Back to resultsA Trial to Evaluate the Safety and Tolerability of a Novel Medical Food for Management of Iron Deficiency Anemia
Primary Purpose
Iron Deficiency Anemia
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Novel Medical Food
Sponsored by
About this trial
This is an interventional other trial for Iron Deficiency Anemia
Eligibility Criteria
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
Sites / Locations
- Penn State Milton S. Hershey Medical Center
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Experimental
Arm Label
Cohort 1
Cohort 2
Cohort 3
Cohort 4
Arm 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
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
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
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
Outcomes
Primary Outcome Measures
Dose-limiting toxicity, (DLT)
Still evaluating data
Secondary Outcome Measures
Full Information
NCT ID
NCT03038633
First Posted
October 27, 2015
Last Updated
January 30, 2017
Sponsor
Milton S. Hershey Medical Center
1. Study Identification
Unique Protocol Identification Number
NCT03038633
Brief Title
A Trial to Evaluate the Safety and Tolerability of a Novel Medical Food for Management of Iron Deficiency Anemia
Official Title
A Trial to Evaluate the Safety and Tolerability of a Novel Medical Food for Management of Iron Deficiency Anemia
Study Type
Interventional
2. Study Status
Record Verification Date
January 2017
Overall Recruitment Status
Completed
Study Start Date
May 2014 (Actual)
Primary Completion Date
June 2015 (Actual)
Study Completion Date
June 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Milton S. Hershey Medical Center
4. Oversight
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
Data Monitoring Committee
No
5. Study Description
Brief Summary
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
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Deficiency Anemia
7. Study Design
Primary Purpose
Other
Study Phase
Phase 1
Interventional Study Model
Crossover Assignment
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)
Allocation
Non-Randomized
Enrollment
2 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Cohort 1
Arm Type
Experimental
Arm 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
Arm Title
Cohort 2
Arm Type
Experimental
Arm 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
Arm Title
Cohort 3
Arm Type
Experimental
Arm 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
Arm Title
Cohort 4
Arm Type
Experimental
Arm 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
Intervention Type
Other
Intervention Name(s)
Novel Medical Food
Intervention Description
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.
Primary Outcome Measure Information:
Title
Dose-limiting toxicity, (DLT)
Description
Still evaluating data
Time Frame
28 Days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Penn State Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Undecided
IPD Sharing Plan Description
Data under review
Learn more about this trial
A Trial to Evaluate the Safety and Tolerability of a Novel Medical Food for Management of Iron Deficiency Anemia
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