Safety and Efficacy Study of Oral Ferric Iron To Treat Iron Deficiency Anaemia in Quiescent Ulcerative Colitis (AEGIS-1)

Shield Therapeutics128 enrolled

Overview

The purpose of this study is to determine whether ST10-021, an oral ferric iron preparation, is safe and effective in the treatment of iron deficiency anaemia (IDA) in subjects with non-active ulcerative colitis (UC).

As no curative treatment is currently available for ulcerative colitis (UC), treatment options are restricted to controlling symptoms, maintaining remission and preventing relapse. As such, treatment of iron deficiency anaemia (IDA), a key symptom of the disease, is integral to the medical management of UC. Iron deficiency anaemia in UC is a chronically debilitating disorder which has a significant impact on the quality of life of affected subjects. Characteristic symptoms of IDA include chronic fatigue, headache, and subtle impairment of cognitive function. Up to one third of subjects with UC suffer from recurrent anaemia, with hospitalization required in severe cases. First line standard therapy for mild to moderate IDA in UC is typically oral ferrous products (OFP), however this is often not successful. Many subjects are intolerant and suffer from continuously occurring side effects, occasional exacerbation of inflammatory lesions and failure to correct iron deficiency. Common adverse effects of OFP include nausea, epigastric discomfort and constipation, all of which are dose-related and appear especially evident in subjects with UC. As compared to oral ferrous iron, oral ferric iron can be administered with improved tolerability and the total dose exposure of unabsorbed iron within the gastrointestinal tract is significantly reduced. In addition, the iron is retained in its chelated form if not absorbed and this may reduce the risk of irritation within the gastrointestinal tract. Clinical studies conducted to date provide preliminary evidence for the therapeutic potential of ST10-021 in patients with IDA in Inflammatory Bowel Disease, including UC. The purpose of this study is to determine whether ST10-021 is safe and effective in the treatment of IDA in subjects with non-active UC. In an effort to target an underserved population, the study will include only those subjects who have failed OFP in the past, or where OFP cannot be used.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

128

Conditions

Iron Deficiency Anaemia Inflammatory Bowel Disease Ulcerative Colitis

Interventions

ST10-021DRUG

30 mg capsules to be taken orally twice a day for 12 weeks

Placebo ComparatorDRUG

Matching sugar pill to be taken orally twice a day for 12 weeks

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Competency to understand and sign the IEC/IRB approved informed consent form prior to any study mandated procedure, and willing/able to comply with study requirements * Age ≥ 18 years * Current diagnosis of quiescent UC as defined by SCCAI score of \< 4 * Current diagnosis of IDA as defined by Hb ≥ 9.5 g/dl and \<12.0 g/dl for women and ≥ 9.5 g/dl and \<13.0 g/dl for men; ferritin \< 30 µg/l * Prior OFP failure as defined per protocol * If receiving protocol-allowed immunosuppressant must be on stable dose * Females of childbearing potential must agree to use a reliable method of contraception Exclusion Criteria: * Anaemia due to any cause other than iron deficiency * Intramuscular or intravenous injection or administration of depot iron preparation, blood infusions, or erythropoietin within 3 months * Oral iron supplementation use within 1 month * Use of immunosuppressant with known effect of anaemia induction within 1 month * Vitamin B12 or Folic Acid injection/infusion within 4 weeks * Untreated Vitamin B-12 or Folic Acid deficiency * Known hypersensitivity or allergy to ST10-021 or components of the study medication, or contraindication for treatment with iron preparations * Other chronic or acute inflammatory or infectious diseases * Creatinine \> 2.0 mg/dl * AST or ALT levels ≥ 5 times the upper limit of normal * Cardiovascular, liver, renal, hematologic, gastrointestinal, immunologic, endocrine, metabolic, or central nervous system disease that may adversely affect the safety of the subject and/or efficacy of the study drug or severely limit the lifespan of the subject * History of malignancy within the past 5 years (except in situ removal of basal cell carcinoma) * Significant neurologic or psychiatric symptoms resulting in disorientation, memory impairment, or inability to report accurately that might interfere with treatment compliance, study conduct or interpretation of the results * Participation in another interventional clinical study within 30 days or during the study * Inmates of a psychiatric ward, prison, or other state institution * Investigator or any other team member involved directly or indirectly in the conduct of the clinical study * Scheduled or expected hospitalization and/or surgery during the course of the study * Females who are pregnant or lactating

Outcomes

Primary Outcomes

Change in Haemoglobin (Hb) Concentration From Baseline to Week 12 (Full Analysis Set, FAS)

Primary efficacy endpoint, defined as the change in Hb concentration from Baseline to Week 12. Baseline was defined as the pre-dose Hb concentration measured at the Randomisation Visit (Week 0). Missing Randomisation Hb values were replaced by Screening Hb values, if the randomisation was within the protocol-specified window. Hb concentration (g/dL) was analysed by a central laboratory from blood samples collected at every clinic visit: Screening, Randomisation (Week 0), Weeks 4, 8, 12, 14, 16, 20, 24, 36, 48, 64, Weeks 14 to 64 were open-label. The baseline, absolute concentration and change from baseline in Hb at all post-randomisation visits were listed and summarised by week using descriptive statistics. An analysis of covariance (ANCOVA) was used to analyse the primary endpoint; this included treatment, gender and disease as factors and baseline Hb as a covariate.

Time frame: Baseline to Week 12 - double-blind phase

Secondary Outcomes

Proportion of Subjects That Achieved ≥1 g/dL Change From Baseline in Hb Concentration at Week 12 (Full Analysis Set, FAS)

Logistic regression analysis of proportion of subjects that achieved ≥1 g/dL change from baseline in Hb concentration at Week 12 in the double-blind phase

Time frame: Subjects that achieved ≥1 g/dL change from baseline in Hb concentration at Week 12 - double-blind phase

Proportion of Subjects That Achieved ≥2 g/dL Change From Baseline in Hb Concentration at Week 12 (Full Analysis Set, FAS)

Logistic regression analysis of proportion of subjects that achieved ≥2 g/dL change from baseline in Hb concentration at Week 12 in the double-blind phase

Time frame: Baseline to Week 12 - double-blind phase

Proportion of Subjects That Achieved Hb Concentration Within Normal Range at Week 12 (Full Analysis Set, FAS)

Logistic regression analysis of proportion of subjects that achieved Hb concentration within normal range at Week 12 - end of double-blind phase

Data from ClinicalTrials.gov

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