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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02175758




Registration number
NCT02175758
Ethics application status
Date submitted
24/06/2014
Date registered
26/06/2014
Date last updated
30/04/2019

Titles & IDs
Public title
Safety and Efficacy of Sofosbuvir + Ribavirin in Adolescents and Children With Genotype 2 or 3 Chronic HCV Infection
Scientific title
A Phase 2, Open-Label, Multicenter, Multi-cohort, Single-Arm Study to Investigate the Safety and Efficacy of Sofosbuvir + Ribavirin in Adolescents and Children With Genotype 2 or 3 Chronic HCV Infection
Secondary ID [1] 0 0
2014-002283-32
Secondary ID [2] 0 0
GS-US-334-1112
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hepatitis C Virus Infection 0 0
Condition category
Condition code
Infection 0 0 0 0
Studies of infection and infectious agents
Infection 0 0 0 0
Other infectious diseases
Infection 0 0 0 0
Sexually transmitted infections
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - SOF
Treatment: Drugs - RBV

Experimental: 12 to < 18 Years Old, SOF+RBV 12 Weeks (GT 2) - Participants between 12 to \< 18 years of age with genotype (GT) 2 HCV infection weighing = 45 kg will receive SOF (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 12 weeks.

Experimental: 12 to < 18 Years Old, SOF+RBV 24 Weeks (GT 3) - Participants between 12 to \< 18 years of age with genotype 3 HCV infection weighing = 45 kg will receive SOF (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 24 weeks.

Experimental: 6 to < 12 Years Old, SOF+RBV 12 Weeks (GT 2) - Participants between 6 to \< 12 years of age with genotype 2 HCV infection weighing = 17 kg and \< 45 kg will receive SOF (2 x 100 mg tablets or 4 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 12 weeks.

Experimental: 6 to <12 Years Old, SOF+RBV 24 Weeks (GT 3) - Participants between 6 to \< 12 years of age with genotype 3 HCV infection weighing = 17 kg and \< 45 kg will receive SOF (2 x 100 mg tablets or 4 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 24 weeks.

Experimental: 3 to < 6 Years Old, SOF+RBV 12 Weeks (GT 2) - Participants between 3 to \< 6 years of age with genotype 2 HCV infection weighing = 17kg will receive SOF (4 x 50 mg oral granules) plus RBV (up to 1400 mg) for 12 weeks and those weighing \< 17 kg will receive SOF (3 x 50 mg oral granules) + RBV (up to 1400 mg) for 12 weeks.

Experimental: 3 to < 6 Years Old, SOF+RBV 24 Weeks (GT 3) - Participants between 3 to \< 6 years of age with genotype 2 HCV infection weighing = 17kg will receive SOF (4 x 50 mg oral granules) plus RBV (up to 1400 mg) for 24 weeks and those weighing \< 17 kg will receive SOF (3 x 50 mg oral granules) + RBV (up to 1400 mg) for 24 weeks.


