DOSING REGIMENS FOR THE TREATMENT OF POMPE DISEASE
The presently disclosed subject matter provides a dosing regimen and administration schedule for the use of 1-deoxynojirimycin and enzyme replacement therapy for the treatment of Pompe disease. The presently disclosed subject matter further provides a dosing regimen and administration schedule for the use of 1-deoxynojirimycin hydrochloride and alglucosidase alfa for the treatment of Pompe disease.
1 . A method of treating Pompe disease in a subject, the method comprising administering from about 25 mg to about 1000 mg of 1-deoxynojirimycin and an effective amount of acid α-glucosidase enzyme replacement therapy to a patient in need thereof.
2 . The method of claim 1 , wherein the amount of 1-deoxynojirimycin administered is about 50 mg to about 600 mg.
3 . The method of claim 1 , wherein the amount of 1-deoxynojirimycin administered is selected from the group consisting of about 50 mg, about 100 mg, about 250 mg and about 600 mg.
4 . The method of claim 1 , wherein the patient fasts for a period of time beginning about 0.5 to about 4 hours prior to and ending about 0.5 to about 4 hours following administration of 1-deoxynojirimycin.
5 . The method of claim 4 , wherein the patient fasts for at least about 2 hours prior to and at least about 2 hours following administration of 1-deoxynojirimycin.
6 . The method of claim 1 , wherein the 1-deoxynojirimycin is administered simultaneously with to about 4 hours prior to the administration of the acid α-glucosidase enzyme replacement therapy.
7 . The method of claim 6 , wherein the 1-deoxynojirimycin is administered about 2 hours prior to the administration of the acid α-glucosidase enzyme replacement therapy.
8 . The method of claim 6 , wherein the 1-deoxynojirimycin is administered about 1 hour prior to the administration of the acid α-glucosidase enzyme replacement therapy.
9 . The method of claim 1 , wherein the 1-deoxynojirimycin is 1-deoxynojirimycin hydrochloride.
10 . The method of claim 1 , wherein the acid α-glucosidase enzyme replacement therapy is rhGAA.
11 . The method of claim 1 , wherein the acid α-glucosidase enzyme replacement therapy is alglucosidase alfa.
12 . The method of claim 1 , wherein the 1-deoxynojirimycin is administered as an adjuvant to the acid α-glucosidase enzyme replacement therapy.
13 . The method of claim 1 , wherein the 1-deoxynojirimycin and acid α-glucosidase enzyme replacement therapy are administered as a combination therapy.
14 . The method of claim 6 , wherein the patient is administered a second dose of 1-deoxynojirimycin between the administration of the acid α-glucosidase enzyme replacement therapy and about 4 hours thereafter.
15 . The method of claim 7 or 8 , wherein the 1-deoxynojirimycin and acid α-glucosidase enzyme replacement therapy are administered every 1 to 4 weeks.
16 . The method of claim 15 , wherein the 1-deoxynojirimycin and acid α-glucosidase enzyme replacement therapy are administered every 2 weeks.
17 . A kit for treating Pompe disease in a subject, the kit comprising from about 25 mg to about 1000 mg of 1-deoxynojirimycin and an effective amount of acid α-glucosidase enzyme replacement therapy.
18 . The kit of claim 17 , wherein the amount of 1-deoxynojirimycin is selected from the group consisting of about 50 mg, about 100 mg, about 250 mg and about 600 mg.
19 . A method of treating Pompe disease in a subject, the method comprising administering from about 25 mg to about 1000 mg of 1-deoxynojirimycin derivative and an effective amount of acid α-glucosidase enzyme replacement therapy to a patient in need thereof.
20 . The method of claim 19 , wherein the amount of 1-deoxynojirimycin derivative administered is about 50 mg to about 600 mg.
21 . The method of claim 19 , wherein the amount of 1-deoxynojirimycin derivative administered is selected from the group consisting of about 50 mg, about 100 mg, about 250 mg and about 600 mg.
22 . The method of claim 19 , wherein the patient fasts for a period of time beginning about 0.5 to about 4 hours prior to and ending about 0.5 to about 4 hours following administration of 1-deoxynojirimycin derivative.
23 . The method of claim 22 , wherein the patient fasts for at least about 2 hours prior to and at least about 2 hours following administration of 1-deoxynojirimycin derivative.
24 . The method of claim 19 , wherein the 1-deoxynojirimycin derivative is administered simultaneously with to about 4 hours prior to the administration of the acid α-glucosidase enzyme replacement therapy.
25 . The method of claim 24 , wherein the 1-deoxynojirimycin derivative is administered about 2 hours prior to the administration of the acid α-glucosidase enzyme replacement therapy.
26 . The method of claim 24 , wherein the 1-deoxynojirimycin derivative is administered about 1 hour prior to the administration of the acid α-glucosidase enzyme replacement therapy.
27 . The method of claim 19 , wherein the 1-deoxynojirimycin derivative is 1-N-Butyl-DNJ.
28 . The method of claim 19 , wherein the acid α-glucosidase enzyme replacement therapy is rhGAA.
29 . The method of claim 19 , wherein the acid α-glucosidase enzyme replacement therapy is algiucosidase alfa.
30 . The method of claim 19 , wherein the 1-deoxynojirimycin derivative is administered as an adjuvant to the acid α-glucosidase enzyme replacement therapy.
31 . The method of claim 19 , wherein the 1-deoxynojirimycin derivative and acid α-glucosidase enzyme replacement therapy are administered as a combination therapy.
32 . The method of claim 24 , wherein the patient is administered a second dose of 1-deoxynojirimycin derivative between the administration of the acid α-glucosidase enzyme replacement therapy and about 4 hours thereafter.
33 . The method of claim 25 or 26 , wherein the 1-deoxynojirimycin derivative and acid α-glucosidase enzyme replacement therapy are administered every 1 to 4 weeks.
34 . The method of claim 33 , wherein the 1-deoxynojirimycin derivative and acid α-glucosidase enzyme replacement therapy are administered every 2 weeks.
35 . A kit for treating Pompe disease in a subject, the kit comprising from about 25 mg to about 1000 mg of 1-deoxynojirimycin derivative and an effective amount of acid α-glucosidase enzyme replacement therapy.
36 . The kit of claim 35 , wherein the amount of 1-deoxynojirimycin derivative is selected from the group consisting of about 50 mg, about 100 mg, about 250 mg and about 600 mg.
37 . A method of treating Pompe disease in a subject, the method comprising administering from about 25 mg to about 1000 mg of 1-deoxynojirimycin or 1-deoxynojirimycin derivative and an effective amount of acid α-glucosidase enzyme replacement therapy to a patient in need thereof.