Iron-Refractory Iron-Deficiency Anemia Clinical Trial
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).
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.
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