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A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa (Mini-COMET)

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ClinicalTrials.gov Identifier: NCT03019406
Recruitment Status : Recruiting
First Posted : January 12, 2017
Last Update Posted : October 24, 2018
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:

Primary Objective:

To evaluate the safety profile of avalglucosidase alfa in patients with infantile-onset Pompe disease (IOPD) previously treated with alglucosidase alfa.

Secondary Objective:

To characterize the pharmacokinetic profile of avalglucosidase alfa and to evaluate the preliminary efficacy of avalglucosidase alfa in comparison to alglucosidase alfa.


Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Type II-Pompe's Disease Drug: avalglucosidase alfa GZ402666 Drug: alglucosidase alfa GZ419829 Phase 2

Detailed Description:
The duration of the study per patient will be up to 3 years that will consist of a 14-day screening period, that may be extended to up to 4 weeks in pre-specified situations, a 25-week treatment period, a 120-week extension period, and a 4-week post-treatment observation period.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response
Actual Study Start Date : October 12, 2017
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : April 2022


Arm Intervention/treatment
Experimental: avalglucosidase alfa
Administered intravenously every 2 weeks as an ascending dose cohort
Drug: avalglucosidase alfa GZ402666
Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous

Active Comparator: alglucosidase alfa
administered intravenously at current stable dose (i.e. administered regularly for a minimum of 6 months immediately prior to study entry)
Drug: alglucosidase alfa GZ419829
Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous




Primary Outcome Measures :
  1. Number of participants with adverse events [ Time Frame: Baseline to 6 months ]
  2. Number of participants with immunogenicity response [ Time Frame: Baseline to 6 months ]

Secondary Outcome Measures :
  1. Assessment of pharmacokinetic parameter: maximum concentration (Cmax) [ Time Frame: Baseline to 6 months ]
  2. Assessment of pharmacokinetic parameter: area under curve (AUC) [ Time Frame: Baseline to 6 months ]
  3. Change from baseline in Gross Motor Function Measure-88 Test [ Time Frame: Baseline to 6 months ]
  4. Change from revised Gross Motor Function Classification System score [ Time Frame: Baseline to 6 months ]
  5. Change from baseline in Pompe specific Pediatric Evaluation of Disability Inventory (Pompe PEDI) Functional Skills Scale: Mobility Domain Test score (normative standard score) [ Time Frame: Baseline to 6 months ]
  6. Change from baseline in Pompe PEDI Functional Skills Scale: Mobility Domain Test score (scaled score) [ Time Frame: Baseline to 6 months ]
  7. Change from baseline in Quick Motor Function Test scores [ Time Frame: Baseline to 6 months ]
  8. Change from baseline in Left Ventricular Mass Index [ Time Frame: Baseline to 6 months ]
  9. Change from baseline in Left Ventricular Mass Index Z score [ Time Frame: Baseline to 6 months ]
  10. Change from baseline in Eyelid position measurements: Interpalpebral fissure distance (IPFD) [ Time Frame: Baseline to 6 months ]
  11. Change from baseline in Eyelid position measurements: Margin reflex distance-1 (MRD-1) [ Time Frame: Baseline to 6 months ]
  12. Change from baseline in Eyelid position measurements: Margin pupil distance (MPD) [ Time Frame: Baseline to 6 months ]
  13. Change from baseline in Creatine kinase value [ Time Frame: Baseline to 6 months ]


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Ages Eligible for Study:   6 Months to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria:

  • The patient has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source.
  • The patient who has reached legal age of majority as defined by local regulation, or the patient's legal guardian(s) must provide signed informed consent prior to performing any study-related procedures. If the patient is legally minor per local regulations, assent shall be obtained from patients, if applicable.
  • The patient (and patient's legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol.
  • The patient is <18 years old.
  • The patient, if female and of childbearing potential, must have a negative serum pregnancy test (beta-human chorionic gonadotropin) and must not be breastfeeding at screening/baseline.
  • The patient has cardiomyopathy at the time of diagnosis: i.e, left ventricular mass index (LVMI) equivalent to mean age specific LVMI plus 2 standard deviations.
  • The patient has been receiving a stable dose of alglucosidase alfa regularly for a minimum of 6 months immediately prior to study entry.
  • For participants in Stage 1: The patient has documented evidence of clinical decline in at least 1 of the following parameters related to Pompe Disease and NOT related to intercurrent illness as assessed by the Investigator: respiratory function, motor skills, and/or cardiac parameters.
  • For participants in Stage 2: The patient has documented evidence of suboptimal clinical response in at least 1 of the following parameters related to Pompe Disease and NOT related to intercurrent illness as assessed by the Investigator: respiratory function, motor skills, and/or new onset of ptosis.

Exclusion criteria:

  • The patient has high antibody titer to alglucosidase alfa.
  • The patient has a high risk for a severe allergic reaction to neoGAA.
  • The patient requires any prohibited concomitant medications (e.g., immune modulatory treatment) for the duration of the study.
  • The patient has previously participated in any ACT14132 study cohort.
  • Female patient of childbearing potential not protected by highly effective contraceptive method of birth control and/or who is unwilling or unable to be tested for pregnancy

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.


Information from the National Library of Medicine

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): NCT03019406


Contacts
Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-Us@sanofi.com

Locations
United States, New York
Investigational Site Number 8400002 Recruiting
Valhalla, New York, United States, 10595
United States, North Carolina
Investigational Site Number 8400001 Recruiting
Durham, North Carolina, United States, 27710
United States, Washington
Investigational Site Number 8400005 Recruiting
Seattle, Washington, United States, 98105
France
Investigational Site Number 2500002 Recruiting
Paris Cedex 19, France, 75935
Investigational Site Number 2500001 Recruiting
Tours Cedex 1, France, 37044
Japan
Investigational Site Number 3920001 Recruiting
Fuchu-Shi, Japan
Investigational Site Number 3920002 Recruiting
Fuchu-Shi, Japan
Taiwan
Investigational Site Number 1580001 Recruiting
Taipei, Taiwan, 10043
United Kingdom
Investigational Site Number 8260001 Recruiting
London, United Kingdom, WC1N 3JH
Investigational Site Number 8260002 Recruiting
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Clinical Sciences & Operations Sanofi

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT03019406     History of Changes
Other Study ID Numbers: ACT14132
2016-003475-21 ( EudraCT Number )
U1111-1179-4616 ( Other Identifier: UTN )
First Posted: January 12, 2017    Key Record Dates
Last Update Posted: October 24, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Glycogen Storage Disease
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
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases