| Protocol No: | ECCT/22/03/06 | Date of Protocol: | 16-08-2021 |
| Study Title: | A Phase 2a Randomized, Double-Blind, Placebo-Controlled Study to Characterize the Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients with Sickle Cell Disease |
| Study Objectives: | The objectives of this study are: To characterize the pharmacokinetics/pharmacodynamics (PK/PD) of rifaximin ER and DER in subjects with sickle cell disease (SCD) and to assess the safety and tolerability of rifaximin (Extended Release) ER and Delayed Extended Release (DER). |
| Laymans Summary: | The objective of this study is to understand/characterize the pharmacokinetic/PK (how our bodies affect the drug) and the Pharmacodynamics/PD (meaning how the drug affects our bodies) of a drug called Rifaximin, which is used in treatment of patients with sickle cell disease. Of the current treatment options available for sickle cell disease, only bone marrow transplant is a permanent cure, but few patients qualify for this treatment. Patients on the other treatment options e.g., hydroxyurea and L glutamine may still experience acute clogging of blood vessels called Vaso Occlusive crisis (VOC) There is a need to find more treatment options for sickle cell disease that prevents the VOC. |
| Abstract of Study: | Sickle cell disease (SCD) is a related group of common and rare hemoglobin (Hb) genotypes in which all affected individuals are either homozygotes for the sickle Hb (HbS) mutation of the β-globin subunit or compound heterozygotes for HbS and another globin gene mutation. A hallmark symptom of SCD is a painful Vaso-occlusive crisis (VOC). An interplay of the various pathologic processes in SCD perpetuates and feeds forward an inflammatory priming that, in the presence of a precipitating event, can lead to a VOC. VOCs are responsible for a wide variety of clinical complications of SCD, including pain syndromes, acute chest syndrome (ACS), stroke, priapism, leg ulcers, spontaneous abortion, renal insufficiency, and multiorgan failure leading to death. Frequency of VOCs, along with Absolute Neutrophil count (ACS), is the most common predictor of death in patients with SCD. Thus, early identification, treatment, and prevention of VOCs could significantly impact the natural course of the disease. Interventions for treatment and prevention of longer-term complications are urgently needed.
About 240,000 children in Africa, are born each year with Sickle Cell Disease (SCD), while here in Kenya, it is estimated that 6,000 children are born with the disease. Sadly, between 50 to 80 per cent of these children die before their fifth birthday. The recurrent pain and complications caused by the disease, can interfere with many aspects of the patient’s life, including education, employment and psychosocial development. |
| 4 | The ECCT secretariat Pharmacy and Poisons Board P.O Box 27663-00506 Nairobi Kenya. 19Jul 2023
Dear Sir,
RE: Amendment 3 (Site 501) for protocol Code: ECCT/22/06/03. Protocol Title: A Phase 2a Randomized, Double-Blind, Placebo Controlled Study to Characterize the Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients with Sickle Cell Disease
We wish to submit an amendment for the above referenced protocol. Site 501 (Principal Investigator-Prof Fredrick Asirwa Chite) would like to amend their Site-Specific Addendums and main Informed Consent forms. The changes are as below.
1. Add language regarding participant overnight stay at a place near hospital to enable required fasting time and starting of study procedures during these visits take longer hours and may need to start very early in the morning. 2. Increase in blood collection to enable blood re draws. 3. Reworded language on reimbursement and changed the reimbursement amount by reducing it by Ksh 500 which was costs for meals but since they will be staying overnight and meals provided at a place close to the site.
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