The Use of Eculizumab in HELLP Syndrome

NCT04103489 | Completed Phase 1 DMC FDA Drug

• 2 other identifiers: IRB001935491K12HD085845-01
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

This research study is being performed to see if women diagnosed with early preterm Hemolysis, Elevated Liver Enzymes, Low Platelets (HELLP) syndrome (estimated gestational ages of 23-30 weeks) benefit from a medication called eculizumab (ECU). This drug blocks a part of the immune system called complement. By blocking this part of the immune system, eculizumab may stop or reverse the progression of the HELLP syndrome disease. The investigators will also look to see if this drug is effective and benefits both the mother and fetus.

Conditions

HELLP Syndrome (HELLP), Third Trimester; Complement Abnormality; Morbidity;Newborn; Maternal Injury; Preeclampsia Severe

Outcome Measures

Primary Outcomes (3)

• Change in aspartate aminotransferase (AST) level

  AST measured in units/L.

  Baseline, 72 hours

• Change in alanine aminotransferase (ALT)

  ALT measured in IU/L.

  Baseline, 72 hours

• Change in lactate dehydrogenase levels (LDH)

  LDH measured in units/L.

  Baseline, 72 hours

Secondary Outcomes (3)

• Latency of pregnancy

  Up to 7 days

• Maternal number of units of blood products transfused

  Up to 7 days

• Maternal postpartum length of stay

  Up to 36 days

Study Arms (1)

HELLP Syndrome at less than 30 weeks gestation

EXPERIMENTAL

Women diagnosed with HELLP syndrome at 23-30 weeks gestation will receive eculizumab.

Drug: Eculizumab

Interventions

Eculizumab DRUG

Participants will receive eculizumab at diagnosis of HELLP syndrome. Participants will receive a maximum of 4 doses.

Also known as: Soliris

HELLP Syndrome at less than 30 weeks gestation

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Pregnant women diagnosed with HELLP syndrome less than 30 weeks gestation.

You may not qualify if:

• Women with
• Disseminated intravascular coagulopathy
• Non-reassuring fetal status necessitating delivery
• Non-viable fetuses
• Stroke
• Fetal demise intra-utero
• Eclamptic seizure
• Known atypical hemolytic uremic syndrome
• Familial or acquired thrombocytopenia purpura
• Paroxysmal nocturnal hemoglobinuria
• Allergy to eculizumab will be excluded.

Sponsors & Collaborators

• Johns Hopkins University: lead
• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): collaborator

Study Sites (1)

The Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

MeSH Terms

Conditions

HELLP Syndrome

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Hypertension, Pregnancy-Induced; Pregnancy Complications; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases

Study Officials

• Arthur J Vaught

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This will be an open label, phase 1 clinical trial. The investigators will investigate if eculizumab halts or prevents worsening or HELLP syndrome in women at 23-28 weeks gestation.

Study Record Dates

• First Submitted: September 24, 2019
• First Posted: September 25, 2019
• Study Start: February 23, 2021
• Primary Completion: August 31, 2023
• Study Completion: September 4, 2023
• Last Updated: November 14, 2023

Record last verified: 2023-11

Locations

US (1)