Long-chain Fatty Acid Transport DeficiencyPhase 3NCT02201368
Triheptanoin Treatment Trial for Patients With Long-chain Fatty Acid Beta-oxidation Defects
A Phase 3 study testing Triheptanoin (SpezialölÒ 107®) and MCT (Medium-Chain Triglycerides) in people with Long-chain Fatty Acid Transport Deficiency, run by Maria Luz Couce Pico at 1 site in Spain.
Trial summary
The purpose of this study is to determine if administration Triheptanoin is an effective treatment for defects of the long-chain fatty acid beta-oxidation in young adults or adults. Period of treatment and follow-up will be 16 months. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Long-chain Fatty Acid Transport Deficiency
- What is being tested?
- Triheptanoin (SpezialölÒ 107®) and MCT (Medium-Chain Triglycerides)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteSpain
- Study dates
- Nov 2009to Oct 2011
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- All patients with any of the following conditions:
- Long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency (LCHAD).
- Very long-chain acyl-coenzyme A dehydrogenase deficiency (VLCAD.)
- Mitochondrial trifunctional protein (MTP).
- Carnitine palmitoyltransferase I deficiency (CPT I).
- Carnitine Palmitoyltransferase II (CPT II).
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- Carnitine-acylcarnitine translocase deficiency (CACT).
- Positive skin biopsy: patients were deemed to commence the dietary treatment with Triheptanoin after evaluating the individual response in vitro in cultured fibroblasts. This response is based on the measurement of the production of propionyl-CoA, the incubation with fatty acids odd-chain, compared with control group fibroblasts.
- The informed consent must be signed by the patient or family, in the case of minors.
Who cannot join
- No patient/family collaboration or the application of dietary treatment.
- No in vitro test response.
- Do not meet the inclusion criteria.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Study to Evaluate the Effectiveness of Dietary Treatment With Triheptanoin in Patients With Long-chain Fatty Acid Beta-oxidation Defects
The purpose of this study is to determine if administration Triheptanoin is an effective treatment for defects of the long-chain fatty acid beta-oxidation in young adults or adults. Period of treatment and follow-up will be 16 months.
Read the detailed description
Triheptanoin treatment, in patients with long-chain fatty acid beta-oxidation defects, could cause not only a great improvement in their quality of life, also could prevent life-threatening signs, reducing symptoms and serious complications of their disease, like cardiomyopathy, Reye-like syndrome episodes and rhabdomyolysis. This result would occur by the effect of propionyl CoA primer on the Krebs cycle and, at the same time, would produce a gluconeogenic effect.
This treatment opens the door to be used in other diseases such as pyruvate carboxylase deficiency, glycogen storage disease and other diseases with energy problems.
All patients will be followed up until 16 months.
- Being tested
- Triheptanoin (SpezialölÒ 107®), MCT (Medium-Chain Triglycerides)
- Keywords
- Triglycerides; Lipid Metabolism; Fatty Acids.
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Triheptanoin (SpezialölÒ 107®)Drug
- Randomization: Treatment with Triheptanoin for 6 months
- Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months
- Crossover: Treatment with MCT(Medium-Chain Triglycerides)for 6 months
MCT (Medium-Chain Triglycerides)Dietary supplement
- Randomization: Treatment with MCT(Medium-Chain Triglycerides)for 6 months
- Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months
- Crossover: Treatment with Triheptanoin for 6 months
| Study group | Role | What they receive |
|---|---|---|
| Triheptanoin | Experimental | Not described Receives: Drug: Triheptanoin (SpezialölÒ 107®); Dietary Supplement: MCT (Medium-Chain Triglycerides) |
| MCT (Medium-Chain Triglycerides) | Active comparator | Not described Receives: Drug: Triheptanoin (SpezialölÒ 107®); Dietary Supplement: MCT (Medium-Chain Triglycerides) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of metabolic decompensation.
This is a combined endpoint, including the number and/or severity of episodes of hypoglycemia, rhabdomyolysis, cardiomyopathy and liver failure after starting treatment with trihepatnoin.
Measured: up to 16 months
Secondary outcomes (3)
Differences in the profiles of acylcarnitines with control.
Measured: 6 months and 6 months in each arm treatment
Average values of transaminase and creatin kinase.
Measured: 6 months and 6 months in each arm treatment
Differences in the fatty acid composition of plasma and red blood cells.
