A Study Of The Safety, Tolerability And Effective Of Voriconazole For The Treatment Of Serious Candida Infection And Candida Infection Of The Throat In Pediatric Patients

• Sponsor: Pfizer (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT01092832
• Collaborator: (none)
• Enrollment: 23
• Locations: 14
• Arm: 1
• Duration (Months): 32
• Patients Per Site: 1.6
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

The purpose of this study is to determine whether voriconazole is safe and effective for the treatment of serious Candida infection and Candida infection of the esophagus in children and adolescents.

Condition or Disease	Intervention/Treatment	Phase
Candidiasis	Drug: voriconazole	Phase 3

Study Design

• Study Type: Interventional
• Actual Enrollment: 23 participants
• Allocation: Non-Randomized
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Prospective, Open-label, Non-comparative Study To Assess The Safety, Tolerability And Efficacy Of Voriconazole For The Primary And Salvage Treatment Of Invasive Candidiasis, Candidemia, And Esophageal Candidiasis In Pediatric Subjects
• Study Start Date: Oct 1, 2010
• Actual Primary Completion Date: May 1, 2013
• Actual Study Completion Date: Jun 1, 2013

Arms and Interventions

Arm

Intervention/Treatment

Experimental: Active voriconazole All subjects in this study will receive active voriconazole in an open-label fashion; there is no comparator in this study.

Drug: voriconazole; Subjects 12 - <18 yrs (excluding subjects 12 - 14 yrs weighing <50kg): For IC, the loading dose is 6 mg/kg IV q12h on Day 1, followed by 4 mg/kg IV q12h thereafter. Oral therapy, if given, will be administered at a dose of 200 mg q12h. For EC, no loading dose is required, and subjects will be initiated on 3 mg/kg IV q12h. Oral therapy, if given, will be administered at a dose of 200 mg q12h. Subjects 2 - <12 yrs, and subjects 12 - 14 weighing <50kg: For IC, the loading dose is 9 mg/kg IV q12h on Day 1, followed by 8 mg/kg IV q12h thereafter. For EC, no loading dose is required, and subjects will be initiated on 4 mg/kg IV q12. Oral therapy, if given, will be administered at a dose of 9 mg/kg q12h (maximum initial dose of 300 mg). Subjects will be treated for a minimum of 14 days for invasive candidiasis/candidemia or a minimum of 7 days for esophageal candidiasis. Subjects will be treated for up to a maximum of 42 days.; Other Names: Vfend

Outcome Measures

Primary Outcome Measures

1. Percentage of Participants With Adverse Events - Overall Summary [Baseline up to 1 month follow-up]

   Percentage of participants with adverse events (AEs), serious adverse events (SAEs), severe AEs, who discontinued due to AEs, or who had dose redued or temporarily discontinued due to AEs.

Secondary Outcome Measures

1. Percentage of Participants With a Global Response of Success at End of Treatment (EOT) [EOT (from 7 to 42 days of treatment)]

   Global response was determined programmatically based on investigator assessment of clinical and microbiological response. Global response of success was defined as clinical cure or improvement AND microbiological eradication or presumed eradication. Exact 95 percent (%) confidence interval for binomial proportions using Clopper-Pearson method.

2. All-Cause Mortality - Number of Participant Deaths [Day 28 and 1 Month Follow-up]

3. Time to Death [Baseline up to 1 month follow-up]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 2 Years to 17 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Male or female 2 to <18 years of age; Subjects 2 to <12 years of age will be permitted to enroll in this study only after a Pfizer sponsored pharmacokinetic study confirms the proposed dosage corresponding to this age group is appropriate.

• Patients with confirmed Candida infection of the blood, body tissues, or the esophagus.

• Patient's doctor feels voriconazole is an appropriate choice of therapy.

Exclusion Criteria:

• A known allergy to voriconazole or to azole to antifungal drugs.

