First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
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| ClinicalTrials.gov Identifier: NCT02675465 |
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Recruitment Status :
Active, not recruiting
First Posted : February 5, 2016
Last Update Posted : June 13, 2022
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Pompe Disease | Drug: ATB200 Drug: AT2221 | Phase 1 Phase 2 |
This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the effect of a highly targeted rhGAA (ATB200) co-administered with a chaperone (AT2221).
The study aims to evaluate safety, tolerability, pharmacodynamics (PD), and immunogenicity of ATB200 co-administered with AT2221. The study will be conducted in 3 stages.
In Stage 1, safety, tolerability, and PK will be evaluated following sequential single ascending doses of intravenously infused ATB200.
In Stage 2, safety, tolerability, and PK will be evaluated following single- and multiple-ascending dose combinations of ATB200 and AT2221.
In Stage 3, long term safety and efficacy will be assessed following 24 month treatment of ATB200 co-administered with AT2221 (Miglustat)
In Stage 4, treatment period will begin at the end of Stage 3 and will continue as open label extension until commercialization, study discontinuation or subject withdrawal, with functional assessments every 6 months
No Muscle biopsies will be performed in this study.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 32 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease |
| Study Start Date : | January 2016 |
| Estimated Primary Completion Date : | December 2023 |
| Estimated Study Completion Date : | December 2023 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: ATB200
In Stage 1, safety, tolerability, and PK will be evaluated following sequential single ascending doses of intravenously infused ATB200 for 3 dosing periods.
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Drug: ATB200 |
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Experimental: ATB200 + AT2221
In Stage 2, safety, tolerability, and PK will be evaluated following single- and multiple-ascending dose combinations of ATB200 co-administered with AT2221 (Miglustat) In Stage 3, long term safety and efficacy will be assessed following 24 month treatment of ATB200 co-administered with AT2221 (Miglustat)
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Drug: ATB200 Drug: AT2221 Other Name: Miglustat |
- Plasma GAA activity levels as measured by maximum observed plasma concentration (Cmax). [ Time Frame: 18 Weeks ]
- Plasma GAA activity levels as measured by time to reach the maximum observed plasma concentration (tmax). [ Time Frame: 18 Weeks ]
- Plasma GAA activity levels as measured by area under the plasma-drug concentration time curve. [ Time Frame: 18 Weeks ]
- Safety and tolerability as measured by counts of Treatment Emergent Adverse Events (TEAEs), including Infusion Associated Reactions (IARs). [ Time Frame: 18 weeks ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Key Inclusion Criteria:
- Male and female subjects between 18 and 75years of age, inclusive
- Diagnosis of Pompe disease
Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):
- Has received ERT with alglucosidase alfa for the previous 2-6 years, inclusive
- Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency of once every other week
- Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )
- Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value
ERT-experienced subjects (non-ambulatory):
- Has received ERT with alglucosidase alfa (Myozyme/Lumizyme) for ≥2 years
- Is wheelchair-bound
ERT-naïve subjects (ambulatory):
- Must be able to walk 200-500 meters on the 6MWT
- Has upright FVC must be 30% to 80% of predicted normal value
- Subject has never received alglucosidase alfa
Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):
- Has received ERT with alglucosidase alfa for >7years, inclusive
- Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency of once every other week
- Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )
- Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value
Exclusion Criteria:
- Subject has received treatment with prohibited medications within 30 days of Baseline Visit
- Subject, if female, is pregnant or breastfeeding at screening
- Subject, whether male or female, planning to conceive a child during the study
- Subject has a medical or any other extenuating condition or circumstance that may, in opinion of investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements
- Subject has a history of allergy or sensitivity to miglustat or other iminosugars
- Subjects with active systemic autoimmune disease such as lupus, scleroderma, or rheumatoid arthritis. All subjects with autoimmune disease must be discussed with the Amicus Medical Monitor
- Subjects with active bronchial asthma. All subjects with bronchial asthma must be discussed with the Amicus Medical Monitor
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02675465
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| Responsible Party: | Amicus Therapeutics |
| ClinicalTrials.gov Identifier: | NCT02675465 |
| Other Study ID Numbers: |
ATB200-02 |
| First Posted: | February 5, 2016 Key Record Dates |
| Last Update Posted: | June 13, 2022 |
| Last Verified: | June 2022 |
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Pompe, rhGAA |
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Glycogen Storage Disease Type II Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Glycogen Storage Disease Carbohydrate Metabolism, Inborn Errors Lysosomal Storage Diseases |
Metabolic Diseases Miglustat Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Anti-HIV Agents Anti-Retroviral Agents Antiviral Agents Anti-Infective Agents Glycoside Hydrolase Inhibitors Hypoglycemic Agents Physiological Effects of Drugs |