I just inquired about this trial and apparently we might be eligible even though we're in Australia and non-US citizens. We still need to have a phone call and see if we tick all the boxes plus we need to decide if we can commit to going when they need us to!
Just in case if anyone is interested, this is what the consent form says about the trial. Sorry about the formating, i could only copy and paste from a PDF.
Why is this research study being done?
We are doing this research study to find out if BIIB067 is safe and tolerable in adult subjects with Amyotrophic Lateral Sclerosis. We also want to learn about the pharmacokinetics (PK) of BIIB067. PK looks at the level of the study drug in your body and what happens to this level over time from the moment that it is given to you up to the point at which it is completely eliminated from your body. It is studied by measuring the amount of study drug that gets into your blood, urine and cerebrospinal fluid after you take the study drug. Cerebrospinal fluid (CSF) is the fluid around the spinal cord.
We are asking you to participate in Part B of this research study where you would receive repeated doses of the study drug.
BIIB067 is not approved by the U.S. Food and Drug Administration (FDA). This means that BIIB067 can only be used in research studies.
BIIB067 has not yet been tested in humans. However, BIIB067 has previously been given toanimals. In those studies, BIIB067 did not appear to cause any serious clinical problems in the animals at doses estimated to be greater than those to be tested in this study. Doctors now want to learn about BIIB067 in humans.
This research study will compare BIIB067 to placebo. The placebo looks exactly like BIIB067,
but contains no BIIB067. During this study you may get a placebo instead of BIIB067.
Placebos are used in research studies to see if the results are due to the study drug or due to other
reasons.
We are asking you to take part in this research study because you have ALS. ALS is also known
as Lou Gehrig‟s disease. It is a progressive disease of motor nerve cells (cells that control
movement of your muscles).
You have been invited to take part in Part B of this research study because you have been
diagnosed with ALS, have the SOD1 gene mutation and are currently showing signs of weakness
due to ALS. While sporadic ALS patients have two normal copies of the SOD1 gene, most
SOD1 ALS patients have one mutated copy of the SOD1 gene and one normal copy of the SOD1
gene. The study drug BIIB067 is thought to bind to both the normal and mutated copies of the
SOD1 gene. This binding may reduce the amount of both normal and toxic SOD1 protein.
About 36 subjects will take part in this research study. About 8 subjects will take part at
Massachusetts General Hospital (MGH).
Biogen MA
How long will I take part in this research study?
It will take you about 31 weeks to complete Part B of this research study. During this time, we
will ask you to make approximately 14 study visits to MGH. One of these visits will include an
overnight stay at MGH.
If you have participated in Part A of the study, you may also take part in Part B after a „washout‟
period of approximately 20 weeks since your last dose of study drug. The wash-out period is to
allow time for all of the study drug from Part A to be completely out of your body before you
start Part B.
If you take part in both Parts A and B of the study then the total duration will be approximately
48 weeks.
If you participated in 2 different cohorts in Part A, and then take part in Part B, the total duration
will be approximately 68 weeks.
We're staying positive and hopeful! If we do the trial, we would be based in Boston.