• Females who are pregnant, lactating (breast feeding) or planning a pregnancy during the course of the study, or who are of childbearing potential and not using highly effective method of birth control.

• A patient who is receiving treatment with a drug know to interfere with the heart's electrical system (QTc prolongation).

• A patient who is receiving treatment with a drug that is not permitted to be used with voriconazole.

• For primary therapy: a patient who has received more than 48 hours of antifungal therapy for the current episode of Candida infection.

• A patient with significant underlying liver disease at the time of enrollment in the study.

• A patient with significant renal disease (CrCl < 50 ml/min) at the time of enrollment in the study.

• A patient with a high likelihood of death within 72 hours of study enrollment due to factors unrelated to Candida infection.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology	Wuhan	Hubei	China	430030
2	Beijing Children's Hospital, Capital University of Medical Sciences	Beijing		China	100045
3	Fakultni nemocnice Brno - Klinika detske onkologie	Brno		Czech Republic	625 00
4	Department of Paediatrics and Adolescent Medicine	Hong Kong		Hong Kong	0
5	Queen Mary Hospital	Hong Kong		Hong Kong
6	The Chinese University of Hong Kong, Prince of Wales Hospital	Shatin, N.T.		Hong Kong
7	Semmelweis Egyetem, II. sz. Szemeszeti Klinika	Budapest		Hungary	1085
8	Semmelweis Egyetem, II. sz. Gyermekgyogyaszati Klinika	Budapest		Hungary	1094
9	Fovarosi Onkormanyzat Egyesitett Szent Istvan és Szent Laszlo Korhaz-Rendelointezet	Budapest		Hungary	1097
10	Instituto Nacional de Pediatria	Colonia Insurgentes Cuicuilco, Delegacion Coyoacan	DF	Mexico	04530
11	Medical Research Laboratory Philippine General Hospital University of the Philippines	Ermita,	Manila	Philippines	1000
12	Rm. 112 ICHHD, National Institutes of Health-University of the Philippines Manila	Manila		Philippines	1000
13	Oddzial Pediatryczny I- Hematologiczno-Onkologiczny	Olsztyn		Poland	10-561
14	Detska fakultna nemocnica s poliklinikou Bratislava	Bratislava		Slovakia	833 40

Sponsors and Collaborators

• Pfizer

Investigators

• Study Director: Pfizer CT.gov Call Center, Pfizer

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• To obtain contact information for a study center near you, click here.

Publications

None provided.

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT01092832
• Other Study ID Numbers: A1501085; 2009-012848-16
• First Posted: Mar 25, 2010
• Last Update Posted: Jun 23, 2016
• Last Verified: May 1, 2016

• Keywords provided by Pfizer: Invasive Candidiasis Candidemia Esophageal Candidiasis
• Additional relevant MeSH terms: Candidiasis; Voriconazole

Study Results

Participant Flow

Arm/Group Title	Voriconazole: 2 to <12 Years	Voriconazole: 12 to <18 Years
Arm/Group Description	Participants aged 2 to less than (<)12 years (and young adolescents aged 12 to 14 years weighing <50 kilograms [kg]) with invasive candidiasis/candidemia (ICC) received a loading dose of voriconazole 9 milligrams per kg (mg/kg), intravenously (IV), every 12 hours (q12h) for the first 24 hours, followed by maintenance dosing of voriconazole 8 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. Participants with esophageal candidiasis (EC) received voriconazole 4 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to oral (PO) therapy and received voriconazole 9 mg/kg, PO, q12h (maximum dose of 350 mg). Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.	Participants aged 12 to <18 years (excluding those aged 12-14 years weighing <50 kg) with ICC received a loading dose of voriconazole 6 mg/kg, IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 4 mg/kg, IV, q12h for a minimum of 7 days of IV therapy. Participants with EC received voriconazole 3 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 200 mg, PO, q12h. Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.
Period Title: Overall Study
STARTED	14	8
COMPLETED	13	8
NOT COMPLETED	1	0

