Propionic AcidemiaPhase 3CAMPNCT02426775
Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia
A Phase 3 study testing Carglumic Acid in people with Propionic Acidemia and Methylmalonic Acidemia, run by King Abdullah International Medical Research Center at 2 sites in Saudi Arabia.
Trial summary
A Phase IIIb (Three b), Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA). Read more about this study
- Who is it for?
- Ages Up to 15 years
- Conditions the study is for
- Propionic Acidemia, Methylmalonic Acidemia
- What is being tested?
- Carglumic Acid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 2 sitesSaudi Arabia
- Study dates
- Nov 2015to 19 Mar 2019
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female Children of 15 years old or less
- Parent or legal guardian agree to participate and to sign the (Institutional Review Board) IRB approved Informed Consent Form (assent forms will be waived due to the mental disability of those children)
- Not participating in any other clinical trial in the previous 30 days
- 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.
- Expected survival of at least 6 months, for the purpose of this study Survival expectance will be defined as patient not admitted to the Pediatric Intensive Care Unit (PICU) due to hyperammonemia more than 2 times per year or asymptomatic patients diagnosed by newborn screening program or stable chronic patients who are followed up at outpatient clinic.
Who cannot join
- Patients with other organic acidemia or any other cause of hyperammonemia
- Patient receiving other investigational therapy for PA or MMA
- Past history of hypersensitivity or drug allergy to Carbaglu®
- Patient with PA or MMA and other inherited genetic conditions or congenital anomalies
Trial locations and contacts
2 sites in 1 country.
Saudi Arabia2 sites
King Abdullah Specialist Children Hospital, King Abdulaziz Medical City
Riyadh, 11426
King Fahad Medical City
Riyadh
About this study
Official title: Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness of the Use of Carbaglu® in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA)
A Phase IIIb (Three b), Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA). Carbaglu® clinical experience in Organic Acidemia (OA) is limited to a non-comparative retrospective collection of data from patients who had received Carbaglu® for 1 to 15 days.
There is no current evidence supporting the use of carglumic acid for the chronic management of patients with OA. The investigators are proposing a randomized multicentre prospective clinical trial to evaluate long-term effects of the use of Carbaglu® (50mg/kg/day) combined with standard chronic therapy in patients with PA and MMA compared to standard chronic therapy alone.
- Conditions
- Propionic Acidemia, Methylmalonic Acidemia
- Being tested
- Carglumic Acid
- Medical subject headings
- Propionic Acidemia, Methylmalonic acidemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Carglumic AcidDrug
Also called Carbaglu
Carglumic Acid 50 mg/kg/day (twice daily)
| Study group | Role | What they receive |
|---|---|---|
| Methylmalonic Acidemia Control Arm | No intervention | patients with Methylmalonic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and vitamin B12) |
| Methylmalonic Acidemia Active arm | Experimental | patients with Methylmalonic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and vitamin B12) Receives: Drug: Carglumic Acid |
| Propionic Acidemia Control Arm | No intervention | patients with Propionic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and biotin) |
| Propionic Acidemia Active arm | Experimental | patients with Propionic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and biotin) Receives: Drug: Carglumic Acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of emergency visits due to hyperammonemia within 24 months period
Measured: 24 months
Secondary outcomes (6)
Time to first visit to the ER due to hyperammonemia from starting the treatment.
Measured: 24 months
Plasma ammonia levels over the study treatment period.
Measured: 24 months
Number of days of hospitalization
Measured: 24 months
Acylcarnitine level for all patients
Measured: once on screening visit
Measuring urine organic acid levels for both diseases.
Measured: 24 months
Measuring Plasma aminoacids' levels for both diseases
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2015 (estimated)
- Study startNov 2015
- Primary completion19 Mar 2019
- Study completion19 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Propionic AcidemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- King Abdullah International Medical Research Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dr. Majid Alfadhel, King Abdullah International Medical Research Center
- Investigators
- Majid A. Alfadhel, MD, Principal investigator, King Abdulaziz Medical City, National Guard / Riyadh-Saudi Arabia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02426775
- Sponsor's protocol ID
- RC 13/116
- Acronym
- CAMP
- Status last verified
- Apr 2019
- First submitted
- 22 Apr 2015
- Last updated
- 25 Apr 2019
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Nashabat M, Obaid A, Al Mutairi F, Saleh M, Elamin M, Ahmed H, Ababneh F, Eyaid W, Alswaid A, Alohali L, Faqeih E, Aljeraisy M, Hussein MA, Alasmari A, Alfadhel M. Evaluation of long-term effectiveness of the use of carglumic acid in patients with propionic acidemia (PA) or methylmalonic acidemia (MMA): study protocol for a randomized controlled trial. BMC Pediatr. 2019 Jun 13;19(1):195. doi: 10.1186/s12887-019-1571-y.
PubMed 31196016
Study at a glance
From the registry record, last updated by the sponsor on 25 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness of the Use of Carbaglu® in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA)
- ClinicalTrials.gov ID
- NCT02426775
- Acronym / sponsor ID
- CAMP / RC 13/116
- Conditions
- Propionic Acidemia, Methylmalonic Acidemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- King Abdullah International Medical Research CenterUniversity, hospital or other
- Study start
- Nov 2015
- Primary completion
- 19 Mar 2019 (actual)
- Study completion
- 19 Mar 2019 (actual)
- First posted
- 27 Apr 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase IIIb (Three b), Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA). Carbaglu® clinical experience in Organic Acidemia (OA) is limited to a non-comparative retrospective collection of data from patients who had received Carbaglu® for 1 to 15 days.
There is no current evidence supporting the use of carglumic acid for the chronic management of patients with OA. The investigators are proposing a randomized multicentre prospective clinical trial to evaluate long-term effects of the use of Carbaglu® (50mg/kg/day) combined with standard chronic therapy in patients with PA and MMA compared to standard chronic therapy alone.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Methylmalonic Acidemia Control Arm | No intervention | –patients with Methylmalonic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and vitamin B12) |
| Methylmalonic Acidemia Active arm | Experimental | Drug: Carglumic Acidpatients with Methylmalonic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and vitamin B12) |
| Propionic Acidemia Control Arm | No intervention | –patients with Propionic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and biotin) |
| Propionic Acidemia Active arm | Experimental | Drug: Carglumic Acidpatients with Propionic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and biotin) |
Who may qualify
- Ages
- Up to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Male or female Children of 15 years old or less
- Parent or legal guardian agree to participate and to sign the (Institutional Review Board) IRB approved Informed Consent Form (assent forms will be waived due to the mental disability of those children)
- Not participating in any other clinical trial in the previous 30 days
- PA or MMA confirmed using the following criteria:
- Expected survival of at least 6 months, for the purpose of this study Survival expectance will be defined as patient not admitted to the Pediatric Intensive Care Unit (PICU) due to hyperammonemia more than 2 times per year or asymptomatic patients diagnosed by newborn screening program or stable chronic patients who are followed up at outpatient clinic.
Key exclusion criteria (4 listed)
- Patients with other organic acidemia or any other cause of hyperammonemia
- Patient receiving other investigational therapy for PA or MMA
- Past history of hypersensitivity or drug allergy to Carbaglu®
- Patient with PA or MMA and other inherited genetic conditions or congenital anomalies
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Carglumic AcidAlso: CarbagluCarglumic Acid 50 mg/kg/day (twice daily) | Drug | Methylmalonic Acidemia Active arm, Propionic Acidemia Active arm |
Primary outcome
Number of emergency visits due to hyperammonemia within 24 months period
Time frame: 24 months
6 secondary outcomes
Time to first visit to the ER due to hyperammonemia from starting the treatment.
Time frame: 24 months
Plasma ammonia levels over the study treatment period.
Time frame: 24 months
Number of days of hospitalization
Time frame: 24 months
Acylcarnitine level for all patients
Time frame: once on screening visit
Measuring urine organic acid levels for both diseases.
Time frame: 24 months
Measuring Plasma aminoacids' levels for both diseases
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Apr 2019.
- Derived from this study: PubMed 31196016Nashabat M, Obaid A, Al Mutairi F, Saleh M, Elamin M, Ahmed H, Ababneh F, Eyaid W, Alswaid A, Alohali L, Faqeih E, Aljeraisy M, Hussein MA, Alasmari A, Alfadhel M. Evaluation of long-term effectiveness of the use of carglumic acid in patients with propionic acidemia (PA) or methylmalonic acidemia (MMA): study protocol for a randomized controlled trial. BMC Pediatr. 2019 Jun 13;19(1):195. doi: 10.1186/s12887-019-1571-y.
Related trials for Propionic Acidemia
RecruitingObservational study
Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
Metabolic Disease, Propionic Acidemia
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingPhase 1
AAVrh10-PCCA Gene Therapy for Propionic Acidemia
Propionic Acidemia
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
RecruitingPhase 1/2
A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
Propionic Acidemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Majid A. Alfadhel, MD principal investigator
King Abdulaziz Medical City, National Guard / Riyadh-Saudi Arabia
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Carglumic Acid in people with Propionic Acidemia and Methylmalonic Acidemia, run by King Abdullah International Medical Research Center at 2 sites in Saudi Arabia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Saudi Arabia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of emergency visits due to hyperammonemia within 24 months period (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02426775, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Apr 2019.