PK of SOF/LED in HCV - Infected Adolescents With Haematological Disorders

NCT04353986 | Unknown Phase 3 DMC

• 1 other identifier: CL30114
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective, controlled, open-label, pharmacokinetic study. This study aims at studying the PK of sofosbuvir, ledipasvir and sofosbuvir metabolite (GS-331007) in HCV infected children with hematological Disorders. to develop predictive pharmacokinetic model for the 3 moieties in the studied population. In this study, patients in both treatment groups will receive 12 weeks of treatment with a fixed-dose combination tablet containing 400 mg of sofosbuvir and 90 mg of ledipasvir(SOF/LED) orally, once daily with food.

Conditions

HCV Infection; Beta Thalassemia Major

Keywords

Hepatitis C virus; Pharmacokinetic Modeling; Pharmacokinetics; Sofosbuvir; Ledipasvir; Direct acting antivirals; Beta thalassemia major

Outcome Measures

Primary Outcomes (2)

• Predictive Pharmacokinetic Model

  serial blood samples will be withdrawn to measure the drug level develop a Predictive Pharmacokinetic Model for sofosbuvir, ledipasvir and GS 331007

  10 days

• sustained virologic response

  sustained virologic response

  6 months

Secondary Outcomes (1)

• adverse drug reactions

  3 months

Study Arms (2)

Beta thalassemia

EXPERIMENTAL

HCV infected Beta thalassemia major adolescents

Drug: Sofosbuvir and Ledipasvir

Control

ACTIVE COMPARATOR

HCV infected, otherwise healthy, sex and age matched to the thalassemia group serving as control group

Drug: Sofosbuvir and Ledipasvir

Interventions

Sofosbuvir and Ledipasvir DRUG

fixed dose tablet containing 400 mg sofosbuvir and 90 mg ledipasvir

Also known as: SOF/LED

Beta thalassemia; Control

Eligibility Criteria

• Age: 12 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Adolescents (ages 12-18 years) and/ or weight more than 35 Kg
• Diagnosed with beta-thalassemia major and receiving regular blood transfusion
• spleenectomised
• Chronic HCV infection (defined as more than 6 months history of the disease)
• Naïve non-cirrhotic population with FIB Score: F0 to F3 as measured by Fibroscan
• Screening laboratory values of the beta-thalassemia group within the following thresholds (absolute neutrophil count \> 1500/mm3, platelets \> 7500 cells/mm3 , Serum creatinine \< 1.2 mg/dl, creatinine clearance \> 40 mL/min, albumin \>3.5 gm/dl, and aspartate transaminase (AST) and alanine transaminase (ALT) level less than 5 fold of the normal limit). Control group should have normal biochemical profile.
• Assent of the patients and consent of their legal guardians are required

You may not qualify if:

• Previous treatment for HCV.
• History of clinically significant illness or any other medical disorder that may interfere with individual's treatment, assessment or compliance with the protocol or affect the pharmacokinetics of the study drugs. Such as,
• Ongoing or untreated cancer including haematologic and hepatic cancers
• Co-infection with human immunodeficiency virus (HIV), acute hepatitis A virus or hepatitis B virus
• Clincal hepatic decompensation (i.e., ascites, encephalopathy or variceal haemorrhage)
• Renal dysfunction
• Active infection (any infection showing clinical manifestation at time of sampling)
• Known hypersensitivity to study medications
• Ongoing treatment with cyclosporine, rifampin, phenytoin, carbamazepine, phenobarbital, or amiodarone.

Sponsors & Collaborators

• Ain Shams University: lead

Study Sites (1)

Masri-Crc

Cairo, Egypt

RECRUITING

Related Publications (1)

• El-Baraky IA, Abbassi MM, Ebeid FS, Hassany M, Sabry NA, El-Sayed MH. Beta-thalassemia major alters sofosbuvir/ledipasvir exposure in Hepatitis C virus infected adolescent patients. Clin Res Hepatol Gastroenterol. 2021 Sep;45(5):101747. doi: 10.1016/j.clinre.2021.101747. Epub 2021 Jun 26.

  PMID: 34186262 DERIVED

MeSH Terms

Conditions

Hepatitis C; beta-Thalassemia

Interventions

ledipasvir, sofosbuvir drug combination

Condition Hierarchy (Ancestors)

Blood-Borne Infections; Communicable Diseases; Infections; Hepatitis, Viral, Human; Virus Diseases; Flaviviridae Infections; RNA Virus Infections; Hepatitis; Liver Diseases; Digestive System Diseases; Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Manal H El-Sayed, M.D

  Director of MARSI-CRC

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Manal H El-Sayed, M.D.

CONTACT

01227461120; manalhelsayed@yahoo.co.uk

Fatma S Ebeid, M.D.

CONTACT

01095569596; dr.fatma_ebeid@yahoo.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: This is a prospective, interventional, controlled, open-label, pharmacokinetic study.

Study Record Dates

• First Submitted: July 15, 2019
• First Posted: April 21, 2020
• Study Start: June 11, 2018
• Primary Completion: April 1, 2023
• Study Completion: December 1, 2023
• Last Updated: March 6, 2023

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will not share

Locations

EG (1)