Baseline Characteristics

Arm/Group Title	Voriconazole: 2 to <12 Years	Voriconazole: 12 to <18 Years	Total
Arm/Group Description	Participants aged 2 to <12 years (and young adolescents aged 12 to 14 years weighing <50 kg) with ICC received a loading dose of voriconazole 9 mg/kg), IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 8 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. Participants with EC received voriconazole 4 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 9 mg/kg, PO, q12h (maximum dose of 350 mg). Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.	Participants aged 12 to <18 years (excluding those aged 12-14 years weighing <50 kg) with ICC received a loading dose of voriconazole 6 mg/kg, IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 4 mg/kg, IV, q12h for a minimum of 7 days of IV therapy. Participants with EC received voriconazole 3 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 200 mg, PO, q12h. Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.	Total of all reporting groups
Overall Participants	14	8	22
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	6.8 (2.9)	14.4 (1.7)	9.5 (4.5)
Sex: Female, Male (Count of Participants)
Female	8 57.1%	6 75%	14 63.6%
Male	6 42.9%	2 25%	8 36.4%

Outcome Measures

1. Primary Outcome

Title	Percentage of Participants With Adverse Events - Overall Summary
Description	Percentage of participants with adverse events (AEs), serious adverse events (SAEs), severe AEs, who discontinued due to AEs, or who had dose redued or temporarily discontinued due to AEs.
Time Frame	Baseline up to 1 month follow-up

Outcome Measure Data

Analysis Population Description

Safety population

Arm/Group Title	Voriconazole: 2 to <12 Years	Voriconazole: 12 to <18 Years
Arm/Group Description	Participants aged 2 to <12 years (and young adolescents aged 12 to 14 years weighing <50 kg) with ICC received a loading dose of voriconazole 9 mg/kg), IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 8 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. Participants with EC received voriconazole 4 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 9 mg/kg, PO, q12h (maximum dose of 350 mg). Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.	Participants aged 12 to <18 years (excluding those aged 12-14 years weighing <50 kg) with ICC received a loading dose of voriconazole 6 mg/kg, IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 4 mg/kg, IV, q12h for a minimum of 7 days of IV therapy. Participants with EC received voriconazole 3 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 200 mg, PO, q12h. Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.
Measure Participants	14	8
With AEs	92.9 663.6%	75.0 937.5%
With SAEs	14.3 102.1%	12.5 156.3%
With severe AEs	28.6 204.3%	37.5 468.8%
Discontinued due to AEs	14.3 102.1%	25.0 312.5%
Dose reduced/temporary discontinuation due to AE	21.4 152.9%	0 0%

2. Secondary Outcome

Title	Percentage of Participants With a Global Response of Success at End of Treatment (EOT)
Description	Global response was determined programmatically based on investigator assessment of clinical and microbiological response. Global response of success was defined as clinical cure or improvement AND microbiological eradication or presumed eradication. Exact 95 percent (%) confidence interval for binomial proportions using Clopper-Pearson method.
Time Frame	EOT (from 7 to 42 days of treatment)

Outcome Measure Data

Analysis Population Description

Modified Intent-to-Treat (MITT) Population: all participants who received at least 1 dose of study medication and who have confirmed ICC, EC or participants with EC who do not have confirmation of EC by esophagoscopy, but who had at least confirmation of oropharyngeal candidiasis.

Arm/Group Title	Voriconazole: 2 to <12 Years	Voriconazole: 12 to <18 Years
Arm/Group Description	Participants aged 2 to <12 years (and young adolescents aged 12 to 14 years weighing <50 kg) with ICC received a loading dose of voriconazole 9 mg/kg), IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 8 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. Participants with EC received voriconazole 4 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 9 mg/kg, PO, q12h (maximum dose of 350 mg). Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.	Participants aged 12 to <18 years (excluding those aged 12-14 years weighing <50 kg) with ICC received a loading dose of voriconazole 6 mg/kg, IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 4 mg/kg, IV, q12h for a minimum of 7 days of IV therapy. Participants with EC received voriconazole 3 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 200 mg, PO, q12h. Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.
Measure Participants	9	8
Number (95% Confidence Interval) [percentage of participants]	88.9 635%	62.5 781.3%

3. Secondary Outcome

Title	All-Cause Mortality - Number of Participant Deaths
Time Frame	Day 28 and 1 Month Follow-up

Outcome Measure Data

Analysis Population Description

Safety population

Arm/Group Title	Voriconazole: 2 to <12 Years	Voriconazole: 12 to <18 Years
Arm/Group Description	Participants aged 2 to <12 years (and young adolescents aged 12 to 14 years weighing <50 kg) with ICC received a loading dose of voriconazole 9 mg/kg), IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 8 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. Participants with EC received voriconazole 4 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 9 mg/kg, PO, q12h (maximum dose of 350 mg). Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.	Participants aged 12 to <18 years (excluding those aged 12-14 years weighing <50 kg) with ICC received a loading dose of voriconazole 6 mg/kg, IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 4 mg/kg, IV, q12h for a minimum of 7 days of IV therapy. Participants with EC received voriconazole 3 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 200 mg, PO, q12h. Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.
Measure Participants	14	8
Day 28	0 0%	0 0%
1 Month Follow-up	0 0%	0 0%

4. Secondary Outcome

Title	Time to Death
Time Frame	Baseline up to 1 month follow-up

Outcome Measure Data

Analysis Population Description

No participants died within the safety reporting period, therefore time to death was not applicable.

Arm/Group Title	Voriconazole: 2 to <12 Years	Voriconazole: 12 to <18 Years
Arm/Group Description	Participants aged 2 to <12 years (and young adolescents aged 12 to 14 years weighing <50 kg) with ICC received a loading dose of voriconazole 9 mg/kg), IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 8 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. Participants with EC received voriconazole 4 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 9 mg/kg, PO, q12h (maximum dose of 350 mg). Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.	Participants aged 12 to <18 years (excluding those aged 12-14 years weighing <50 kg) with ICC received a loading dose of voriconazole 6 mg/kg, IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 4 mg/kg, IV, q12h for a minimum of 7 days of IV therapy. Participants with EC received voriconazole 3 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 200 mg, PO, q12h. Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.
Measure Participants	0	0

Adverse Events

Time Frame	Day 1 up to Day 49 (7 days after the last dose of study drug)
Adverse Event Reporting Description	The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another participant, or one participant may have experienced both a serious and nonserious event during the study.
Arm/Group Title	Voriconazole: 2 to <12 Years		Voriconazole: 12 to <18 Years
Arm/Group Description	Participants aged 2 to <12 years (and young adolescents aged 12 to 14 years weighing <50 kg) with ICC received a loading dose of voriconazole 9 mg/kg), IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 8 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. Participants with EC received voriconazole 4 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 9 mg/kg, PO, q12h (maximum dose of 350 mg). Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.		Participants aged 12 to <18 years (excluding those aged 12-14 years weighing <50 kg) with ICC received a loading dose of voriconazole 6 mg/kg, IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 4 mg/kg, IV, q12h for a minimum of 7 days of IV therapy. Participants with EC received voriconazole 3 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the participant was clinically stable, participants were switched to PO therapy and received voriconazole 200 mg, PO, q12h. Voriconazole was administered for at least 7 days (participants with EC) or 14 days (participants with ICC) after last positive blood culture up to a maximum of 42 days of treatment.
All Cause Mortality
	Voriconazole: 2 to <12 Years		Voriconazole: 12 to <18 Years
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	Voriconazole: 2 to <12 Years		Voriconazole: 12 to <18 Years
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	2/14 (14.3%)		1/8 (12.5%)
Blood and lymphatic system disorders
Febrile neutropenia	1/14 (7.1%)		0/8 (0%)
Infections and infestations
Pneumonia	1/14 (7.1%)		0/8 (0%)
Splenic candidiasis	0/14 (0%)		1/8 (12.5%)
Other (Not Including Serious) Adverse Events
	Voriconazole: 2 to <12 Years		Voriconazole: 12 to <18 Years
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	13/14 (92.9%)		6/8 (75%)
Blood and lymphatic system disorders
Anaemia	1/14 (7.1%)		0/8 (0%)
Hypothrombinaemia	0/14 (0%)		1/8 (12.5%)
Leukocytosis	1/14 (7.1%)		0/8 (0%)
Leukopenia	1/14 (7.1%)		0/8 (0%)
Neutropenia	1/14 (7.1%)		0/8 (0%)
Platelet disorder	1/14 (7.1%)		0/8 (0%)
Thrombocytopenia	1/14 (7.1%)		1/8 (12.5%)
Cardiac disorders
Pericardial effusion	1/14 (7.1%)		0/8 (0%)
Tachycardia	0/14 (0%)		1/8 (12.5%)
Ear and labyrinth disorders
Vertigo	0/14 (0%)		1/8 (12.5%)
Eye disorders
Amaurosis	1/14 (7.1%)		0/8 (0%)
Conjunctivitis	1/14 (7.1%)		0/8 (0%)
Corneal opacity	1/14 (7.1%)		0/8 (0%)
Eye pruritus	0/14 (0%)		1/8 (12.5%)
Eyelid disorder	1/14 (7.1%)		0/8 (0%)
Photophobia	2/14 (14.3%)		1/8 (12.5%)
Retinal disorder	0/14 (0%)		1/8 (12.5%)
Visual acuity reduced	1/14 (7.1%)		0/8 (0%)
Gastrointestinal disorders
Abdominal pain	1/14 (7.1%)		0/8 (0%)
Ascites	1/14 (7.1%)		0/8 (0%)
Constipation	1/14 (7.1%)		1/8 (12.5%)
Nausea	0/14 (0%)		1/8 (12.5%)
Oesophagitis	0/14 (0%)		2/8 (25%)
Tongue ulceration	1/14 (7.1%)		0/8 (0%)
Vomiting	1/14 (7.1%)		1/8 (12.5%)
General disorders
Device occlusion	0/14 (0%)		1/8 (12.5%)
Hypothermia	2/14 (14.3%)		1/8 (12.5%)
Pyrexia	2/14 (14.3%)		1/8 (12.5%)
Hepatobiliary disorders
Gallbladder disorder	1/14 (7.1%)		0/8 (0%)
Hepatosplenomegaly	1/14 (7.1%)		0/8 (0%)
Hyperbilirubinaemia	1/14 (7.1%)		0/8 (0%)
Jaundice	1/14 (7.1%)		0/8 (0%)
Liver disorder	1/14 (7.1%)		0/8 (0%)
Infections and infestations
Anorectal cellulitis	1/14 (7.1%)		0/8 (0%)
Bone abscess	1/14 (7.1%)		0/8 (0%)
Bronchopulmonary aspergillosis	0/14 (0%)		1/8 (12.5%)
Cellulitis	1/14 (7.1%)		0/8 (0%)
Oral herpes	1/14 (7.1%)		0/8 (0%)
Rhinitis	1/14 (7.1%)		0/8 (0%)
Splenic candidiasis	0/14 (0%)		1/8 (12.5%)
Injury, poisoning and procedural complications
Incision site pain	0/14 (0%)		1/8 (12.5%)
Refractoriness to platelet transfusion	0/14 (0%)		1/8 (12.5%)
Transplant failure	0/14 (0%)		1/8 (12.5%)
Investigations
Alanine aminotransferase abnormal	3/14 (21.4%)		0/8 (0%)
Alanine aminotransferase increased	1/14 (7.1%)		0/8 (0%)
Aspartate aminotransferase abnormal	1/14 (7.1%)		0/8 (0%)
Aspartate aminotransferase increased	1/14 (7.1%)		0/8 (0%)
Blood alkaline phophatase abnormal	1/14 (7.1%)		0/8 (0%)
Blood triglycerides increased	0/14 (0%)		1/8 (12.5%)
Drug level decreased	0/14 (0%)		1/8 (12.5%)
Gamma-glutamyltransferase abnormal	2/14 (14.3%)		0/8 (0%)
Gamma-glutamyltransferase increased	0/14 (0%)		1/8 (12.5%)
Haematocrit abnormal	1/14 (7.1%)		0/8 (0%)
Hepatic enzyme increased	1/14 (7.1%)		0/8 (0%)
Lymphocyte percentage abnormal	1/14 (7.1%)		0/8 (0%)
Monocyte count abnormal	1/14 (7.1%)		0/8 (0%)
Staphylococcus test positive	0/14 (0%)		1/8 (12.5%)
Metabolism and nutrition disorders
Hyperglycaemia	1/14 (7.1%)		1/8 (12.5%)
Hyperkalaemia	1/14 (7.1%)		0/8 (0%)
Hypermagnesaemia	1/14 (7.1%)		0/8 (0%)
Hypertriglyceridaemia	1/14 (7.1%)		0/8 (0%)
Hypoalbuminaemia	0/14 (0%)		1/8 (12.5%)
Hypocalcaemia	1/14 (7.1%)		0/8 (0%)
Hypochloraemia	1/14 (7.1%)		0/8 (0%)
Hypoglycaemia	1/14 (7.1%)		0/8 (0%)
Hypokalaemia	2/14 (14.3%)		0/8 (0%)
Hyponatraemia	1/14 (7.1%)		0/8 (0%)
Hypophagia	0/14 (0%)		1/8 (12.5%)
Hypophosphataemia	1/14 (7.1%)		1/8 (12.5%)
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain	0/14 (0%)		1/8 (12.5%)
Nervous system disorders
Paraesthesia	0/14 (0%)		1/8 (12.5%)
Renal and urinary disorders
Cystitis haemorrhagic	1/14 (7.1%)		0/8 (0%)
Haematuria	1/14 (7.1%)		0/8 (0%)
Reproductive system and breast disorders
Testicular mass	1/14 (7.1%)		0/8 (0%)
Respiratory, thoracic and mediastinal disorders
Atelectasis	0/14 (0%)		1/8 (12.5%)
Bronchospasm	1/14 (7.1%)		0/8 (0%)
Cough	1/14 (7.1%)		0/8 (0%)
Epistaxis	1/14 (7.1%)		0/8 (0%)
Haemoptysis	0/14 (0%)		1/8 (12.5%)
Hydrothorax	1/14 (7.1%)		0/8 (0%)
Hypoventilation	0/14 (0%)		1/8 (12.5%)
Pharyngeal erythema	1/14 (7.1%)		0/8 (0%)
Pneumothorax	0/14 (0%)		1/8 (12.5%)
Respiratory disorder	1/14 (7.1%)		0/8 (0%)
Skin and subcutaneous tissue disorders
Dermatitis	1/14 (7.1%)		0/8 (0%)
Dermatosis	1/14 (7.1%)		0/8 (0%)
Purpura	1/14 (7.1%)		0/8 (0%)
Rash	3/14 (21.4%)		0/8 (0%)
Scab	1/14 (7.1%)		0/8 (0%)
Vascular disorders
Hypertension	2/14 (14.3%)		0/8 (0%)
Phlebitis	0/14 (0%)		1/8 (12.5%)
Venoocclusive disease	1/14 (7.1%)		0/8 (0%)

Limitations/Caveats

The study was prematurely terminated due to slow enrollment. The study was not terminated due to any safety issues or concerns. Interpretation of the data are limited due to the small sample size and descriptive design.

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Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

Pfizer has the right to review disclosures, requesting a delay of less than 60 days. PI will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. PI may not disclose previously undisclosed confidential information other than study results.