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Long-term Efficacy of Carglumic Acid in Organic Acidemia.

Primary Purpose

Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)

Status
Unknown status
Phase
Not Applicable
Locations
Taiwan
Study Type
Interventional
Intervention
Carglumic Acid (Carbaglu®)
Sponsored by
National Taiwan University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) focused on measuring Carglumic Acid (Carbaglu®)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesAccepts Healthy Volunteers

Inclusion Criteria:

  1. PA or MMA confirmed using the following criteria:

    • PA confirmed by the measurement of acylcarnitine profile, urine organic acid, measurement of propionyl Co-A carboxylase in leukocytes or cultured fibroblasts or by DNA molecular testing of PCCA (Propionyl CoA Carboxylase, Alpha Polypeptide) or PCCB (propionyl CoA carboxylase, beta polypeptide) gene
    • MMA confirmed by the measurement of acylcarnitine profile, urine organic acid, measurement of methymalonyl Co-A mutase in culture fibroblasts or DNA molecular testing of mutgene.
  2. Male or female Children of 18 years old or less.
  3. Had experienced Hyperammonemia(NH3 ≧100 uM) before.
  4. Not participating in any other clinical trial in the previous 30 days

Exclusion Criteria:

  1. Patients with other organic acidemia or any other cause of hyperammonemia
  2. Patient receiving other investigational therapy for PA or MMA
  3. Patient with PA or MMA and other inherited genetic conditions or congenital anomalies
  4. Past history of hypersensitivity or drug allergy to Carbaglu®

Sites / Locations

  • National Taiwan University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Receive Carglumic Acid

Arm Description

Experimental Case_ Carglumic Acid

Outcomes

Primary Outcome Measures

Number of emergency visits due to hyperammonemia within 12 months period
Adverse Events

Secondary Outcome Measures

Time to first visit to the ER due to hyperammonemia from starting the treatment
Adverse Events
Assessment of Plasma ammonia level
Plasma ammonia level over the study treatment period.
Number of days of hospitalization
Number of days of hospitalization during study treatment period.
Assessment of Acylcarnitine level
Acylcarnitine level for all patients
Assessment of urine organic acid level
Measuring urine organic acid level for both diseases.
Assessment of Plasma aminoacids' level
Measuring Plasma aminoacids' level for both diseases.

Full Information

First Posted
February 24, 2020
Last Updated
February 25, 2020
Sponsor
National Taiwan University Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT04284917
Brief Title
Long-term Efficacy of Carglumic Acid in Organic Acidemia.
Official Title
Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).
Study Type
Interventional

2. Study Status

Record Verification Date
November 2019
Overall Recruitment Status
Unknown status
Study Start Date
November 28, 2019 (Actual)
Primary Completion Date
December 2021 (Anticipated)
Study Completion Date
December 2021 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Taiwan University Hospital

4. Oversight

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

5. Study Description

Brief Summary
Evaluate the Long Term Effectiveness & Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).
Detailed Description
Study procedures: A. Study period: 24 months; treatment period: 12 months. B. Patients will receive Carglumic Acid (Carbaglu®) 50 mg/kg/day in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and vitamin B12). C. Patients need to regularly come back for follow up examination on Day 0, month 3, month 6, and month 12. D. Patients should report any adverse event that occur during treatment period. E. Patients' number of emergency visits due to hyperammonemia, ammonia level, acylcarnitine, urinary organic acid analysis, plasma amino acid analysis will be recorded during treatment period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)
Keywords
Carglumic Acid (Carbaglu®)

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Receive Carglumic Acid
Arm Type
Experimental
Arm Description
Experimental Case_ Carglumic Acid
Intervention Type
Drug
Intervention Name(s)
Carglumic Acid (Carbaglu®)
Intervention Description
A. Study period: 24 months; treatment period: 12 months. B. Patients will receive Carglumic Acid (Carbaglu®) 50 mg/kg/day in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and vitamin B12) C. Patients need to regularly come back for follow up examination on Day 0, month 3, month 6, and month 12. (Check Plasma Amino Acid Analysis, Ammonia, acylcarnitine profile, Urinary organic acid analysis.) D. Patients should report any adverse event that occur during treatment period. E. Patients' number of emergency visits due to hyperammonemia, ammonia level, acylcarnitine, urinary organic acid analysis, plasma amino acid analysis will be recorded during treatment period.
Primary Outcome Measure Information:
Title
Number of emergency visits due to hyperammonemia within 12 months period
Description
Adverse Events
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Time to first visit to the ER due to hyperammonemia from starting the treatment
Description
Adverse Events
Time Frame
12 months
Title
Assessment of Plasma ammonia level
Description
Plasma ammonia level over the study treatment period.
Time Frame
Baseline,3,6,9 and 12,months after receiving Carbaglu.
Title
Number of days of hospitalization
Description
Number of days of hospitalization during study treatment period.
Time Frame
12 months
Title
Assessment of Acylcarnitine level
Description
Acylcarnitine level for all patients
Time Frame
Baseline,3,6,9 and 12,months after receiving Carbaglu.
Title
Assessment of urine organic acid level
Description
Measuring urine organic acid level for both diseases.
Time Frame
Baseline,3,6,9 and 12,months after receiving Carbaglu.
Title
Assessment of Plasma aminoacids' level
Description
Measuring Plasma aminoacids' level for both diseases.
Time Frame
Baseline,3,6,9 and 12,months after receiving Carbaglu.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria: PA or MMA confirmed using the following criteria: PA confirmed by the measurement of acylcarnitine profile, urine organic acid, measurement of propionyl Co-A carboxylase in leukocytes or cultured fibroblasts or by DNA molecular testing of PCCA (Propionyl CoA Carboxylase, Alpha Polypeptide) or PCCB (propionyl CoA carboxylase, beta polypeptide) gene MMA confirmed by the measurement of acylcarnitine profile, urine organic acid, measurement of methymalonyl Co-A mutase in culture fibroblasts or DNA molecular testing of mutgene. Male or female Children of 18 years old or less. Had experienced Hyperammonemia(NH3 ≧100 uM) before. Not participating in any other clinical trial in the previous 30 days Exclusion Criteria: Patients with other organic acidemia or any other cause of hyperammonemia Patient receiving other investigational therapy for PA or MMA Patient with PA or MMA and other inherited genetic conditions or congenital anomalies Past history of hypersensitivity or drug allergy to Carbaglu®
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ni-Chung Lee, M.D., Ph.D
Organizational Affiliation
National Taiwan University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
10041
Country
Taiwan

12. IPD Sharing Statement

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Long-term Efficacy of Carglumic Acid in Organic Acidemia.

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