Treatment: Drugs: SOF
SOF administered orally once daily

Treatment: Drugs: RBV
RBV oral solution or capsules will be administered orally in a divided daily dose based on weight
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
For Participants in the PK Lead-in Phase, Pharmacokinetic (PK) Parameter: AUCtau of GS-331007 (Metabolite of SOF)
Assessment method [1] 0 0
AUCtau is defined as concentration of drug over time (the area under the concentration verses time curve over the dosing interval).
Timepoint [1] 0 0
6 to < 18 years of age: predose, 0.5, 1, 2, 3, 4, 8, and 12 hours postdose on Day 7; 3 to < 6 years of age: predose, 2, 4, 8, and 12 hours postdose on Day 7
Primary outcome [2] 0 0
Percentage of Participants Who Permanently Discontinued Any Study Drug Due to an Adverse Event During the PK Lead-in Phase or the Treatment Phase
Assessment method [2] 0 0
Timepoint [2] 0 0
Up to 24 weeks
Primary outcome [3] 0 0
For the Treatment Phase, Percentage of Participants With SVR at 12 Weeks After Discontinuation of Therapy (SVR12)
Assessment method [3] 0 0
SVR12 was defined as HCV RNA \< the lower limit of quantitation (LLOQ; ie, 15 IU/mL) at 12 weeks after stopping study treatment.
Timepoint [3] 0 0
Posttreatment Week 12
Secondary outcome [1] 0 0
For Participants in the PK Lead-in Phase, Change From Baseline in HCV RNA
Assessment method [1] 0 0
Timepoint [1] 0 0
Baseline; Weeks 1, 2, 4, 8, and 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
Secondary outcome [2] 0 0
Percentage of Participants Who Permanently Discontinued Any Study Drug Due to an Adverse Event During the PK Lead-in Phase
Assessment method [2] 0 0
Timepoint [2] 0 0
Up to Day 7
Secondary outcome [3] 0 0
For the Treatment Phase, Percentage of Participants With Sustained Virologic Response (SVR) at 4 Weeks After Discontinuation of Therapy (SVR4)
Assessment method [3] 0 0
SVR4 was defined as HCV RNA \< LLOQ at 4 weeks after stopping study treatment.
Timepoint [3] 0 0
Posttreatment Week 4
Secondary outcome [4] 0 0
For the Treatment Phase, Percentage of Participants With SVR at 24 Weeks After Discontinuation of Therapy (SVR24)
Assessment method [4] 0 0
SVR24 was defined as HCV RNA \< LLOQ at 24 weeks after stopping study treatment.
Timepoint [4] 0 0
Posttreatment Week 24
Secondary outcome [5] 0 0
For the Treatment Phase, Percentage of Participants Experiencing Viral Breakthrough
Assessment method [5] 0 0
Viral breakthrough was defined as having confirmed HCV RNA = LLOQ after having previously had HCV RNA \< LLOQ while on treatment.
Timepoint [5] 0 0
Up to 24 weeks
Secondary outcome [6] 0 0
For the Treatment Phase, Percentage of Participants Experiencing Viral Relapse
Assessment method [6] 0 0
Viral relapse was defined as having confirmed HCV RNA = LLOQ during the posttreatment period having achieved HCV RNA \< LLOQ at last on-treatment visit.
Timepoint [6] 0 0
Up to Posttreatment Week 24
Secondary outcome [7] 0 0
For the Treatment Phase, Change From Baseline in HCV RNA
Assessment method [7] 0 0
Timepoint [7] 0 0
Baseline; Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
Secondary outcome [8] 0 0
For the Treatment Phase, Percentage of Participants With HCV RNA < LLOQ While On Treatment
Assessment method [8] 0 0
Timepoint [8] 0 0
Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
Secondary outcome [9] 0 0
For the Treatment Phase, Percentage of Participants With Alanine Aminotransferase (ALT) Normalization
Assessment method [9] 0 0
ALT normalization was defined as ALT \> the upper limit of normal (ULN) at baseline and ALT = ULN at each visit. One participant in the 3 to \< 6 Years Old 12 Weeks group had ALT \> ULN at Baseline, but had no other available data.
Timepoint [9] 0 0
Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only), and Posttreatment Week 4
Secondary outcome [10] 0 0
For the Treatment Phase, Change From Baseline in Height
Assessment method [10] 0 0
Timepoint [10] 0 0
Baseline; Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only), and Posttreatment Weeks 4, 12, and 24
Secondary outcome [11] 0 0
For the Treatment Phase, Change From Baseline in Weight
Assessment method [11] 0 0
Timepoint [11] 0 0
Baseline; Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only), and Posttreatment Weeks 4, 12, and 24
Secondary outcome [12] 0 0
For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Pubic Hair
Assessment method [12] 0 0
Tanner Stages is a scale that defines physical measurements of development based on external primary and secondary sex characteristics. It was used in this study to assess pubertal development with values ranging from Stage 1 (pre-pubertal characteristics) to Stage 5 (adult or mature characteristics). Any shifts (increase or decrease) in Tanner Stage from Baseline were analyzed and presented.
Timepoint [12] 0 0
Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
Secondary outcome [13] 0 0
For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Genitalia Development
Assessment method [13] 0 0
Tanner Stages is a scale that defines physical measurements of development based on external primary and secondary sex characteristics. It was used in this study to assess pubertal development with values ranging from Stage 1 (pre-pubertal characteristics) to Stage 5 (adult or mature characteristics). Any shifts (increase or decrease) in Tanner Stage from Baseline were analyzed and presented.
Timepoint [13] 0 0
Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
Secondary outcome [14] 0 0
For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Pubic Hair
Assessment method [14] 0 0
Tanner Stages is a scale that defines physical measurements of development based on external primary and secondary sex characteristics. It was used in this study to assess pubertal development with values ranging from Stage 1 (pre-pubertal characteristics) to Stage 5 (adult or mature characteristics). Any shifts (increase or decrease) in Tanner Stage from Baseline were analyzed and presented.
Timepoint [14] 0 0
Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
Secondary outcome [15] 0 0
For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Breast Development
Assessment method [15] 0 0
Tanner Stages is a scale that defines physical measurements of development based on external primary and secondary sex characteristics. It was used in this study to assess pubertal development with values ranging from Stage 1 (pre-pubertal characteristics) to Stage 5 (adult or mature characteristics). Any shifts (increase or decrease) in Tanner Stage from Baseline were analyzed and presented.
Timepoint [15] 0 0
Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
Secondary outcome [16] 0 0
For the Treatment Phase, Palatability of SOF Granules at Day 1 as Assessed by the Percentage of Participants Able/Unable to Taste the SOF Oral Granules
Assessment method [16] 0 0
Participants were asked if they were able to taste the SOF oral granules.
Timepoint [16] 0 0
Day 1

