Iron-Refractory Iron-Deficiency AnemiaPhase 2NCT02905981
Triferic IRIDA (Iron-Refractory Iron-Deficiency Anemia) Protocol
A Phase 2 study testing Fer-In-Sol, Shohl's solution and Triferic in people with Iron-Refractory Iron-Deficiency Anemia, run by Rockwell Medical Technologies, Inc..
Trial summary
The main purpose is to determine whether Triferic, administered orally with Shohl's solution, is safe and effective for the treatment of iron-refractory iron-deficiency anemia (IRIDA). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Iron-Refractory Iron-Deficiency Anemia
- What is being tested?
- Fer-In-Sol, Shohl's solution and Triferic
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- No sites listed yet
- Study dates
- 17 Jan 2017to 15 Mar 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult patients have provided informed consent and have signed the informed consent form. For pediatric patients, parents/legal guardians of the patient have the ability to understand the requirements of the study and have demonstrated a willingness to have their child comply with all study procedures by signing the informed consent form. Where applicable, assent of the pediatric patient has also been obtained for all study procedures prior to any study-related activities.
- Patient has a history of congenital hypochromic microcytic anemia.
- Patient has a mean corpuscular volume (MCV) ≤75 fluid ounce (fL) at screening.
- Patient has a serum transferrin saturation ≤15% at screening.
- Patient has a history of no or incomplete response to oral iron therapy.
- Patient has a history of no or incomplete response to intravenous iron administration.
Show all 11Show fewer
- Patient has a history of hepcidin concentration ≥10 nanograms per milliliter (ng/mL) (3.58 nanometer (nM)) off iron supplements, adjusted for the specific hepcidin assay used to be at least 1 standard deviation (SD) greater than the mean for patients with iron-deficiency anemia.
- Patient is documented to have homozygous or compound heterozygous pathogenic mutations in Transmembrane Serine Protease 6 (TMPRSS6) in a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory.
- Patient has appropriate laboratory values for their disease state at screening (per investigator judgment).
- Patient has no significant abnormal findings on physical examination at screening that would preclude participation in the study (per investigator judgment).
- If the patient is female, she must be pre-pubertal, have had documented surgical sterilization ≥2 years prior to screening, or be practicing adequate birth control. All female patients 9 years of age and older, and also any who have reached menarche before age 9 years, must have a negative serum pregnancy test during screening. It is the investigator's responsibility to determine whether the patient has adequate birth control for study participation.
Who cannot join
- Patient has had intravenous (IV) or oral iron supplements within 2 weeks prior to Visit 2.
- Patient has had a blood transfusion within 3 months prior to Visit 2.
- Patient is receiving intravenous or oral antibiotics or antifungals for any infectious process. Prophylactic antibiotics administered on a regular basis are allowed. Otherwise-eligible patients may be rescreened when they have recovered from any acute illnesses.
- Patient has a body weight of <11 lbs (5 kg) at screening.
- Patient has participated in an investigational drug study within the 30 days prior to Visit 2.
- Patient has any condition that, in the opinion of the investigator, is likely to prevent the patient from complying with or successfully completing the protocol.
About this study
Official title: Triferic (Ferric Pyrophosphate Citrate) Administered Orally With Shohl's Solution for the Treatment of Iron-Refractory Iron-Deficiency Anemia
The main purpose is to determine whether Triferic, administered orally with Shohl's solution, is safe and effective for the treatment of iron-refractory iron-deficiency anemia (IRIDA).
Read the detailed description
This is an open label, 3-period study assessing the safety, efficacy, and pharmacokinetics of Triferic and Shohl's solution administered orally to patients with IRIDA. A total of 28 patients stratified by 4 age groups (age 0 to <6 years, age 6 to <12 years, age 12 to <18 years, and age >=18 years) will be studied.
Total participation in the study is between 4 weeks (Period 1) and 12 months (Periods 1, 2, and 3), depending upon how many Periods each patient participates in. Following screening, in Period 1 (iron absorption testing) patients undergo oral iron absorption testing during 3 visits to confirm that they adequately absorb iron from Triferic when it is administered with Shohl's solution ('Triferic responders': patients with a maximal increase from baseline in serum iron concentration >100 micrograms per deciliter (μg/dL) following an oral Shohl's solution and Triferic dose).
The 'Triferic responders' from Period 1 are then invited to participate in Period 2 (dose titration). The patients will receive Shohl's solution and Triferic orally up to 3 times per day for 4 months, titrated as needed based on laboratory results and patient tolerance, to determine whether their hemoglobin levels respond to this treatment. Period 2 'hemoglobin responders' (patients with an increase from baseline in hemoglobin (Hgb) concentration ≥1.0 grams per deciliter (g/dL) at Visit 9) will be invited to participate in period 3.
In Period 3 patients will receive Shohl's solution and Triferic orally up to 3 times per day for an additional 6 months to determine whether the hemoglobin response observed in Period 2 is sustainable. During Period 3, Shohl's solution and Triferic dose and frequency may continue to be titrated as needed based on laboratory results and patient tolerance.
A follow-up visit will occur approximately 1 week after the last completed Period study visit, regardless of the Period that the patient completes the study.
- Conditions
- Iron-Refractory Iron-Deficiency Anemia
- Being tested
- Fer-In-Sol, Shohl's solution, Triferic
- Keywords
- IRIDA, Triferic, soluble ferric pyrophosphate, Shohl's solution
- Medical subject headings
- Iron-Refractory Iron Deficiency Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fer-In-SolDrug
Also called FeSO4
Fer-In-Sol is an over-the-counter oral iron supplement. It will be administered during Period 1 at a dose of 3 mg/kg body weight.
Shohl's solutionDrug
Also called Citrate, Oracit
Shohl's solution will be supplied as 500-milliliter (mL) bottles containing citric acid United States Pharmacopeia (USP) 640 milligrams per 5 milliliters (mg/5 mL) and hydrous sodium citrate USP 490 mg/5 mL. It will be administered during all Periods at a dose of 0.67 mmol/kg body weight.
TrifericDrug
Also called ferric pyrophosphate citrate (FPC)
Triferic is an iron salt that is approved by the Food and Drug Administration (FDA) for the maintenance of hemoglobin in patients with end stage kidney disease on hemodialysis. It is experimental in this study because it has not yet been approved for patients with IRIDA. It will be administered during Period 1 at a dose of 3 mg/kg body weight. In Periods 2 and 3 the dose may be adjusted based on lab results.
| Study group | Role | What they receive |
|---|---|---|
| Period 1: Iron Absorption Tests | Experimental | During 3 consecutive weekly study visits, patients will receive Fer-In- Sol orally 3 milligram iron per kilogram (mg Fe/kg) body weight, Shohl's solution 0.67 millimoles per kilogram (mmol/kg) followed 5 - 15 minutes later by Fer-In- Sol orally 3 mg Fe/kg body weight, and Shohl's solution 0.67 mmol/kg followed 5 - 15 minutes later by Triferic orally 3 mg Fe/kg body weight (respectively). All oral doses will be administered by study personnel at the research center. Blood tests will be conducted following each administration in order to measure iron absorption to see if patients qualify for Period 2. Receives: Drug: Fer-In-Sol; Drug: Shohl's solution; Drug: Triferic |
| Period 2: Dose Titration | Experimental | Patients who qualified as 'Triferic responders' in Period 1 will participate in Period 2. Patients will be given oral Shohl's solution and Triferic to administer at home 3 times per day, with the dose being titrated higher or lower based on lab results. The initial dose will be the same as Period 1 (Shohl's solution 0.67 mmol/kg followed 5 - 15 minutes later by Triferic orally 3 mg Fe/kg body weight) but may be adjusted during Period 2 based on lab results. Period 2 will involve 5 study visits, scheduled every 4 weeks. At the end of Period 2, blood tests will be performed to determine if patients qualify for Period 3. Receives: Drug: Shohl's solution; Drug: Triferic |
| Period 3: Hemoglobin Maintenance | Experimental | Patients who qualified as 'hemoglobin responders' in Period 2 will participate in Period 3. Patients will continue to take Shohl's solution and Triferic at their titrated dose for an additional six months to confirm that their hemoglobin can be maintained over an extended period of time. The period will be comprised of 3 study visits, scheduled every 8 weeks. Receives: Drug: Shohl's solution; Drug: Triferic |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Efficacy of Triferic Delivered to IRIDA Patients:Change in Hemoglobin (Hgb)
The efficacy will be done by assessing the change from baseline in hemoglobin concentration
Measured: 4 months
Secondary outcomes (2)
The Efficacy of Triferic Being Delivered to Iron-refractory Iron Deficiency Anemia (IRIDA) Patients:Change in Serum Iron
The efficacy will be done by assessing the change from baseline in serum iron
Measured: 4 months
The Efficacy of Triferic Being Delivered to IRIDA Patients:Change From Baseline in Transferrin Saturation (TSAT)
The efficacy will be done by assessing the change from baseline in TSAT
Measured: 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2016 (estimated)
- Study start17 Jan 2017
- Primary completion15 Mar 2018
- Study completion15 Mar 2018
Why it stopped: Limited subjects, patient compliance
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Iron-Refractory Iron-Deficiency AnemiaStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rockwell Medical Technologies, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Raymond D Pratt, MD, FACP, Study director, Rockwell Medical, Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02905981
- Sponsor's protocol ID
- RMFPC-15
- Status last verified
- Sep 2020
- First submitted
- 14 Sep 2016
- Last updated
- 22 Sep 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Triferic (Ferric Pyrophosphate Citrate) Administered Orally With Shohl's Solution for the Treatment of Iron-Refractory Iron-Deficiency Anemia
- ClinicalTrials.gov ID
- NCT02905981
- Acronym / sponsor ID
- RMFPC-15
- Conditions
- Iron-Refractory Iron-Deficiency Anemia
- Keywords
- IRIDA, Triferic, soluble ferric pyrophosphate, Shohl's solution
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Limited subjects, patient compliance
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Rockwell Medical Technologies, Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Jan 2017
- Primary completion
- 15 Mar 2018 (actual)
- Study completion
- 15 Mar 2018 (actual)
- First posted
- 19 Sep 2016 (estimated)
- Results
- Posted 2 Sep 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main purpose is to determine whether Triferic, administered orally with Shohl's solution, is safe and effective for the treatment of iron-refractory iron-deficiency anemia (IRIDA).
Detailed description
This is an open label, 3-period study assessing the safety, efficacy, and pharmacokinetics of Triferic and Shohl's solution administered orally to patients with IRIDA. A total of 28 patients stratified by 4 age groups (age 0 to <6 years, age 6 to <12 years, age 12 to <18 years, and age >=18 years) will be studied.
Total participation in the study is between 4 weeks (Period 1) and 12 months (Periods 1, 2, and 3), depending upon how many Periods each patient participates in. Following screening, in Period 1 (iron absorption testing) patients undergo oral iron absorption testing during 3 visits to confirm that they adequately absorb iron from Triferic when it is administered with Shohl's solution ('Triferic responders': patients with a maximal increase from baseline in serum iron concentration >100 micrograms per deciliter (μg/dL) following an oral Shohl's solution and Triferic dose).
The 'Triferic responders' from Period 1 are then invited to participate in Period 2 (dose titration). The patients will receive Shohl's solution and Triferic orally up to 3 times per day for 4 months, titrated as needed based on laboratory results and patient tolerance, to determine whether their hemoglobin levels respond to this treatment. Period 2 'hemoglobin responders' (patients with an increase from baseline in hemoglobin (Hgb) concentration ≥1.0 grams per deciliter (g/dL) at Visit 9) will be invited to participate in period 3.
In Period 3 patients will receive Shohl's solution and Triferic orally up to 3 times per day for an additional 6 months to determine whether the hemoglobin response observed in Period 2 is sustainable. During Period 3, Shohl's solution and Triferic dose and frequency may continue to be titrated as needed based on laboratory results and patient tolerance.
A follow-up visit will occur approximately 1 week after the last completed Period study visit, regardless of the Period that the patient completes the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Period 1: Iron Absorption Tests | Experimental | Drug: Fer-In-Sol, Drug: Shohl's solution, Drug: TrifericDuring 3 consecutive weekly study visits, patients will receive Fer-In- Sol orally 3 milligram iron per kilogram (mg Fe/kg) body weight, Shohl's solution 0.67 millimoles per kilogram (mmol/kg) followed 5 - 15 minutes later by Fer-In- Sol orally 3 mg Fe/kg body weight, and Shohl's solution 0.67 mmol/kg followed 5 - 15 minutes later by Triferic orally 3 mg Fe/kg body weight (respectively). All oral doses will be administered by study personnel at the research center. Blood tests will be conducted following each administration in order to measure iron absorption to see if patients qualify for Period 2. |
| Period 2: Dose Titration | Experimental | Drug: Shohl's solution, Drug: TrifericPatients who qualified as 'Triferic responders' in Period 1 will participate in Period 2. Patients will be given oral Shohl's solution and Triferic to administer at home 3 times per day, with the dose being titrated higher or lower based on lab results. The initial dose will be the same as Period 1 (Shohl's solution 0.67 mmol/kg followed 5 - 15 minutes later by Triferic orally 3 mg Fe/kg body weight) but may be adjusted during Period 2 based on lab results. Period 2 will involve 5 study visits, scheduled every 4 weeks. At the end of Period 2, blood tests will be performed to determine if patients qualify for Period 3. |
| Period 3: Hemoglobin Maintenance | Experimental | Drug: Shohl's solution, Drug: TrifericPatients who qualified as 'hemoglobin responders' in Period 2 will participate in Period 3. Patients will continue to take Shohl's solution and Triferic at their titrated dose for an additional six months to confirm that their hemoglobin can be maintained over an extended period of time. The period will be comprised of 3 study visits, scheduled every 8 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Adult patients have provided informed consent and have signed the informed consent form. For pediatric patients, parents/legal guardians of the patient have the ability to understand the requirements of the study and have demonstrated a willingness to have their child comply with all study procedures by signing the informed consent form. Where applicable, assent of the pediatric patient has also been obtained for all study procedures prior to any study-related activities.
- Patient has a history of congenital hypochromic microcytic anemia.
- Patient has a mean corpuscular volume (MCV) ≤75 fluid ounce (fL) at screening.
- Patient has a serum transferrin saturation ≤15% at screening.
- Patient has a history of no or incomplete response to oral iron therapy.
Key exclusion criteria (6 listed)
- Patient has had intravenous (IV) or oral iron supplements within 2 weeks prior to Visit 2.
- Patient has had a blood transfusion within 3 months prior to Visit 2.
- Patient is receiving intravenous or oral antibiotics or antifungals for any infectious process. Prophylactic antibiotics administered on a regular basis are allowed. Otherwise-eligible patients may be rescreened when they have recovered from any acute illnesses.
- Patient has a body weight of <11 lbs (5 kg) at screening.
- Patient has participated in an investigational drug study within the 30 days prior to Visit 2.
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 |
|---|---|---|
| Fer-In-SolAlso: FeSO4Fer-In-Sol is an over-the-counter oral iron supplement. It will be administered during Period 1 at a dose of 3 mg/kg body weight. | Drug | Period 1: Iron Absorption Tests |
| Shohl's solutionAlso: Citrate, OracitShohl's solution will be supplied as 500-milliliter (mL) bottles containing citric acid United States Pharmacopeia (USP) 640 milligrams per 5 milliliters (mg/5 mL) and hydrous sodium citrate USP 490 mg/5 mL. It will be administered during all Periods at a dose of 0.67 mmol/kg body weight. | Drug | Period 1: Iron Absorption Tests, Period 2: Dose Titration, Period 3: Hemoglobin Maintenance |
| TrifericAlso: ferric pyrophosphate citrate (FPC)Triferic is an iron salt that is approved by the Food and Drug Administration (FDA) for the maintenance of hemoglobin in patients with end stage kidney disease on hemodialysis. It is experimental in this study because it has not yet been approved for patients with IRIDA. It will be administered during Period 1 at a dose of 3 mg/kg body weight. In Periods 2 and 3 the dose may be adjusted based on lab results. | Drug | Period 1: Iron Absorption Tests, Period 2: Dose Titration, Period 3: Hemoglobin Maintenance |
Primary outcome
The Efficacy of Triferic Delivered to IRIDA Patients:Change in Hemoglobin (Hgb)
Time frame: 4 months
The efficacy will be done by assessing the change from baseline in hemoglobin concentration
2 secondary outcomes
The Efficacy of Triferic Being Delivered to Iron-refractory Iron Deficiency Anemia (IRIDA) Patients:Change in Serum Iron
Time frame: 4 months
The Efficacy of Triferic Being Delivered to IRIDA Patients:Change From Baseline in Transferrin Saturation (TSAT)
Time frame: 4 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes
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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.
Raymond D Pratt, MD, FACP study director
Rockwell Medical, Inc
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fer-In-Sol, Shohl's solution and Triferic in people with Iron-Refractory Iron-Deficiency Anemia, run by Rockwell Medical Technologies, Inc..
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 11 inclusion and 6 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: stopped early, as of the sponsor's last update on 22 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Sep 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
The Efficacy of Triferic Delivered to IRIDA Patients:Change in Hemoglobin (Hgb) (time frame: 4 months). 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 22 Sep 2020; AzTrials last refreshed its copy on 2 Nov 2026.