PK of SOF/LED in HCV - Infected Adolescents With Haematological Disorders
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ClinicalTrials.gov Identifier: NCT04353986 |
Recruitment Status : Unknown
Verified April 2020 by Manal Hamdy El-Sayed, Ain Shams University.
Recruitment status was: Recruiting
First Posted : April 21, 2020
Last Update Posted : April 21, 2020
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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.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
HCV Infection Beta Thalassemia Major | Drug: Sofosbuvir and Ledipasvir | Phase 3 |
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, as prescribed by the attending physician. Twelve eligible HCV-infected patients with hematological disorder and 12 matching HCV control patients without haematological disorder or comorbidities will be enrolled in the study. At baseline, careful history of the recruited patients including demographic characteristics (age, height, weight, and gender), comorbidities, medication history, familial history, social history, blood transfusion history, and baseline laboratory tests will be documented.
The baseline laboratory tests will include renal function tests (serum creatinine), liver function tests (bilirubin, albumin, AST, and ALT), international normalised ratio (INR), alpha fetoprotein (AFP), complete blood count (CBC), degree of liver fibrosis by Fibroscan,viral load by PCR and HCV genotype Follow-up will be done for all participants at baseline, after 10 days of treatment for the evaluation of the steady state PK parameters of SOF/LED in those patients, after 12 weeks of treatment, and after 12 weeks from the end of treatment. For a total of 4 follow-up visits.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 24 participants |
Allocation: | Non-Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | This is a prospective, interventional, controlled, open-label, pharmacokinetic study. |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Pharmacokinetics of Sofosbuvir and Ledipasvir in Hepatitis C Virus - Infected Adolescent Patients With Haematological Disorders |
Actual Study Start Date : | June 11, 2018 |
Estimated Primary Completion Date : | April 2020 |
Estimated Study Completion Date : | December 2020 |

Arm | Intervention/treatment |
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Experimental: Beta thalassemia
HCV infected Beta thalassemia major adolescents
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Drug: Sofosbuvir and Ledipasvir
fixed dose tablet containing 400 mg sofosbuvir and 90 mg ledipasvir
Other Name: SOF/LED |
Active Comparator: Control
HCV infected, otherwise healthy, sex and age matched to the thalassemia group serving as control group
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Drug: Sofosbuvir and Ledipasvir
fixed dose tablet containing 400 mg sofosbuvir and 90 mg ledipasvir
Other Name: SOF/LED |
- Predictive Pharmacokinetic Model [ Time Frame: 10 days ]serial blood samples will be withdrawn to measure the drug level develop a Predictive Pharmacokinetic Model for sofosbuvir, ledipasvir and GS 331007
- sustained virologic response [ Time Frame: 6 months ]sustained virologic response
- adverse drug reactions [ Time Frame: 3 months ]record any adverse drug reactions experienced by the patients

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 12 Years to 18 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Inclusion criteria:
- 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
Exclusion Criteria:
- Previous treatment for HCV.
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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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04353986
Contact: Manal H El-Sayed, M.D. | 01227461120 ext 002 | manalhelsayed@yahoo.co.uk | |
Contact: Fatma S Ebeid, M.D. | 01095569596 ext 002 | dr.fatma_ebeid@yahoo.com |
Egypt | |
Masri-Crc | Recruiting |
Cairo, Egypt | |
Contact: Manal H El-Sayed, M.D. 01227461120 ext 002 manalhelsayed@yahoo.co.uk | |
Contact: Fatma S Ebeid, M.D. 01095569596 ext 002 dr.fatma_ebeid@yahoo.com | |
Sub-Investigator: Eman A El-Baraky, Ms.C. | |
Sub-Investigator: Nirmeen A Sabry, Ph.D. | |
Principal Investigator: Maggie M Abbassi, Ph.D |
Principal Investigator: | Manal H El-Sayed, M.D | Director of MARSI-CRC |
Responsible Party: | Manal Hamdy El-Sayed, Professor, Ain Shams University |
ClinicalTrials.gov Identifier: | NCT04353986 |
Other Study ID Numbers: |
CL30114 |
First Posted: | April 21, 2020 Key Record Dates |
Last Update Posted: | April 21, 2020 |
Last Verified: | April 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Hepatitis C virus Pharmacokinetic Modeling Pharmacokinetics Sofosbuvir |
Ledipasvir Direct acting antivirals Beta thalassemia major |
Hepatitis C Thalassemia beta-Thalassemia Hematologic Diseases Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hemoglobinopathies Genetic Diseases, Inborn Blood-Borne Infections Communicable Diseases Infections |
Hepatitis, Viral, Human Virus Diseases Flaviviridae Infections RNA Virus Infections Hepatitis Liver Diseases Digestive System Diseases Sofosbuvir Ledipasvir Ledipasvir, sofosbuvir drug combination Antiviral Agents Anti-Infective Agents |