An Open-Label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in Naive and Alglucosidase Alfa Treated Late-Onset Pompe Disease Patients

Study ID
STU 052013-004

Cancer Related

Healthy Volunteers

Study Sites

Penny Currykosky

Principal Investigator
Jaya Trivedi


TDR12857 is an open label, multicenter, multinational, ascending dose study with repeated intravenous infusions of neoGaa every 2 weeks for a total of 13 infusions. The population to be included is late-onset Pompe disease patients greater than or equal to 18 years of age naive to treatment (Group 1) or previously treated for a minimum of 9 months with alglucosidase alfa (Group 2). Group 1 and Group 2 will be initiated simultaneously.

Patients will receive an iV infusion of neoGaa qow. Prior to each infusion, the patient should be assessed by the investigator or appropriate designee to determine if the patient is free of acute illness and is clinically stable to receive the infusion. infusions will be administered in a step-wise manner. it is recommended that the infusion be administered at an initial rate of approximately 1 mg/kg/hr and may be augmented incrementally (by 2 mg/kg/hr) every 30 minutes [if vital signs are stable and there are no signs of infusion associated reactions (iaRs)] until a maximum rate of approximately 7 mg/kg/hr is reached.

The follow-up observation period for the end-of-study visit and for treatment-emergent adverse events (Teae) is anticipated to be approximately 4 weeks after the last administration of neoGaa considering the long tissue half-life of eRTs used to treat lysosomal storage diseases.

For both parts, safety data collected through 48 hours following the first patient's neoGaa infusion will be internally reviewed by the Sponsor prior to initiating the first neoGaa infusion in the second patient. The same interval will be used between subsequent patients. Safety data may include adverse events (aes) including iaRs, biochemistry results, eCGs, blood pressure, and heart rate and any available immunology results as applicable.

Participant Eligibility

Group 2 only
1. The patient has been previously treated with alglucosidase alfa for at least 9 months.

For both Group 1 and Group 2
1. The patient is >= 18 years of age with confirmed GAA enzyme deficiency from any tissue source and/or confirmed GAA gene mutation and without known cardiac hypertrophy.
2. The patient is willing and able to provide signed informed consent.
3. The patient is able to ambulate 50 meters (approximately 160 feet) without stopping and without an assistive device. Use of assistive device for community ambulation is appropriate.
4. The patient has a forced vital capacity (FVC) in upright position of >=50% predicted.
5. The patient, if female and of childbearing potential, must have a negative pregnancy test [urine beta-human chorionic gonadotropin ([BETA]-hCG)] at baseline. Note: Sexually active female patients of childbearing potential and male patients are required to practice true abstinence in line with their preferred and usual lifestyle or use two acceptable effective methods of contraception, a barrier method such as a condom or occlusive cap (diaphragm or cervical/vault cap) with spermicidal foam/gel/film/cream/suppository and an established non-barrier method such as oral, injected, or implanted hormonal methods, an intrauterine device or intrauterine system for the entire duration of the treatment period.