Hepatocyte Microbeads for Acute Liver Failure

Launched by KING'S COLLEGE HOSPITAL NHS TRUST · Aug 5, 2022

Keywords

ClinConnect Summary

The HELP trial is a research study aimed at exploring a new treatment for children with acute liver failure, a serious condition where the liver stops working properly. This trial is testing a therapy that uses liver cells (called hepatocytes) wrapped in tiny beads made from a natural substance. These beads help protect the liver cells and allow them to work in the body without the need for major surgery or strong medications to prevent the body from rejecting them. The goal is to see if this treatment can support the liver while it heals itself or help buy time for a liver transplant if needed.

To be eligible for the trial, children under 16 years old who have acute liver failure can participate, given that their parents or guardians provide written consent. However, there are some criteria that could exclude them, such as having a serious infection or being too unstable to undergo the procedure. Participants in this study can expect to receive the treatment and be monitored closely by doctors to ensure their safety and well-being throughout the process. The trial is currently not recruiting participants, but it holds promise for helping children with this critical condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Infant or child (male or female) under the age of 16 years at recruitment.
• • Written informed consent obtained from a parent / legal guardian
• • Presence of ALF defined as a multisystemic disorder in which severe impairment of liver function with or without encephalopathy occurs in association with hepatocellular necrosis reflected as synthetic liver failure in a child with no recognised underlying chronic liver disease. Children must fit one of the ALF categories as described in Appendix 1b(study protocol);
• • Willing and able to comply with the study visit schedule.
• Exclusion Criteria:
• • Severe ascites causing high intra-abdominal pressure and / or respiratory compromise;
• • Intra-abdominal sepsis suspected or proven;
• • Clinical condition too unstable to tolerate procedure without compromise;
• • Proven pre-existing allergy or intolerance to alginate on medical history;
• • Proven pre-existing allergy to gentamicin on medical history;
• • Intraperitoneal or intra-abdominal malignancy;
• • Adhesions or fistulae to anterior abdominal wall;
• • Children who weigh in excess of 33kg
• • Pregnant or lactating patients (positive pregnancy test for females of child bearing potential at screening).
• • Female patients of childbearing potential who are not willing to use highly effective methods of contraception to prevent pregnancy or abstain from heterosexual activity for 52 weeks post treatment.
• • \*Females of child bearing potential are females who have experienced menarche and are not surgically sterilised (e.g. by tubal occlusion, hysterectomy, bilateral salpingectomy) or post-menopausal (defined as at least 1 year since last regular menstrual period).
• • \*\* Highly effective methods of birth control are those with a failure rate of \< 1% per year when employed consistently and correctly. Highly effective methods of contraception as per HMA / CTFG working group are combined (Estrogen and Progestogen containing) hormonal contraception associated with inhibition of ovulation, the preparation may be oral, intravaginal or transdermal; progesterone-only hormonal contraception associated with inhibition of ovulation which may be oral, injectable or implantable; intrauterine device (IUD); intrauterine hormone-releasing system (IUS); bilateral tubal occlusion; vasectomised partner; sexual abstinence for 52 weeks post study treatment;
• • \*\*\* Sexual abstinence is considered to be highly effective method only if defined as refraining from heterosexual activity from the date of consent until the week 52 visit post study treatment. The reliability of this method should be evaluated in relation to the duration of the study and the preferred and usual lifestyle of the participant.
• • Male patients who are not willing to use an effective method of contraception (condom, vasectomy, sexual abstinence) for 52 weeks post study treatment, when engaging in sexual activity with a female of childbearing potential;
• • Participation in concurrent therapeutic trial for ALF;
• • Imminent liver transplantation expected within 12 hours of infusion;
• • Total hepatectomy;
• • Dependent on Extracorporeal Membrane Oxygenation (ECMO);
• • Previous liver transplant