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

Penn State Health

Status and phase: Completed; Phase 1

Conditions: Iron Deficiency Anemia

Treatments: Other: Novel Medical Food

Study type: Interventional

Funder types: Other

Identifiers

NCT03038633

34696

Details and patient eligibility

About

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.

Full 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.

Enrollment: 2 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Other

Allocation: Non-Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

---

2 participants in 4 patient groups

Cohort 1

Experimental group

Description:

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

Treatment:

Other: Novel Medical Food

Cohort 2

Experimental group

Description:

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

Treatment:

Other: Novel Medical Food

Cohort 3

Experimental group

Description:

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

Treatment:

Other: Novel Medical Food

Cohort 4

Experimental group

Description:

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

Treatment:

Other: Novel Medical Food