Clinical trial • Endocrinology|Musculoskeletal|Rare Disease

AVALGLUCOSIDASE ALFA for Late-onset Pompe disease|Pompe disease

Clinical trial of AVALGLUCOSIDASE ALFA for Late-onset Pompe disease|Pompe disease. 10 participants.

Overview

Trial Therapeutic Area
Endocrinology|Musculoskeletal|Rare Disease
Trial Disease
Late-onset Pompe disease|Pompe disease
Drug Modality
Peptide/protein/enzyme

Key dates

Initial CTIS Submission Date
14-11-2023
First CTIS Authorization Date
16-01-2024

Trial design

Clinical trial across 1 site in Denmark.

Target Sample Size
10
Trial Duration For Participant
365

Eligibility

Recruits 10 No vulnerable population selected; participants are adults (Age ≥18). No assent/parental consent handling is indicated in the record..

Pregnancy Exclusion
Pregnancy
Vulnerable Population
No vulnerable population selected; participants are adults (Age ≥18). No assent/parental consent handling is indicated in the record.

Inclusion criteria

  • {"criterion_text":"- Subjects with genetically verified Late-onset Pompe Disease"}
  • {"criterion_text":"- Age ≥18"}
  • {"criterion_text":"- Naïve to enzyme replacement therapy"}

Exclusion criteria

  • {"criterion_text":"- Ferrous objects in or around the body"}
  • {"criterion_text":"- Pacemaker or other implanted electronic devices"}
  • {"criterion_text":"- Claustrophobia"}
  • {"criterion_text":"- Inability to understand the purpose of the trial or corporate for the conduction of the experiments"}
  • {"criterion_text":"- Participation in other trials that may interfere with the results"}
  • {"criterion_text":"- Competing conditions at risk of compromising the results of the study, evaluated by the investigator"}
  • {"criterion_text":"- Pregnancy"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Change in glycogen concentration of the hamstring muscles between baseline and 12-month follow-up","definition_or_measurement_approach":""}

Secondary endpoints

  • {"endpoint_text":"- Change in glycogen concentration of the calf, hamstring, anterior thigh, and lumbar muscles over time – both within the first treatment cycle, and at 3, 6 and 12-months follow-up","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 3, 6 and 12-months follow-up.","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Correlations between baseline glycogen levels and change in FVC between baseline and 12-months follow-up.","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Correlations between baseline glycogen levels and change in 6MWT between baseline and 12-months follow-up.","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Correlations between baseline glycogen levels and change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 12-months follow-up.","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
10
Recruitment Window Months
60
Consent Approach
Informed consent obtained from adult participants (Age ≥18). Subject information and informed consent form documents are listed in the dossier but details (e.g. languages) are not provided in the record.

Geography

Total Number Of Sites
1
Total Number Of Participants
10

Denmark

Earliest CTIS Part Ii Submission Date
28-11-2023
Latest Decision Or Authorization Date
22-08-2025
Processing Time Days
633
Number Of Sites
1
Number Of Participants
10

Sites

Site Name
Rigshospitalet
Department Name
Copenhagen Neuromuscular Center
Principal Investigator Name
John Vissing
Principal Investigator Email
john.vissing@regionh.dk
Contact Person Name
John Vissing
Contact Person Email
john.vissing@regionh.dk
Number Of Participants
10

Sponsor

Primary sponsor

Full Name
Rigshospitalet
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
Denmark

Third parties

  • {"country":"Denmark","full_name":"Frederiksberg Hospital","duties_or_roles":"sponsorDuties code 1","organisation_type":"Hospital/Clinic/Other health care facility"}

Investigational products

Investigational Product Name
Nexviadyme 100 mg powder for concentrate for solution for infusion
Active Substance
AVALGLUCOSIDASE ALFA
Modality
Peptide/protein/enzyme
Routes Of Administration
INFUSION
Route
INFUSION
Authorisation Status
Authorised (marketing authorisation EU/1/21/1579/001)
Maximum Dose
Max daily dose amount 1.5 mg/Kg; Max total dose amount 520 mg/Kg

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