Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload

NCT03802916 | Completed Phase 2 Results FDA Drug

• 1 other identifier: LA61-0218
• Study Type: interventional
• Target: 30
• Locations: 4 countries
• Sites: 5

Brief Summary

Safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload.

Conditions

Iron Overload Due to Repeated Red Blood Cell Transfusions

Keywords

iron overload; chelation; deferiprone; Ferriprox

Outcome Measures

Primary Outcomes (1)

• The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern.

  Levels of the liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST) will be assessed throughout the study to determine if any patients have post-dose increases that are considered to be a safety concern. The criteria for being considered a safety concern are meeting one of the following: * For a patient whose level was within the normal range at baseline, the criterion is reaching a value of 5 times the upper limit of normal (ULN) * For a patient whose level was above the ULN at baseline, the criterion is reaching either 5 times the baseline value or 10 x ULN

  Day 28

Secondary Outcomes (2)

• The Percentage of Patients in Each Treatment Group Who Report Post-dose Occurrences of Gastrointestinal (GI) Distress.

  Day 28

• The Percentage of Patients in Each Group Who Indicate That They Prefer the Deferiprone DR Formulation Over the Immediate-release Formulation.

  Day 28

Study Arms (2)

Low dosage

EXPERIMENTAL

Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 75 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.

Drug: Deferiprone DR tablets 1000 mg (Low dosage)

High dosage

EXPERIMENTAL

Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 100 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.

Drug: Deferiprone DR tablets 1000 mg (High dosage)

Interventions

Deferiprone DR tablets 1000 mg (Low dosage) DRUG

Deferiprone DR tablets 1000 mg

Low dosage

Deferiprone DR tablets 1000 mg (High dosage) DRUG

Deferiprone DR tablets 1000 mg

High dosage

Eligibility Criteria

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

You may qualify if:

• Male or female aged ≥ 18 years.
• Diagnosis of thalassemia syndrome, sickle cell disease, or other disorder requiring a regular regimen of red blood cell transfusions.
• On a stable regimen (≥3 months) of Ferriprox tablets for the treatment of systemic iron overload.
• Absolute neutrophil count ≥1.5 x 10\^9/L at screening.
• A record of at least 12 measured alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels.

You may not qualify if:

• Receipt of any iron chelator other than Ferriprox (i.e., combination therapy) in the last 3 months, or planning to receive it at any time during the period of the study.
• ALT and/or AST value \> 5 times the upper limit of normal (ULN) at screening
• Active case of hepatitis B or C at screening.

Sponsors & Collaborators

• ApoPharma: lead

Study Sites (5)

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

New York Presbyterian Hospital/Weill Cornell Medical Center

New York, New York, 10065, United States

Location

St.Paul's Hospital

Vancouver, British Columbia, V6Z 1Y6, Canada

Location

National and Kapodistrian University of Athens, Aghia Sophia Children's Hospital

Goudi, Athens, 11527, Greece

Location

San Luigi Gonzaga University Hospital Reparto Microcitemie-Pediatria

Orbassano (TO), Regione Gonzole, 10043, Italy

Location

MeSH Terms

Conditions

Iron Overload

Interventions

Deferiprone

Condition Hierarchy (Ancestors)

Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Fernando Tricta, MD
• Organization: Chiesi Canada Corp.

ftricta@chiesi.com

1-416-558-6342

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: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Parallel Assignment

Study Record Dates

• First Submitted: January 7, 2019
• First Posted: January 14, 2019
• Study Start: March 6, 2019
• Primary Completion: December 4, 2019
• Study Completion: December 19, 2019
• Last Updated: July 16, 2021
• Results First Posted: June 18, 2021

Record last verified: 2021-06

Locations

CA (1); GR (1); US (2); IT (1)