Trial Outcomes & Findings for A Study to Characterize the PK and PD Profile of IV FCM in Pediatric Subjects 1-17 Years Old With IDA (NCT NCT02410213)

NCT ID: NCT02410213

Last Updated: 2022-07-25

Results Overview

Maximum observed serum concentration; obtained directly from the serum concentration-time profile.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

35 participants

Primary outcome timeframe

prior to dosing and 1, 2, 6, 12, 48 and 72 hours post dosing

Results posted on

2022-07-25

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort 1: Ferric Carboxymaltose (FCM) 7.5 mg/kg FCM at 7.5 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller Ferric Carboxymaltose (FCM)	Cohort 2: Ferric Carboxymaltose (FCM) 15 mg/kg FCM at 15mg/kg to a maximum single dose of 750mg iron, whichever is smaller
Overall Study STARTED	16	19
Overall Study COMPLETED	16	19
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Eligible subjects were enrolled sequentially in Cohort 1 or Cohort 2, for a total of 35 subjects.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ferric Carboxymaltose (FCM) n=35 Participants FCM at 7.5 mg/kg or 15 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller Ferric Carboxymaltose (FCM)
Age, Continuous Cohort 1 (FCM at 7.5 mg/kg to a maximum of 750mg)	9.1 years STANDARD_DEVIATION 6.13 • n=16 Participants • Eligible subjects were enrolled sequentially in Cohort 1 or Cohort 2, for a total of 35 subjects.
Age, Continuous Cohort 2 (FCM at 15 mg/kg to a maximum of 750mg)	10.3 years STANDARD_DEVIATION 5.77 • n=19 Participants • Eligible subjects were enrolled sequentially in Cohort 1 or Cohort 2, for a total of 35 subjects.
Sex: Female, Male Cohort 1 (FCM at 7.5 mg/kg to a maximum of 750mg) · Female	10 Participants n=16 Participants • Eligible subjects were enrolled sequentially in Cohort 1 or Cohort 2, for a total of 35 subjects.
Sex: Female, Male Cohort 1 (FCM at 7.5 mg/kg to a maximum of 750mg) · Male	6 Participants n=16 Participants • Eligible subjects were enrolled sequentially in Cohort 1 or Cohort 2, for a total of 35 subjects.
Sex: Female, Male Cohort 2 (FCM at 15 mg/kg to a maximum of 750mg · Female	9 Participants n=19 Participants • Eligible subjects were enrolled sequentially in Cohort 1 or Cohort 2, for a total of 35 subjects.
Sex: Female, Male Cohort 2 (FCM at 15 mg/kg to a maximum of 750mg · Male	10 Participants n=19 Participants • Eligible subjects were enrolled sequentially in Cohort 1 or Cohort 2, for a total of 35 subjects.
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=35 Participants
Race (NIH/OMB) Asian	0 Participants n=35 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=35 Participants
Race (NIH/OMB) Black or African American	0 Participants n=35 Participants
Race (NIH/OMB) White	35 Participants n=35 Participants
Race (NIH/OMB) More than one race	0 Participants n=35 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=35 Participants
Region of Enrollment Poland	30 participants n=35 Participants
Region of Enrollment Russia	5 participants n=35 Participants

PRIMARY outcome

Timeframe: prior to dosing and 1, 2, 6, 12, 48 and 72 hours post dosing

Population: In Cohort 1, pharmacokinetic parameter values are excluded for one subject due to anomalous and consistently high concentrations, and for one subject due to missing concentration data at 1 and 2 hours post-dose.

Maximum observed serum concentration; obtained directly from the serum concentration-time profile.

Outcome measures

Outcome measures
Measure	Cohort 1: Ferric Carboxymaltose (FCM) 7.5 mg n=14 Participants FCM at 7.5 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller Ferric Carboxymaltose (FCM)	Cohort 2: Ferric Carboxymaltose (FCM) 15 mg/kg n=19 Participants FCM at 15 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller
Maximum Serum Concentration (Cmax)	157 µg/mL Standard Deviation 33.8	310 µg/mL Standard Deviation 13.9

Adverse Events

Cohort 1: Ferric Carboxymaltose (FCM) 7.5 mg/kg

Serious events: 2 serious events

Other events: 9 other events

Deaths: 0 deaths

Cohort 2: Ferric Carboxymaltose (FCM) 15 mg/kg

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cohort 1: Ferric Carboxymaltose (FCM) 7.5 mg/kg n=16 participants at risk FCM at 7.5 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller Ferric Carboxymaltose (FCM)	Cohort 2: Ferric Carboxymaltose (FCM) 15 mg/kg n=19 participants at risk FCM at 15 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller Ferric Carboxymaltose (FCM)
Infections and infestations Sinusitis	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Infections and infestations Upper Respiratory Tract Infection	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).

Other adverse events

Additional Information

Angelia Butcher

Luitpold Pharmaceuticals, Inc.

Phone: 610-650-4200

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Cohort 1: Ferric Carboxymaltose (FCM) 7.5 mg/kg n=16 participants at risk FCM at 7.5 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller Ferric Carboxymaltose (FCM)	Cohort 2: Ferric Carboxymaltose (FCM) 15 mg/kg n=19 participants at risk FCM at 15 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller Ferric Carboxymaltose (FCM)
Blood and lymphatic system disorders Lymphadenitis	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Eye disorders Blepharospasm	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Gastrointestinal disorders Abdominal pain	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Gastrointestinal disorders Abdominal pain upper	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Gastrointestinal disorders Aphthous stomatitis	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Gastrointestinal disorders Constipation	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Gastrointestinal disorders Enteritis	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Gastrointestinal disorders Gastroduodentitis	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Gastrointestinal disorders Gastrooesophageal reflux disease	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Gastrointestinal disorders Haematochezia	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Gastrointestinal disorders Haemorrhoidal haemorrhage	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
General disorders Hyperthermia	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	10.5% 2/19 • Number of events 2 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
General disorders Infusion site pruritus	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
General disorders Injection site pain	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
General disorders Pyrexia	12.5% 2/16 • Number of events 2 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
General disorders Thirst	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Infections and infestations Nasopharyngitis	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Infections and infestations Pharyngitis	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Infections and infestations Respiratory tract infection	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Infections and infestations Sinusitis	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Infections and infestations Upper respiratory tract infection	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	10.5% 2/19 • Number of events 2 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Infections and infestations Urinary tract infection	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Infections and infestations Viral pharyngitis	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Investigations Alanine aminotransferase increased	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Investigations Aspartate aminotransferase increased	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Nervous system disorders Headache	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Respiratory, thoracic and mediastinal disorders Dysphonia	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Respiratory, thoracic and mediastinal disorders Dyspnoea exertional	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Respiratory, thoracic and mediastinal disorders Rhinorhoea	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	15.8% 3/19 • Number of events 3 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Respiratory, thoracic and mediastinal disorders Wheezing	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Skin and subcutaneous tissue disorders Rash	12.5% 2/16 • Number of events 2 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Skin and subcutaneous tissue disorders Rash papular	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Skin and subcutaneous tissue disorders Urticaria	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	15.8% 3/19 • Number of events 3 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Vascular disorders Hot flush	6.2% 1/16 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	0.00% 0/19 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).
Vascular disorders Hypertension	0.00% 0/16 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).	5.3% 1/19 • Number of events 1 • Initial treatment with study drug through completion of day 35 (28 days post-dose for subjects who early terminated from the study).