dosing increases to a maximum of 88.8mg of iron per day which is significantly below the standard amount of iron (165-195mg per day) recommended for an iron deficient individual or the amount of iron given intravenously (1000 mg) for some iron deficient conditions. As mentioned, the total element iron given a patient treated with FAS is up to 190mg of iron. Assuming a 10% absorption rate these patients get 19mg of iron. So if our goal is approximately 19mg of iron absorbed per day and we assume a 50% absorption rate then our target dose is 6-9 capsules per day (44.4-66.6 mg iron).
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort 1<br>900 mg novel medical food containing 22.2mg iron receive 900 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated receive a phone call from coordinator between Day 5-9 to assess side effects and medications return to General Clinical Research Center, (GCRC) for a blood draw at days 14, 28, 60, and 90 while on novel medical food | Other: Novel Medical Food<br>* This medical food takes advantage of the stability and high level of solubility of the human H-ferritin protein to be absorbed by the body as an iron source; indeed H-ferritin is enriched in breast milk as the mechanism for transferring iron from mother to infants. Once the ferritin has been absorbed from the gut, uptake into each organ is regulated by a specific extracellular receptor pathway to deliver iron in the body's preferred bioavailable form.<br>|
| Experimental: Cohort 2<br>1800 mg novel medical food containing 44.4 mg of iron receive 1800 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated receive a phone call from coordinator between Day 5-9 to assess side effects and medications return to GCRC for a blood draw at days 14, 28, 60, and 90 while on novel medical food | Other: Novel Medical Food<br>* This medical food takes advantage of the stability and high level of solubility of the human H-ferritin protein to be absorbed by the body as an iron source; indeed H-ferritin is enriched in breast milk as the mechanism for transferring iron from mother to infants. Once the ferritin has been absorbed from the gut, uptake into each organ is regulated by a specific extracellular receptor pathway to deliver iron in the body's preferred bioavailable form.<br>|
| Experimental: Cohort 3<br>2700 mg novel medical food containing 66.6 mg of iron receive 2700 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated receive a phone call from coordinator between Day 5-9 to assess side effects and medications return to GCRC for a blood draw at days 14, 28, 60, and 90 while on novel medical food | Other: Novel Medical Food<br>* This medical food takes advantage of the stability and high level of solubility of the human H-ferritin protein to be absorbed by the body as an iron source; indeed H-ferritin is enriched in breast milk as the mechanism for transferring iron from mother to infants. Once the ferritin has been absorbed from the gut, uptake into each organ is regulated by a specific extracellular receptor pathway to deliver iron in the body's preferred bioavailable form.<br>|
| Experimental: Cohort 4<br>3600 mg novel medical food containing 88.8 mg of iron receive 3600 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated receive a phone call from coordinator between Day 5-9 to assess side effects and medications return to GCRC for a blood draw at days 14, 28, 60, and 90 while on novel medical food | Other: Novel Medical Food<br>* This medical food takes advantage of the stability and high level of solubility of the human H-ferritin protein to be absorbed by the body as an iron source; indeed H-ferritin is enriched in breast milk as the mechanism for transferring iron from mother to infants. Once the ferritin has been absorbed from the gut, uptake into each organ is regulated by a specific extracellular receptor pathway to deliver iron in the body's preferred bioavailable form.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Dose-limiting toxicity, (DLT) | Still evaluating data | 28 Days |