Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 2:10 AM
Ignite Modification Date:
2025-12-25 @ 2:10 AM
NCT ID:
NCT04925960
Eligibility Criteria:
Inclusion Criteria:
1. Age ≥ 2 and \< 18 years
2. Diagnosis of PMM2-CDG, based on molecularly confirmed biallelic PMM2 pathogenic variants (can be historical diagnosis with lab report on file)
3. Informed consent (and assent, as applicable) document personally signed by the legally authorized representative of the patient, indicating that the patient's parent/guardian has been informed and agreed to all aspects of the study
4. Be willing and able to adhere to the study assessments and schedule described in the protocol and consent/assent documents
5. Negative urine pregnancy test (only for female subjects of child-bearing potential)
6. For subjects of child-bearing potential-only, subject has been counseled on and agrees to the requirement either for double barrier contraceptive methods and/or for total abstinence from prior to randomization through 3-months after the cessation of treatment.
Exclusion Criteria:
1. Known or suspected other known CDG
2. Known allergy to aldose reductase inhibitors
3. Hypersensitivity to epalrestat
4. Hepatic impairment defined as any one of the following:
1. AST/ALT \>5x ULN in the 6 months prior to screening
2. Bilirubin \>2X ULN in the last 6 months prior to screening
3. Synthetic liver dysfunction (albumin deficiency \< 2.8 mmol/L) at screening, or
4. Diagnosis of liver fibrosis (Fibroscan \> 7 kPa) confirmed by liver elastogram at screening
5. Renal impairment defined as serum creatinine: \> 0.5 mg/dL (≤ 6 years); \> 0.7 mg/dL (7-10 years); \> 1.24 mg/dL (≥ 11 years)
6. Low platelet count (\< 125x109 /L)
7. Any other clinically significant lab abnormality which, in the opinion of the investigator, should be exclusionary
8. Anemia (Hgb \< 10 g/dL)
9. Use of an investigational drug, including acetazolamide, in the past 28 days; use of an investigational biologic in the past 12 months
10. Concurrent or planned participation in interventional protocol or use of any other unapproved therapeutics, and,
11. Any other medical condition, which, in the opinion of the investigator, will interfere with the patient's ability to comply with the protocol, compromises patient safety, or interferes with the interpretation of the study results.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
2 Years
Maximum Age:
17 Years
Study:
NCT04925960
Study Brief:
Protocol Section:
NCT04925960