Iron Deficiency AnemiaPhase 1NCT03038633
A Trial to Evaluate the Safety and Tolerability of a Novel Medical Food for Management of Iron Deficiency Anemia
A Phase 1 study testing Novel Medical Food in people with Iron Deficiency Anemia, run by Milton S. Hershey Medical Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Iron Deficiency Anemia
- What is being tested?
- Novel Medical Food
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- May 2014to Jun 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Hemoglobin, (Hgb) 7-10 g/dL; transferrin saturation <20%; and serum ferritin <20ng/mL
Who cannot join
- 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.
Show all 20Show fewer
- 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 locations and contacts
1 site in 1 country.
United States1 site
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
About this study
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.
Read the 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.
- Conditions
- Iron Deficiency Anemia
- Being tested
- Novel Medical Food
- Medical subject headings
- Anemia, Iron-Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Novel Medical FoodOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 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 Receives: Other: Novel Medical Food |
| Cohort 2 | Experimental | 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 Receives: Other: Novel Medical Food |
| Cohort 3 | Experimental | 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 Receives: Other: Novel Medical Food |
| Cohort 4 | Experimental | 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 Receives: Other: Novel Medical Food |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose-limiting toxicity, (DLT)
Still evaluating data
Measured: 28 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2017 (estimated)
- Study startMay 2014
- Primary completionJun 2015
- Study completionJun 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Iron Deficiency AnemiaStudies of the same condition, open and under way.
- More trials run by Milton S. Hershey Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Crossover
- 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)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Milton S. Hershey Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jeffrey J. Pu, MD, PhD, Milton S. Hershey Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03038633
- Sponsor's protocol ID
- 34696
- Status last verified
- Jan 2017
- First submitted
- 27 Oct 2015
- Last updated
- 31 Jan 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Data under review
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Trial to Evaluate the Safety and Tolerability of a Novel Medical Food for Management of Iron Deficiency Anemia
- ClinicalTrials.gov ID
- NCT03038633
- Acronym / sponsor ID
- 34696
- Conditions
- Iron Deficiency Anemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Milton S. Hershey Medical CenterUniversity, hospital or other
- Study start
- May 2014 (actual)
- Primary completion
- Jun 2015 (actual)
- Study completion
- Jun 2015 (actual)
- First posted
- 31 Jan 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 4
- 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).
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Other: Novel Medical Food900 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 |
| Cohort 2 | Experimental | Other: Novel Medical Food1800 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 |
| Cohort 3 | Experimental | Other: Novel Medical Food2700 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 |
| Cohort 4 | Experimental | Other: Novel Medical Food3600 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 |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Hemoglobin, (Hgb) 7-10 g/dL; transferrin saturation <20%; and serum ferritin <20ng/mL
Key exclusion criteria (20 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Novel Medical FoodThis 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. | Other | Cohort 1, Cohort 2, Cohort 3, Cohort 4 |
Primary outcome
Dose-limiting toxicity, (DLT)
Time frame: 28 Days
Still evaluating data
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2017.
- Individual participant dataSharing planned: Undecided. Data under review
Related trials for Iron Deficiency Anemia
RecruitingPhase 4
The Effects of Ferric Derisomaltose in Patients With Acute Heart Failure and Iron Deficiency on Exercise Capacity and Quality of Life(COREVIVE-HFrEF)
Heart Failure With Reduced Ejection Fraction, Iron Deficiency
RecruitingPhase 4
The Effects of Ferric Derisomaltose in Patients With Acute Heart Failure and Iron Deficiency on Exercise Capacity and Quality of Life
Heart Failure With Preserved Ejection Fraction, Iron Deficiencies
Recruiting
The Effects of Iron Treatment on Vaccine Immunogenicity in Kenyan Infants With Iron Deficiency
Iron Deficiencies
RecruitingPhase 4
The Effects of Low-Dose Versus High-Dose Intravenous IRON Therapy With Ferric DerisomaltOSE in Patients With Chronic Heart Failure and Iron Deficiency
Heart Failure, Iron Deficiency
Enrolling by invitationObservational study
Long-term Impacts of Psychosocial Stimulation and Iron Supplementation
Iron Deficiency Anemia, Parenting Intervention
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Novel Medical Food in people with Iron Deficiency Anemia, run by Milton S. Hershey Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose-limiting toxicity, (DLT) (time frame: 28 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jan 2017.