A Blinded, Placebo-Controlled Study of the Safety and Pharmacokinetics of Single Doses of Intravenous Deferiprone in Healthy Volunteers

NCT ID: NCT01989455

Last Updated: 2014-12-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 64 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-11-30
• Study Completion Date: 2014-03-31

Brief Summary

Single center, randomized, double-blind, placebo-controlled, adaptive sequential ascending-dose study for the evaluation of the safety, tolerability, and pharmacokinetics of single doses of deferiprone administered by intravenous infusion to healthy males and females. A bioavailability comparison will be included.

Conditions

Healthy Volunteers

Keywords

Healthy; Deferiprone; DFP; L1

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Blinding Strategy: QUADRUPLE

Study Groups

Cohort 1

Single dose of 500mg deferiprone or placebo administered via intravenous infusion.

First 2 subjects to enroll: 1 will be randomized to receive deferiprone and the other to receive placebo.

Next 14 subjects enrolled: 13 will be randomized to receive deferiprone and 1 to receive placebo.

Group Type: EXPERIMENTAL

Deferiprone

Intervention Type: DRUG

Deferiprone for infusion, 10mg/mL for intravenous infusion.

Oral dose of deferiprone: 80mg/mL oral solution. (Cohort 2)

Placebo

Intervention Type: DRUG

Placebo: normal saline solution.

Cohort 2

Single dose of 1000mg deferiprone or placebo administered via intravenous infusion and oral solution.

Randomization will be done for all 16 subjects at the same time: 14 will be randomized to receive deferiprone and 2 to receive placebo.

Subjects enrolled to cohort 2 will receive an oral dose of deferiprone.

Group Type: EXPERIMENTAL

Deferiprone

Intervention Type: DRUG

Deferiprone for infusion, 10mg/mL for intravenous infusion.

Oral dose of deferiprone: 80mg/mL oral solution. (Cohort 2)

Placebo

Intervention Type: DRUG

Placebo: normal saline solution.

Cohort 3

Single dose of 1500mg deferiprone or placebo administered via intravenous infusion.

Randomization will be done for all 16 subjects at the same time, 14 will be randomized to receive deferiprone and 2 to receive placebo.

Group Type: EXPERIMENTAL

Deferiprone

Intervention Type: DRUG

Deferiprone for infusion, 10mg/mL for intravenous infusion.

Oral dose of deferiprone: 80mg/mL oral solution. (Cohort 2)

Placebo

Intervention Type: DRUG

Placebo: normal saline solution.

Cohort 4

Single dose of 2000mg deferiprone or placebo administered via intravenous infusion.

First 2 subjects to enroll: 1 will be randomized to receive deferiprone and the other to receive placebo.

Next 14 subjects enrolled: 13 will be randomized to receive deferiprone and 1 to receive placebo.

Group Type: EXPERIMENTAL

Deferiprone

Intervention Type: DRUG

Deferiprone for infusion, 10mg/mL for intravenous infusion.

Oral dose of deferiprone: 80mg/mL oral solution. (Cohort 2)

Placebo

Intervention Type: DRUG

Placebo: normal saline solution.

Interventions

Deferiprone

Deferiprone for infusion, 10mg/mL for intravenous infusion.

Oral dose of deferiprone: 80mg/mL oral solution. (Cohort 2)

Intervention Type: DRUG

Placebo

Placebo: normal saline solution.

Intervention Type: DRUG

Other Intervention Names

Ferriprox; DFP; L1; Saline solution.

Eligibility Criteria

Inclusion Criteria

1. Healthy adult males or females, at least 18 years old but not older than 50 years.

2. Body weight at least 60kg.

3. Body Mass Index (BMI) ≥ 18.50 and ≤ 30.00 kg/m2

4. Medically healthy with clinically insignificant screening results (e.g., laboratory profiles, medical history, ECG, vital signs, physical examination.

5. Non or ex-smoker (someone who has completely stopped smoking 6 months before study start)

6. For females, negative result on a serum pregnancy test.

Exclusion Criteria

1. Absolute neutrophil count (ANC) <1.5x10^9/L.

2. History or presence of hypersensitivity to deferiprone or any related products.

3. History or presence of gastrointestinal, liver or kidney disease, or any other conditions known to interfere with the absorption, distribution, metabolism or excretion of drugs.

4. Presence of significant cardiovascular, pulmonary, hematologic, neurologic, psychiatric, endocrine, immunologic or dermatologic disease.

5. Any history of tuberculosis (TB) or prophylaxis for TB.

6. Suicidal tendency, history of seizures, head trauma with coma or craniotomy/trepanation, state of confusion or relevant psychiatric disease.

7. Inadequate venous access in either arm.

8. Presence of out-of-range cardiac interval or clinically significant ECG abnormalities (PR <110 msec or > 220 msec, QRS <60 msec or >119 msec, QTcB > 450 msec for males and >460 msec for females).

9. Use of acetaminophen, acetylsalicylic acid (ASA), or non-steroidal anti-inflammatory drugs (NSAIDs) in the previous 7 days before study start.

10. Use of any enzyme-modifying drugs, including strong inhibitors of P450 (CYP) enzymes such as cimetidine, fluoxetine, quinidine, erythromycin, ciprofloxacin, fluconazole, ketoconazole, diltiazem, and HIV antivirals OR strong inducers of CYP enzymes such as: barbiturates, carbamazepine, glucocorticoids, phenytoin, rifampin, and St. John's wart within 28 days prior to study start.

11. Maintenance therapy with any drug, or significant history of drug dependency or alcohol abuse.

12. Had a clinically significant illness during the 28 days prior to study start.

13. Receipt of an investigational product in another clinical trial within 28 days prior prior to study start.

14. Enrolment in a previous cohort of this study.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

ApoPharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Fernando Tricta, MD

Role: STUDY_CHAIR

ApoPharma Inc.

Locations

Algorithme Pharma Inc.

Mount Royal, Quebec, Canada

Countries

Canada

Other Identifiers

LA42-0113

Identifier Type: -

Identifier Source: org_study_id