Health TrialsbyHealthPanel

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

ConditionPompe Disease (Late-onset)
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age6+
SponsorGeneCradle Inc

About this trial

This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with late-onset Pompe disease （LOPD） who are ≥ 6 years old will be studied.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age ≥ 6 years, males or females;

Patient has a diagnosis of LOPD;

Patient has upright FVC ≥ 30% of predicted normal value;

A 6MWT ≥ 40 meters, assistive device allowed;

Disqualifiers

Patient who has any history or concurrent clinical organic disease, including cardiovascular and liver diseases, respiratory system, nervous system disease, or any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.

Patient who requires invasive mechanical ventilation, or rely on noninvasive non-non-invasive assisted ventilation when sitting upright;

Patient who is positive for human immunodeficiency (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;

Patient with a history of glucocorticoid allergy;

Trial design

Design model

Sequential

Treatments tested in this trial

  • GC301

    Genetic

    GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene

Treatment groups

33 Participants
are divided into 2 treatment groups
Group A: Low dosage groupExperimental treatment 1 intervention
Group B: High dosage groupExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number of Participants With Adverse Events

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Time frame
52 weeks
2

Dose-limiting toxicity (DLT) rate based on protocol-specific adverse events (Phase 1)

Time frame
within 30 days after treatment
3

Percent Predicted Upright Forced Vital Capacity (FVC)(Phase 2)

Change from baseline in percentage of predicted FVC measured by pulmonary function testing

Time frame
52 weeks

Secondary outcomes

1

6-Minute Walk Test

Change from baseline in the distance walked in the 6 minute walk test (6MWT), which is a standardized assessment of how far an individual can walk on a hard, flat surface in a period of 6 minutes

Time frame
52 weeks
2

Maximum Inspiratory Pressure (MIP)

Change from baseline in MIP measured by pulmonary function testing

Time frame
52 weeks
3

Maximum Expiratory Pressure (MEP)

Change from baseline in MEP measured by pulmonary function testing

Time frame
52 weeks
4

Muscle Status Testing - Quick Motor Function Test (QMFT) Measure

Measurement of functional motor abilities using the Quick Motor Function Test (QMFT) will be performed and the results compared with baseline.

Time frame
52 weeks

Other outcomes

1

GAA enzymatic activity

Change from baseline in GAA enzymatic activity in muscle biopsies

Time frame
52 weeks
2

GAA enzymatic activity

Change from baseline in GAA enzymatic activity in blood

Time frame
52 weeks
3

Glycogen content in muscle

Change from baseline in glycogen content in muscle biopsies

Time frame
52 weeks

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

GeneCradle Inc

Lead sponsor

Source ClinicalTrials.gov
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