Treatment of chronic iron overload with a chelation therapy (FBS0701)

• Register: EUCTR
• Last refreshed on: 23 March 2015
• Main ID: EUCTR2011-005675-16-IT
• Date of registration: 02/08/2012
• Prospective Registration: No
• Primary sponsor: FERROKIN BIOSCIENCES INC.
• Public title: Treatment of chronic iron overload with a chelation therapy (FBS0701)
• Scientific title: A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two FBS0701 Doses in the Treatment of Chronic Iron Overload Requiring Chelation Therapy - FBS0701 in the treatment of chronic iron overload
• Date of first enrolment: 15/05/2012
• Target sample size: 50
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-005675-16
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: - same IMP used at different dosage Number of treatment arms in the trial: 2

Countries of recruitment

Canada, Italy, Lebanon, Turkey, United States

Contacts

• Name: Clinical Research Dpt
• Affiliation: Ferrokin Bioscience Ltd

• Name: Clinical Research Dpt
• Affiliation: Ferrokin Bioscience Ltd

Key inclusion & exclusion criteria

Inclusion criteria:
1. The patient must be willing to sign the informed consent form approved and must be able to do so. 2. Age: 18-60 years at screening 3. Patients who, until then, had more than 20 transfusions and iron overload are affected by transfusion-dependent, requiring chronic treatment with a chelating agent of iron. Please note that Patients with sickle cell disease who carry out periodic exchange transfusions, and patients with iron overload with thalassemia intermedia are eligible to participate in the study. 4. Agree to discontinue any iron chelation therapy in progress, for a minimum period of one to five days before the first dose of FBS0701, for 24 weeks of study days and 1 week after the last dose, for a total of about 26 weeks. 5. Agree to stay on an empty stomach two hours before and one hour after each dose. 6. Present a serum ferritin> 500 ng / ml at screening. 7. The concentration of iron in the liver, the extent of reference (from Day -14 to Day -7) must be = 5 mg of iron per g (dry weight equivalent) determined by MRI FerriScan. 8. The average concentrations of the three previous pre-transfusion hemoglobin must be = 7.5 g / dl. 9. The patient agrees to use a contraceptive method approved by the time of screening up to 28 days after the last dose of study medication. Among the accepted methods of contraception are cited as follows: condoms, birth control pills approved, patches, implants or injections, vaginal diaphragm with spermicide, intrauterine device (IUD) and / or surgical sterilization (vasectomy or tubal ligation at least six months prior to administration of doses). Patients who practice abstinence must agree to use an approved method of contraception, if they become sexually active during the course of the study. All women will be fertile again subjected to pregnancy tests every month during the screening and for the duration of the study.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Following clinical evaluation, physical examinations or investigations carried out during the screening, the principal investigator considered the patient unsuitable for participation in the study. 2. Non-elective hospitalization in the last 30 days before the exams reference. 3. Evidence of the presence of oral diseases, cardiovascular, gastrointestinal, hepatic, renal, endocrine, pulmonary, neurological, psychiatric, immunological, bone marrow or skin, clinically relevant, by the principal investigator confirmed that the administration of FBS0701 inadvisable. 4. Iron overload due to causes other than siderosis transfusion-dependent. 5. Found significant renal insufficiency, eg. serum creatinine greater than 1.5 the upper limit of normal, proteinuria greater than 1 g per day or calculated creatinine clearance less than 40 ml / minute. 6. Severe iron overload, including: a. Of MRI T2 *> 10 ms; b. liver iron concentration determined by R2 MRI of> 30 mg / g liver (dry weight). 7. Known sensitivity to magnesium stearate, sodium croscarmellose, or a FBS0701. 8. Platelet count less than 100,000 and / or absolute neutrophil count less than 1500/mm3 at the screening. 9. Poor venous access that precludes the possibility of blood draws for laboratory tests required for safety assessment 10. ALT at screening = 200 IU / L. 11. Use of any investigational drug within 30 days preceding the baseline procedures. 12. Pregnant or breastfeeding


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Patients with transfusional iron overload, with the following primary diagnosis:hereditary anemia (such as sickle cell disease),ß-thalassemia and Diamond Blackfan anemia

Intervention(s)

Product Name: FBS0701 CAPSULE
Product Code: FBS0701
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: iron chelating agents
CAS Number: 1173092-59-5
Current Sponsor code: FBS0701
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-

Product Name: FBS0701 CAPSULE
Product Code: FBS0701
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Iron chelating agents
CAS Number: 1173092-59-5
Current Sponsor code: FBS0701
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-

Product Name: FBS0701 CAPSULE
Product Code: FBS0701
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Iron chelating agents
CAS Number: 1173092-59-5
Current Sponsor code: FBS0701
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-

Product Name: FBS0701 CAPSULE
Product Code: FBS0701
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: iron chelating agents
CAS Number: 1173092-59-5
Current Sponsor code: FBS0701
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 375-

Product Name: FBS0701 CAPSULE
Product Code: FBS0701
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: iron chelating agents
CAS Number: 1173092-59-5
Current Sponsor code: FBS0701
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 500-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: NA

Secondary Objective: NA

Primary end point(s): - Pharmacokinetic parameters of FBS0701 after a period of 8-16 weeks of exposure to FBS0701 capsules of 50 mg and 75 mg / kg per day. -Change in liver iron concentration determined by magnetic resonance imaging (MRI) of two doses of FSB0701 after 12 and 24 weeks of administration -Safety and tolerability of two doses of FBS0701 based on clinical assessments (such as incidence and severity of adverse events, physical examinations including vital signs, clinical laboratory values ??and ECG).

Main Objective: Evaluate, in patients with transfusion-dependent iron overload, whose primary diagnosis is hereditary or congenital anemia: - Pharmacokinetic parameters provided a constant (steady state) of two doses of FBS0701; - Changes in the concentrations of iron in the liver, after 12 and 24 weeks following administration of two doses of FBS0701; - The safety and tolerability of two doses of FBS0701, administered daily for 24 weeks.

Secondary Outcome(s)

Secondary end point(s): NA

Timepoint(s) of evaluation of this end point: NA

Secondary ID(s)

FBS0701-CTP-16

Source(s) of Monetary Support

Ferrokin Inc