An Open Label Study of the Effects of Eculizumab in CD59 Deficiency

NCT01579838 | Unknown Phase 1

• 1 other identifier: CD59-Mevorach-1
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

The investigators have identified patients with CD59 deficiency that suffers from chronic hemolysis and peripheral demyelinating disease. It was shown that complement terminal pathway can cause inflammation in the peripheral nervous system. Complement can greatly increase the immune attack in the nerves. Eculizumab has already been shown to be effective in a rare blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH). Attacks of PNH are also mediated through complement. Therefore, the investigators of this study are investigating whether by 'turning off' complement in CD59 deficiency, further attacks of hemolysis and nerve injury can be avoided and whether the neurological status will ameliorate.

Conditions

Chronic Hemolysis

Outcome Measures

Primary Outcomes (4)

• Whether Eculizumab reduces chronic hemolysis

  The primary objectives of this study are to determine: Whether Eculizumab reduces chronic hemolysis as judged by LDH levels, and haptoglobin, and level of hemoglobin.

  8 months

• Steroid and iv IgG cumulative dosage

  Cumulative steroid and IgG dosage before and after treatment

  8 months

• Safety.

  Whether the subjects develop known or possible related side effect to the treatment. The number of participants that develop adverse effects will be determined. reports from parents will be taken avery other week and documented clini or hospital visits will be included.

  8 months

• Whether Eculizumab ameliorates the neurological status compared to one month before treatment

  The neurological status in the last month prior to treatment will be determined followed by neurological staus examination every two weeks.

  8 months

Secondary Outcomes (4)

• Whether eculizumab maintains or improves limbs motion

  8 months

• function and quality of life as measured by a variety of established disability scales.

  8 months

• The severity of an individual attack and the degree of recovery.

  8 months

• Levels of membrane attack complex

  8 months

Study Arms (1)

Eculizumab

EXPERIMENTAL

Eculizumab will be administrated according to known protocols.

Drug: Eculizumab

Interventions

Eculizumab DRUG

PNH and or atypical TTP classical protocols

Also known as: Soliris

Eculizumab

Eligibility Criteria

• Age: 2 Months - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• CD59 deficiency

You may not qualify if:

• recent exposure to meningococcal infections

Sponsors & Collaborators

• Hadassah Medical Organization: lead

Study Sites (1)

Hadassah Medical Center

Jerusalem, 91120, Israel

RECRUITING

Related Publications (1)

• Mevorach D, Reiner I, Grau A, Ilan U, Berkun Y, Ta-Shma A, Elpeleg O, Shorer Z, Edvardson S, Tabib A. Therapy with eculizumab for patients with CD59 p.Cys89Tyr mutation. Ann Neurol. 2016 Nov;80(5):708-717. doi: 10.1002/ana.24770. Epub 2016 Sep 19.

  PMID: 27568864 DERIVED

MeSH Terms

Interventions

eculizumab

Study Officials

• Dror Mevorach, MD

  hmo

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Dror Mevorach, MD

CONTACT

mevorachd@hadassah.org.il

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of Medicine and head of Rheumatology Research Center

Study Record Dates

• First Submitted: February 23, 2012
• First Posted: April 18, 2012
• Study Start: February 1, 2012
• Primary Completion: March 1, 2013
• Study Completion: March 1, 2013
• Last Updated: April 18, 2012

Record last verified: 2012-04

Locations

IL (1)