Measured: 6 months and 6 months in each arm treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jul 2014 (estimated)
- Study startNov 2009
- Primary completionMar 2010
- Study completionOct 2011
Why it stopped: IMP management difficulties
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Long-chain Fatty Acid Transport DeficiencyStudies 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
- Crossover
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Maria Luz Couce Pico
- Sponsor type
- University, hospital or other
- Collaborators
- Fundación Ramón Domínguez
- Responsible party
- Sponsor-investigator, Maria Luz Couce Pico, Hospital Clinico Universitario de Santiago
- Investigators
- Mª Luz Couce Pico, Study chair, Hospital Clínico Universitario de SantiagoJaume Campistol Plana, Principal investigator, Hospital Sant Joan de Déu. BarcelonaMercedes Martínez-Pardo, Principal investigator, Hospital Universitario Ramon y CajalMónica Ruiz Pons, Principal investigator, Hospital Clínico Universitario Ntra. Sra. de Candelaria. TenerifeMª Teresa García Silva, Principal investigator, Hospital 12 de Octubre. MadridPablo Sanjurjo, Principal investigator, Hospital de Cruces de Baracaldo - BilbaoKoldo Aldamiz, Principal investigator, Hospital de Cruces de Baracaldo - BilbaoInmaculada García Jiménez, Principal investigator, Hospital Universitario Miguel Servet, Zaragoza
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02201368
- Sponsor's protocol ID
- 2007/084
- Other IDs
- 2007-005578-29
- Status last verified
- Jul 2014
- First submitted
- 22 Mar 2013
- Last updated
- 28 Jul 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study to Evaluate the Effectiveness of Dietary Treatment With Triheptanoin in Patients With Long-chain Fatty Acid Beta-oxidation Defects
- ClinicalTrials.gov ID
- NCT02201368
- Acronym / sponsor ID
- 2007/084
- Keywords
- Triglycerides; Lipid Metabolism; Fatty Acids.
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: IMP management difficulties
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Maria Luz Couce PicoUniversity, hospital or other
- Collaborators
- Fundación Ramón Domínguez
- Study start
- Nov 2009
- Primary completion
- Mar 2010 (actual)
- Study completion
- Oct 2011 (actual)
- First posted
- 28 Jul 2014 (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.
The purpose of this study is to determine if administration Triheptanoin is an effective treatment for defects of the long-chain fatty acid beta-oxidation in young adults or adults. Period of treatment and follow-up will be 16 months.
Detailed description
Triheptanoin treatment, in patients with long-chain fatty acid beta-oxidation defects, could cause not only a great improvement in their quality of life, also could prevent life-threatening signs, reducing symptoms and serious complications of their disease, like cardiomyopathy, Reye-like syndrome episodes and rhabdomyolysis. This result would occur by the effect of propionyl CoA primer on the Krebs cycle and, at the same time, would produce a gluconeogenic effect.
This treatment opens the door to be used in other diseases such as pyruvate carboxylase deficiency, glycogen storage disease and other diseases with energy problems.
All patients will be followed up until 16 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Triheptanoin | Experimental | Drug: Triheptanoin (SpezialölÒ 107®), Dietary Supplement: MCT (Medium-Chain Triglycerides) |
| MCT (Medium-Chain Triglycerides) | Active comparator | Drug: Triheptanoin (SpezialölÒ 107®), Dietary Supplement: MCT (Medium-Chain Triglycerides) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency (LCHAD).
- Very long-chain acyl-coenzyme A dehydrogenase deficiency (VLCAD.)
- Mitochondrial trifunctional protein (MTP).
- Carnitine palmitoyltransferase I deficiency (CPT I).
- Carnitine Palmitoyltransferase II (CPT II).
Key exclusion criteria (3 listed)
- No patient/family collaboration or the application of dietary treatment.
- No in vitro test response.
- Do not meet the inclusion criteria.
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 |
|---|---|---|
| Triheptanoin (SpezialölÒ 107®)* Randomization: Treatment with Triheptanoin for 6 months * Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months * Crossover: Treatment with MCT(Medium-Chain Triglycerides)for 6 months | Drug | MCT (Medium-Chain Triglycerides), Triheptanoin |
| MCT (Medium-Chain Triglycerides)* Randomization: Treatment with MCT(Medium-Chain Triglycerides)for 6 months * Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months * Crossover: Treatment with Triheptanoin for 6 months | Dietary supplement | MCT (Medium-Chain Triglycerides), Triheptanoin |
Primary outcome
Number of metabolic decompensation.
Time frame: up to 16 months
This is a combined endpoint, including the number and/or severity of episodes of hypoglycemia, rhabdomyolysis, cardiomyopathy and liver failure after starting treatment with trihepatnoin.
3 secondary outcomes
Differences in the profiles of acylcarnitines with control.
Time frame: 6 months and 6 months in each arm treatment
Average values of transaminase and creatin kinase.
Time frame: 6 months and 6 months in each arm treatment
Differences in the fatty acid composition of plasma and red blood cells.
Time frame: 6 months and 6 months in each arm treatment
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2014.
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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.
Mª Luz Couce Pico study chair
Hospital Clínico Universitario de Santiago
Jaume Campistol Plana principal investigator
Hospital Sant Joan de Déu. Barcelona
Mercedes Martínez-Pardo principal investigator
Hospital Universitario Ramon y Cajal
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Triheptanoin (SpezialölÒ 107®) and MCT (Medium-Chain Triglycerides) in people with Long-chain Fatty Acid Transport Deficiency, run by Maria Luz Couce Pico at 1 site in Spain.
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 9 inclusion and 3 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: withdrawn, as of the sponsor's last update on 28 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. 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 was withdrawn before enrolling participants, so there are no results.
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 metabolic decompensation. (time frame: up to 16 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Jul 2014.