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A One Year Open Label, Non-comparative Extension to a Randomized, Multicenter, Phase II Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration (LIC) of Repeated Doses of 5-30mg/kg/Day ICL670 Relative to Deferoxamine (DFO) in Sickle Cell Disease (SCD) Patients With Transfusional Hemosideresis (THS) [Amendment 3: Extension Prolonged to 4-years]


Phase 2
2 Years
N/A
Not Enrolling
Both
Sickle Cell Disease

Thank you

Trial Information

A One Year Open Label, Non-comparative Extension to a Randomized, Multicenter, Phase II Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration (LIC) of Repeated Doses of 5-30mg/kg/Day ICL670 Relative to Deferoxamine (DFO) in Sickle Cell Disease (SCD) Patients With Transfusional Hemosideresis (THS) [Amendment 3: Extension Prolonged to 4-years]


The treatment period started once the patient completed the core study and signed informed
consent. It is continued for up to 4 years. Safety parameters were assessed every 4 weeks.
Eye and Ear examinations were performed on a yearly basis. To further investigate the extent
of iron overload, serum ferritin, iron, and transferrin were monitored every four weeks. The
Program Safety Board monitored the safety of ICL670 during the study to evaluate and
categorize any serious case reported in association with ICL670.


Inclusion Criteria:



Patients were included who met the following criteria:

- Completion of the core [Study 0109]

- Serum ferritin greater than or equal to 500 µg/L

- Ability to comply with all study-related procedures, medications, and evaluations

- Sexually active post-menarche female patients must use double-barrier contraception,
oral contraceptive plus barrier contraceptive, or must have undergone clinically
documented total hysterectomy and/or oophorectomy, tubal ligation or be
postmenopausal defined by amenorrhea for at least 12 months.

- Written informed consent and assent by the patient and or their parents or legal
guardian.

Additional inclusion criteria for pediatric patients The definition of the term
'pediatric' for enrollment and study conduct was in accordance with local law. Parents or
the legal guardians were fully informed by the investigator as to the requirements of the
study. The pediatric patients themselves were informed according to their capabilities in
a language and terms that they were able to understand. Written informed consent was
obtained from their legal guardian on the patient's behalf in accordance with national
legislation. If capable, all patients had to also personally sign their written informed
consent.

Exclusion Criteria:

Patients who met the following criteria were to be excluded:

- History of non-compliance to medical regimens and patients who are considered
potentially unreliable and/or not cooperative

- Serum creatinine above the age-appropriate upper limit of normal within one week
prior to entry

- Patients with ALT ≥ 500 U/L within one week prior to entry

- Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to
baseline

- Pregnancy (as indicated by serum β-HCG pregnancy test for all female patients with
the potential to become pregnant) and patients who are breastfeeding

- Patients treated with systemic investigational drug within 4 weeks prior to or with
topical investigational drug within 7 days prior to the baseline visit

Other protocol-defined inclusion/exclusion criteria may apply

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Participants With Adverse Events After Start of ICL670

Outcome Description:

Safety as assessed by the number of participants with adverse event or death after the start of ICL670.

Outcome Time Frame:

0 - 60 months

Safety Issue:

Yes

Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals

Authority:

United States: Food and Drug Administration

Study ID:

CICL670A0109E1

NCT ID:

NCT01090323

Start Date:

July 2004

Completion Date:

Related Keywords:

  • Sickle Cell Disease
  • Deferasirox
  • ICL670A
  • Iron chelators
  • Sickle Cell Disease
  • Transfusional Hemosiderosis
  • Anemia, Sickle Cell

Name

Location

Howard University HospitalWashington, District of Columbia  20060
Milton S. Hershey Medical CenterHershey, Pennsylvania  17033
Loma Linda University Medical CenterLoma Linda, California  92354
New York Methodist HospitalBrooklyn, New York  11215-3609
Children's Hospital Los AngelesLos Angeles, California  90027-0700
Children's Hospital of PittsburghPittsburgh, Pennsylvania  15213
University of Illinois at ChicagoChicago, Illinois  60612
Children's Hospital of the King's DaughtersNorfolk, Virginia  23507
Montefiore Medical CenterBronx, New York  10467-2490
Tulane University Medical CenterNew Orleans, Louisiana  70112
Medical College of GeorgiaAugusta, Georgia  30912
Children's Hospital BostonBoston, Massachusetts  02115
University of Colorado Health Science CenterAurora, Colorado  80010-0510
Scott & White Memorial HospitalTemple,, Texas  76508
Texas Children's HospitalHouston, Texas  
Children's Memorial HospitalChicago, Illinois  60614
The Methodist HospitalHouston, Texas  77030
New York Presbyterian HospitalNew York, New York  10021
Santee Hematology/OncologySumter, South Carolina  29150
Karmanos Cancer InstituteDetroit, Michigan  48201
University of CincinnatiCincinnati, Ohio  45267-0502
Children's Hospital Medical CenterCincinnati, Ohio  45229
Wake Forest University Health SciencesWinston-Salem, North Carolina  27157
St. Jude's Children Research HospitalMemphis, Tennessee  38105
Children's Hospital & Research Center at OaklandOakland, California  94609
Palmetto Health RichlandColumbia, South Carolina  29203
University of South Alabama College of MedicineMobile, Alabama  36604
St Joseph Children's Hospital of TampaTampa, Florida  33607
Grady Hospital, Georgia Comprehensive Sickle Cell CenterAtlanta, Georgia  30303
LSUHSC Dept of PediatricsShreveport, Louisiana  71130
YasinPhiladelphia, Pennsylvania  19104