C1-inhibitor improves efficacy of red blood cell transfusion in patients suffering from autoimmune hemolytic anemia – an open-labeled pilot trial

• Register: EUCTR
• Last refreshed on: 18 October 2021
• Main ID: EUCTR2012-003710-13-NL
• Date of registration: 21/03/2013
• Prospective Registration: Yes
• Primary sponsor: Academic Medical Center
• Public title: C1-inhibitor improves efficacy of red blood cell transfusion in patients suffering from autoimmune hemolytic anemia – an open-labeled pilot trial
• Scientific title: C1-inhibitor improves efficacy of red blood cell transfusion in patients suffering from autoimmune hemolytic anemia – an open-labeled pilot trial - C1-inhibitor improves efficacy of red blood cell transfusion in patients suffering from autoimmune h
• Date of first enrolment: 21/03/2013
• Target sample size: 10
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-003710-13
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: yes; Placebo: no; Other: no; Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Netherlands

Contacts

• Name: M. Spiering
• Affiliation: Academic Medical Center

• Name: M. Spiering
• Affiliation: Academic Medical Center

Key inclusion & exclusion criteria

Inclusion criteria:
• Positive (=1+) monospecific antiglobulin test for C3b and/or C3d with/without positivity for IgM or IgG
• Indication for a transfusion with at least 2 red packed cell concentrates based on the clinical assessment by the hematologist in charge
• Hemoglobin value at least < 5 mmol/L (8 g/dl) with/without clinical symptoms
• Clinical signs of hemolysis: not-detectable haptoglobin (mandatory) and increased lactate dehydrogenase (LDH) eventually combined with hyperbilirubinemia (increased direct and/or indirect bilirubin), lactate.
• Age = 18 years
• Written informed consent
• Women of child bearing potential must have had a negative serum pregnancy test 7 days prior to the start of study drug

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 9
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion criteria:
• History of arterial and/or venous thromboembolic events in the absence of an actual treatment with Vitamin K-antagonists
• Concomitant use of therapeutic doses of heparin
• Female patients who are pregnant or breast feeding or adults of reproductive potential who are not using effective birth control methods. If barrier contraceptives are being used, these must be continued throughout the trial by both sexes. Oral contraceptives only are not acceptable.
• Patients with known HIV seropositivity or chronic active hepatitis
• Patients who have any severe and/or uncontrolled medical condition or other conditions that could affect their participation in the study such as:
– cerebrovascular accidents = 6 months before study drug start
– uncontrolled hypertension



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

AutoImmune Hemolytic Anemia
MedDRA version: 20.0 Level: LLT Classification code 10003825 Term: Autoimmune hemolytic anemia System Organ Class: 100000004851

Intervention(s)

Trade Name: CINRYZE® (C1 esterase inhibitor [human])
Pharmaceutical Form: Concentrate and solvent for solution for infusion

Primary Outcome(s)

Main Objective: to test whether co-administration of C1-inh concentrate in patients suffering from AIHA needing RBC transfusion
• improves recovery of RBC transfusion
• inhibits complement activation and deposition on RBC via the classical pathway of complement
• is safe?

Secondary Objective: to investigate whether co-administration of C1-inhibitor concentrate in patients suffering from AIHA needing RBC transfusion
• attenuates the pro-inflammatory response in AIHA
• affects the response to the basic treatment targeting autoantibody production

Primary end point(s): • improves recovery of RBC transfusion
• inhibits complement activation and deposition on RBC via the classical pathway of complement

Timepoint(s) of evaluation of this end point: • At entry: laboratory tests within 4 hours before start of treatment, before and after every dose C1-inh as well as 48 hours, 60 hours and 72 hours after the first dose
NB: in case that the time between study entry and start treatment exceeds 4 hours, laboratory tests performed at study entry must be repeated

Secondary Outcome(s)

Secondary end point(s): not applicable

Timepoint(s) of evaluation of this end point: not applicable

Secondary ID(s)

NL41820.018.12

Source(s) of Monetary Support

Academic Medical Center

Ethics review

Status: Approved
Approval date: 27/11/2012
Contact: