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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01579838
Recruitment Status : Unknown
Verified April 2012 by Mevorach Dror, Hadassah Medical Organization.
Recruitment status was:  Recruiting
First Posted : April 18, 2012
Last Update Posted : April 18, 2012
Sponsor:
Information provided by (Responsible Party):
Mevorach Dror, Hadassah Medical Organization

Tracking Information
First Submitted Date  ICMJE February 23, 2012
First Posted Date  ICMJE April 18, 2012
Last Update Posted Date April 18, 2012
Study Start Date  ICMJE February 2012
Estimated Primary Completion Date March 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 17, 2012)
  • Whether Eculizumab reduces chronic hemolysis [ Time Frame: 8 months ]
    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.
  • Steroid and iv IgG cumulative dosage [ Time Frame: 8 months ]
    Cumulative steroid and IgG dosage before and after treatment
  • Safety. [ Time Frame: 8 months ]
    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.
  • Whether Eculizumab ameliorates the neurological status compared to one month before treatment [ Time Frame: 8 months ]
    The neurological status in the last month prior to treatment will be determined followed by neurological staus examination every two weeks.
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: April 17, 2012)
  • Whether eculizumab maintains or improves limbs motion [ Time Frame: 8 months ]
    Whether there is a neurological amelioration
  • function and quality of life as measured by a variety of established disability scales. [ Time Frame: 8 months ]
    we will be using evry other week parents report of his clinical condition. We will use modified SF36 and local questionairre.
  • The severity of an individual attack and the degree of recovery. [ Time Frame: 8 months ]
  • Levels of membrane attack complex [ Time Frame: 8 months ]
    This will be measured using flow cytometry for the presence of membrane attack complex on neutrophild and res blood cells.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Open Label Study of the Effects of Eculizumab in CD59 Deficiency
Official Title  ICMJE An Open Label Study of the Effects of Eculizumab in CD59 Deficiency
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.
Detailed Description

It has been shown in some scientific studies that lack of CD59 in the context of the disease paroxysmal nocturnal hemoglobinuria (PNH)leads to chronic hemolysis. The investigators have identified patients wirh CD59 deficiency that suffers from chronic hemolysis and demyelinating disease. It was shown that complement terminal pathway can cause cause inflammation in 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 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.

The primary (most important) objectives of this study are to determine:

Whether Eculizumab ameliorate the neurological condition documented in the last month before treatment and whether it reduces the relapse frequency in patients with relapsing chronic inflammatory demyilinating polyneuropathy. The number of attacks during the one year treatment period will be compared to the number of attacks that occurred prior to initiation of eculizumab treatment. For patients with more than 2 year disease duration, the average number of attacks in the preceding 2 years will be calculated. For patients with less than 2 years disease duration the number of attacks in the preceding year will be used.

Whether Eculizumab reduces chronic hemolysis as judged by LDH levels, and haptoglobin, and level of hemoglobin. the same for corticosteroids and or I.V. IgG consumption before and after treatment with eculizumab.

The safety profile of eculizumab in patients with CD59 deficiency will be determined by parents report evry other week, documentation of clinic referral and hospitalizations. The number of participants with advers events will be determined.

The secondary objectives are to determine:

Whether eculizumab maintains or improves limbs motion, function and quality of life as measured by a variety of established disability scales like the modified SF36 and like a questionaire developed for this age group. The investigators will also assess the severity of an individual attack and the degree of recovery.

How the drug behaves in the patient's blood by measuring the presence of membrane attack complex on neutrophils and red blood cells.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Chronic Hemolysis
Intervention  ICMJE Drug: Eculizumab
PNH and or atypical TTP classical protocols
Other Name: Soliris
Study Arms  ICMJE Experimental: Eculizumab
Eculizumab will be administrated according to known protocols.
Intervention: Drug: Eculizumab
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: April 17, 2012)
5
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2013
Estimated Primary Completion Date March 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • CD59 deficiency

Exclusion Criteria:

  • recent exposure to meningococcal infections
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Months to 70 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Israel
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01579838
Other Study ID Numbers  ICMJE CD59-Mevorach-1
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Mevorach Dror, Hadassah Medical Organization
Study Sponsor  ICMJE Hadassah Medical Organization
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Dror Mevorach, MD hmo
PRS Account Hadassah Medical Organization
Verification Date April 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP