Status:
COMPLETED
Plasma Exchange With Albumin in AMN Patients
Lead Sponsor:
Pujol, Aurora, M.D.
Conditions:
Adrenomyeloneuropathy
Adrenoleukodystrophy
Eligibility:
MALE
18-65 years
Phase:
PHASE2
PHASE3
Brief Summary
Adrenoleukodystrophy (X-ALD) is the most common genetic disorder of the brain white matter with an incidence of 1:14,700 births. It is caused by mutations in the ABCD1 gene, which encodes a transporte...
Eligibility Criteria
Inclusion
- Men of 18 to 65 years old, inclusive
- Elevated plasma VLCFA and gene mutation identified
- Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to run
- Presence of motor deficit according to the EDSS scale
- Ability to perform the 2MWT
- Normal brain MRI or brain MRI showing the following abnormalities that can be observed in AMN patients without the cerebral form of X-ALD, obtained in the 6 months prior to screening:
- abnormal hyperintensity of pyramidal tract fibers in the brain stem on FLAIR or T2 sequence
- abnormal hyperintensity of pyramidal tract fibers in the internal capsules on FLAIR or T2 sequence
- cerebellar atrophy
- moderate cortical atrophy
Exclusion
- Any contraindication for plasma exchange due to behavioral disorders or abnormal coagulation parameters, such for example:
- Hypocalcemia (Ca++ \< 8.7 mg/dl)
- Thrombocytopenia (\< 100.000/µl)
- Fibrinogen \< 1.5 g/l
- Prothrombin time (Quick) p\< 60% versus control (INR \> 1.5)
- Beta-blocker treatment and bradycardia \< 55/min
- Treatment with ACIs (increased risk of allergic reactions)
- Hemoglobin \< 10 g/dl
- Difficult venous access precluding plasma exchange
- A history of frequent adverse reactions (serious or otherwise) to blood products
- Hipersensibility to albumin o allergies to any of the components of Albunorm® 5%
- Plasma creatine \> 2 mg/dl
- Uncontrolled high blood pressure (systolic blood pressure of 160 mmHg or higher and/or diastolic blood pressure of 100 mmHg or higher despite regular treatment during the last 3 months)
- Liver cirrhosis or any liver problem with GPT \> 2.5 x ULN, or bilirubin \> 2 mg/dl
- Heart diseases as evidenced by myocardial infarction, severe or unstable angina, or heart failure in the past 12 months
- Gadolinium enhancement on T1 sequence of any abnormal hypersignal of white matter, including myelinated pyramidal tracts, visible at brain MRI on FLAIR sequences
- Significant peripheral edema (2+ or more on the Assessment Chart for Pitting Edema) of the extremities of any etiology
- Any evolutive malignancy during the last five years or any condition complicating adherence to the study protocol
- Smokers (one pack/ day or more for at least 20 years), current or former
- Any psychiatric disease
- Present participation to another therapeutic clinical trial for X-ALD, or the receipt of any other investigational drug in the three months prior to the start of the study
- Patients being treated with anticoagulants or antiplatelet therapy
- Not easily contactable by the investigator in case of emergency or not capable to call the investigator
Key Trial Info
Start Date :
March 9 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 13 2021
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT04303416
Start Date
March 9 2020
End Date
September 13 2021
Last Update
September 22 2022
Active Locations (1)
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1
Bellvitge University Hospital
L'Hospitalet de Llobregat, Barcelona, Spain, 08908