Clinical Trial Search

Primary: Insulin iAUC Obtained During the Oral Glucose Tolerance Test at the Final Experimental Visit. — 13,698.60; 10,619.70 mIU/dL∙min — p=0.005

Primary: Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa — 204000; 307000 nanograms per milliliter (ng/mL)

Primary: Phase I - Maximum Tolerated Dose (MTD) — NA mg

Primary: Cumulative Number of New T1 Gadolinium-Enhancing (Gd+) Lesions on Magnetic Resonance Imaging (MRI) Scan From Week 12 to Week 24 — 1.4; 1.1; 3.5; 6.2 Lesions — p=<0.0001

Primary: Proportion of Patients With Clinical Remission at Week 12 — 0.00; 0.042; 0.087; 0.071 Proportion of Participants

Primary: Annualized Confirmed Relapse Rate — 0.202; 0.290 relapses per year — p=0.0003

Primary: Full-dose Donor Chimerism (FDC) at Day 28 Following TLI/ATG/TBI Conditioning. — 13 Participants

Primary: Complement C3 Genotype — 22; 45; 2; 6 Participants

Primary: Number of Participants to Complete Model Adapted Radiations Fractionation Scheduled — 14 Participants

Primary: Overall Comfort — 86.0; 81.6; 81.0; 87.7 units on a scale

Primary: Mean Change in Amblyopic-eye Visual Acuity (VA) Older Cohort — 1.3; 1.7; 1.3; 1.7 Letter score — p=0.71

Primary: Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 6; 9; 1; 4 Participants

Primary: Procedural Pain — 6.50; 5.00 score on a scale

Primary: Objective Response Rate (ORR) — 0.522 proportion of participants

Primary: Modified City of Hope Quality of Life Questionnaire for Patients With Thyroid Disease — -36.33 score on a scale