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

NCT02905981 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: RMFPC-15
• Study Type: interventional
• Target: 1
• Locations: 0 countries
• Sites: N/A

Brief 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).

Conditions

Iron-Refractory Iron-Deficiency Anemia

Keywords

IRIDA; Triferic; soluble ferric pyrophosphate; Shohl's solution

Outcome Measures

Primary Outcomes (1)

• 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

  4 months

Secondary Outcomes (2)

• The Efficacy of Triferic Being Delivered to Iron-refractory Iron Deficiency Anemia (IRIDA) Patients:Change in Serum Iron

  4 months

• The Efficacy of Triferic Being Delivered to IRIDA Patients:Change From Baseline in Transferrin Saturation (TSAT)

  4 months

Study Arms (3)

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.

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.

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.

Drug: Shohl's solution; Drug: Triferic

Interventions

Fer-In-Sol DRUG

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.

Also known as: FeSO4

Period 1: Iron Absorption Tests

Shohl's solution DRUG

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.

Also known as: Citrate, Oracit

Period 1: Iron Absorption Tests; Period 2: Dose Titration; Period 3: Hemoglobin Maintenance

Triferic DRUG

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.

Also known as: ferric pyrophosphate citrate (FPC)

Period 1: Iron Absorption Tests; Period 2: Dose Titration; Period 3: Hemoglobin Maintenance

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Rockwell Medical Technologies, Inc.: lead

MeSH Terms

Conditions

Iron-Refractory Iron Deficiency Anemia

Interventions

ferrous sulfate; Citrates; Citric Acid; ferric pyrophosphate; spleen fibrinolytic proteinase (human)

Intervention Hierarchy (Ancestors)

Tricarboxylic Acids; Acids, Acyclic; Carboxylic Acids; Organic Chemicals

Limitations and Caveats

Study terminated after one patient was enrolled. Patient discontinued due to lack of improvement in hemoglobin levels and subject compliance issues. Termination after only one subject enrolled does not allow definitive conclusions to be reached.

Results Point of Contact

• Title: Ray Pratt, MD
• Organization: Rockwell Medical

rpratt@rockwellmed.com

248 960-9009

Study Officials

• Raymond D Pratt, MD, FACP

  Rockwell Medical, Inc

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 14, 2016
• First Posted: September 19, 2016
• Study Start: January 17, 2017
• Primary Completion: March 15, 2018
• Study Completion: March 15, 2018
• Last Updated: September 22, 2020
• Results First Posted: September 2, 2020

Record last verified: 2020-09

Data Sharing

• IPD Sharing: Will share