ALS (Amyotrophic Lateral Sclerosis) Clinical Trial
Official title:
Expansion and Infusion of T-Regulatory Cells in Amyotrophic Lateral Sclerosis
This is an open-label pilot study to determine the safety and tolerability of infusions of autologous CD4+ CD25+ regulatory T cells with concomitant subcutaneous IL-2 injections in 4 subjects with ALS.
This Pilot Study will consist of 4 ALS subjects who will undergo 4 infusions of autologous
expanded Tregs with concomitant subcutaneous injections of IL-2 (2 x 105 IU/m2) 3 times
weekly for 52 weeks or at least until such time that interim analyses can confirm or negate
its benefit.
During the enrollment period up to four subjects will be recruited from patients known to our
clinic for screening, baseline measures and leukapheresis. The Treg cell manufacturing will
be performed in a GMP laboratory. The first subject will receive infusions of their expanded
Tregs (1x106 /kg) with concomitant subcutaneous IL-2 injections (2 x 105 IU/m2) 25 days (+/-
2 days) post leukapheresis. The 2nd subject will begin after the first subject has completed
the first 4 weeks and has experienced no untoward effects during this period. Once subjects
#1 and #2 have completed the first 4 weeks and no toxic events have occurred they will
therefore be considered safely past the first milestone and subject #3 will begin infusions.
After subject #3 has completed the first 4 weeks with no untoward effects, subject #4 may
begin infusions with the below modified schedule:
The 4th subject will undergo infusion of autologous expanded Tregs once every four weeks (+/-
2 days) for a total of four infusions, with concomitant subcutaneous injections of IL-2 (2 x
105 IU/m2) 3 times weekly. In addition, subject #4 will begin subcutaneous IL-2 injections 4
weeks before his first autologous Treg infusion. An additional office visit will take place 2
weeks after initiating IL-2 for clinical evaluation, scoring and blood draw. Office visits
will then be completed every two weeks while the subject is receiving Treg infusions for
clinical evaluation, scoring, and blood draws. Then, the subject will be seen in office
visits once per month for one year total for clinical evaluation, scoring, and blood draws.
In addition, subjects #1, 2 and 3 will repeat the leukapheresis (under a separate protocol)
and undergo Treg infusions at the modified schedule of every 4 weeks, with concomitant
subcutaneous injections of IL-2 (2 x 105 IU/m2) 3 times weekly. The subjects will be called
on Day 7, and 21. Office visits will be completed on the day after infusions and every two
weeks while the subjects are undergoing Treg infusions for clinical evaluation, scoring, and
blood draws. The subjects will then be seen during office visits once per month for one year
total from their initial baseline visit for clinical evaluation, scoring, and blood draws
Monthly interim analyses will monitor the subjects using validated ALS scales such as the
ALSFRS-R and Appel Scale, which incorporates muscle strength and dysfunction, activities of
daily living and pulmonary function. The analyses will also include interim medical history
and physical exam, EKG when indicated, safety labs (such as CBC, chemistry, liver function,
T4 and TSH) as well as more technical research labs such as T Regulatory Cell, Th1 and Th17
counts, FoxP3 RNA expression, and Treg Suppression Assays. A PT/PTT will be performed only if
the subject has an abnormal coagulation result at baseline or if the subject is on
anti-coagulation therapy.
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