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

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.

Study Overview

• Status: Completed
• Conditions: Iron Deficiency Anemia
• Intervention / Treatment: Other: Novel Medical Food

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 2
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Milton S. Hershey Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: OTHER
• Allocation: NON_RANDOMIZED
• Interventional Model: CROSSOVER
• Masking: NONE

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Cohort 1; 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; 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.
EXPERIMENTAL: Cohort 2; 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; 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.
EXPERIMENTAL: Cohort 3; 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; 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.
EXPERIMENTAL: Cohort 4; 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; 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.

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Milton S. Hershey Medical Center

Study record dates

Study Major Dates

• Study Start (ACTUAL): May 1, 2014
• Primary Completion (ACTUAL): June 1, 2015
• Study Completion (ACTUAL): June 1, 2015

Study Registration Dates

• First Submitted: October 27, 2015
• First Submitted That Met QC Criteria: January 30, 2017
• First Posted (ESTIMATE): January 31, 2017

Study Record Updates

• Last Update Posted (ESTIMATE): January 31, 2017
• Last Update Submitted That Met QC Criteria: January 30, 2017
• Last Verified: January 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Hematologic Diseases; Nutrition Disorders; Anemia, Hypochromic; Iron Metabolism Disorders; Malnutrition; Anemia, Iron-Deficiency; Anemia; Deficiency Diseases
• Other Study ID Numbers: 34696

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: Data under review

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes