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Triferic IRIDA (Iron-Refractory Iron-Deficiency Anemia) Protocol | Clinical Trials | Clareo Health

TERMINATEDPHASE2

Triferic IRIDA (Iron-Refractory Iron-Deficiency Anemia) Protocol

Triferic (Ferric Pyrophosphate Citrate) Administered Orally With Shohl's Solution for the Treatment of Iron-Refractory Iron-Deficiency Anemia

NCT02905981•Rockwell Medical Technologies, Inc.

View on ClinicalTrials.gov

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).

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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. 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.
•2. Patient has a history of congenital hypochromic microcytic anemia.
•3. Patient has a mean corpuscular volume (MCV) ≤75 fluid ounce (fL) at screening.
•4. Patient has a serum transferrin saturation ≤15% at screening.
•5. Patient has a history of no or incomplete response to oral iron therapy.
•6. Patient has a history of no or incomplete response to intravenous iron administration.
•7. 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.
•8. 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.
•9. Patient has appropriate laboratory values for their disease state at screening (per investigator judgment).
•10. Patient has no significant abnormal findings on physical examination at screening that would preclude participation in the study (per investigator judgment).
+1 more criteria

Exclusion Criteria

•1. Patient has had intravenous (IV) or oral iron supplements within 2 weeks prior to Visit 2.
•2. Patient has had a blood transfusion within 3 months prior to Visit 2.
•3. 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.
•4. Patient has a body weight of \<11 lbs (5 kg) at screening.
•5. Patient has participated in an investigational drug study within the 30 days prior to Visit 2.
•6. 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.

Key Dates

Start Date

January 17, 2017

Primary Completion Date

March 15, 2018

Completion Date

March 15, 2018

Last Updated

September 22, 2020

Enrollment

ACTUAL participants

Conditions

Iron-Refractory Iron-Deficiency Anemia

Interventions

Fer-In-Sol

DRUG

Shohl's solution

DRUG

Triferic

DRUG