Eligibility
Key inclusion criteria
Key

* Consent of parent or legal guardian required
* Chronic HCV infection genotype 2 or 3
* Screening laboratory values within defined thresholds
* PK Lead-in only: all individuals must be treatment naive

Key
Minimum age
3 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* History of clinically significant illness or any other medical disorder that may interfere with subject treatment, assessment or compliance with the protocol
* Co-infection with HIV, acute hepatitis A virus, or hepatitis B virus
* Clinical hepatic decompensation (ie, ascites, encephalopathy or variceal hemorrhage)
* Pregnant or nursing females
* Known hypersensitivity to study medication
* Use of any prohibited concomitant medications as within 28 days of the Day 1 visit

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
7/07/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
13/09/2018
Sample size
Target
Accrual to date
Final
106
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
- Westmead
Recruitment hospital [2] 0 0
- Melbourne
Recruitment hospital [3] 0 0
- New Lambton Heights
Recruitment postcode(s) [1] 0 0
- Westmead
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment postcode(s) [3] 0 0
- New Lambton Heights
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
Country [11] 0 0
United States of America
State/province [11] 0 0
Tennessee
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
United States of America
State/province [13] 0 0
Washington
Country [14] 0 0
United States of America
State/province [14] 0 0
West Virginia
Country [15] 0 0
Belgium
State/province [15] 0 0
Brussels
Country [16] 0 0
Germany
State/province [16] 0 0
Nordrhein-westfalen
Country [17] 0 0
Germany
State/province [17] 0 0
Berlin
Country [18] 0 0
Italy
State/province [18] 0 0
Bologna
Country [19] 0 0
Italy
State/province [19] 0 0
Firenze
Country [20] 0 0
Italy
State/province [20] 0 0
Milano
Country [21] 0 0
Italy
State/province [21] 0 0
Padova
Country [22] 0 0
Italy
State/province [22] 0 0
San Giovanni Rotondo
Country [23] 0 0
Italy
State/province [23] 0 0
Torino
Country [24] 0 0
New Zealand
State/province [24] 0 0
Auckland
Country [25] 0 0
Russian Federation
State/province [25] 0 0
Moscow
Country [26] 0 0
Russian Federation
State/province [26] 0 0
Novokuznetsk
Country [27] 0 0
Russian Federation
State/province [27] 0 0
Saint-Petersburg
Country [28] 0 0
Russian Federation
State/province [28] 0 0
Tolyatti
Country [29] 0 0
United Kingdom
State/province [29] 0 0
Birmingham
Country [30] 0 0
United Kingdom
State/province [30] 0 0
Leeds
Country [31] 0 0
United Kingdom
State/province [31] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Gilead Sciences
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Gilead Study Director
Address 0 0
Gilead Sciences
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

TypeOther DetailsAttachment
Study protocol Study Protocol: Original https://cdn.clinicaltrials.gov/large-docs/58/NCT02175758/Prot_000.pdf
Study protocol Study Protocol: Amendment 1 https://cdn.clinicaltrials.gov/large-docs/58/NCT02175758/Prot_001.pdf
Study protocol Study Protocol: Amendment 2 https://cdn.clinicaltrials.gov/large-docs/58/NCT02175758/Prot_002.pdf
Study protocol Study Protocol: Amendment 3 https://cdn.clinicaltrials.gov/large-docs/58/NCT02175758/Prot_003.pdf
Study protocol Study Protocol: Amendment 4 https://cdn.clinicaltrials.gov/large-docs/58/NCT02175758/Prot_004.pdf
Study protocol Study Protocol: Amendment 5 https://cdn.clinicaltrials.gov/large-docs/58/NCT02175758/Prot_005.pdf
Study protocol Study Protocol: Amendment 6 https://cdn.clinicaltrials.gov/large-docs/58/NCT02175758/Prot_006.pdf
Statistical analysis plan https://cdn.clinicaltrials.gov/large-docs/58/NCT02175758/SAP_007.pdf



Results publications and other study-related documents


Results are available at https://clinicaltrials.gov/study/NCT02175758