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Long-term Efficacy of Carglumic Acid in Organic Acidemia.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04284917
Recruitment Status : Enrolling by invitation
First Posted : February 26, 2020
Last Update Posted : February 26, 2020
Sponsor:
Information provided by (Responsible Party):
National Taiwan University Hospital

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).

Condition or disease Intervention/treatment Phase
Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) Drug: Carglumic Acid (Carbaglu®) Not Applicable

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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).
Actual Study Start Date : November 28, 2019
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2021


Arm Intervention/treatment
Experimental: Receive Carglumic Acid
Experimental Case_ Carglumic Acid
Drug: Carglumic Acid (Carbaglu®)

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 Measures :
  1. Number of emergency visits due to hyperammonemia within 12 months period [ Time Frame: 12 months ]
    Adverse Events


Secondary Outcome Measures :
  1. Time to first visit to the ER due to hyperammonemia from starting the treatment [ Time Frame: 12 months ]
    Adverse Events

  2. Assessment of Plasma ammonia level [ Time Frame: Baseline,3,6,9 and 12,months after receiving Carbaglu. ]
    Plasma ammonia level over the study treatment period.

  3. Number of days of hospitalization [ Time Frame: 12 months ]
    Number of days of hospitalization during study treatment period.

  4. Assessment of Acylcarnitine level [ Time Frame: Baseline,3,6,9 and 12,months after receiving Carbaglu. ]
    Acylcarnitine level for all patients

  5. Assessment of urine organic acid level [ Time Frame: Baseline,3,6,9 and 12,months after receiving Carbaglu. ]
    Measuring urine organic acid level for both diseases.

  6. Assessment of Plasma aminoacids' level [ Time Frame: Baseline,3,6,9 and 12,months after receiving Carbaglu. ]
    Measuring Plasma aminoacids' level for both diseases.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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®

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): NCT04284917


Locations
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Taiwan
National Taiwan University Hospital
Taipei, Taiwan, 10041
Sponsors and Collaborators
National Taiwan University Hospital
Investigators
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Principal Investigator: Ni-Chung Lee, M.D., Ph.D National Taiwan University Hospital
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Responsible Party: National Taiwan University Hospital
ClinicalTrials.gov Identifier: NCT04284917    
Other Study ID Numbers: 201810033MIPB
First Posted: February 26, 2020    Key Record Dates
Last Update Posted: February 26, 2020
Last Verified: November 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by National Taiwan University Hospital:
Carglumic Acid (Carbaglu®)
Additional relevant MeSH terms:
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Propionic Acidemia
Amino Acid Metabolism, Inborn Errors
Acidosis
Acid-Base Imbalance
Metabolic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn