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30 Diseases |  |
10 Trials |
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10 Trials |  |
301 News |  |
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- | triheptanoin (UX007) / Ultragenyx
Clinical, Journal: Long-term follow-up in an open-label trial of triheptanoin in GLUT1 deficiency syndrome: a sustained dramatic effect. (Pubmed Central) - Jun 18, 2020
No abstract available
- || triheptanoin (UX007) / Ultragenyx
Clinical, Journal: Open-label long-term treatment of add-on triheptanoin in adults with drug-resistant epilepsy. (Pubmed Central) - Jun 13, 2020
However, only two people finished and extended the 48-week add-on treatment phase, despite lack of safety or tolerability issues.More studies focused on improved treatment formulations, the potential of lower dosages, and efficacy are needed. Trial registration number: ACTRN12615000406505.
- triheptanoin (UX007) / Ultragenyx
Mortality associated with long-chain fatty acid oxidation disorders: Observations from an Expanded Access Program for triheptanoin () - May 6, 2020 - Abstract #AMCP2020AMCP_106;
Additional research is needed to better understand the mortality of this disease. Sponsorship: Ultragenyx Pharmaceutical Inc.
- || Dojolvi (triheptanoin) / Ultragenyx
Enrollment open: Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I (clinicaltrials.gov) - May 5, 2020
P1, N=5, Recruiting, Sponsor: Areeg El-Gharbawy
Sponsorship: Ultragenyx Pharmaceutical Inc. Not yet recruiting --> Recruiting
- || Dojolvi (triheptanoin) / Ultragenyx
Trial completion date, Trial primary completion date: Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program (clinicaltrials.gov) - Apr 22, 2020
P=N/A, N=20, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Not yet recruiting --> Recruiting Trial completion date: Apr 2020 --> Jul 2020 | Trial primary completion date: Apr 2020 --> Jul 2020
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment closed: Diet Treatment Glucose Transporter Type 1 Deficiency (G1D) (clinicaltrials.gov) - Apr 14, 2020
P2, N=45, Active, not recruiting, Sponsor: University of Texas Southwestern Medical Center
Trial completion date: Apr 2020 --> Jul 2020 | Trial primary completion date: Apr 2020 --> Jul 2020 Recruiting --> Active, not recruiting
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment closed, Phase classification: Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D (clinicaltrials.gov) - Apr 12, 2020
P2, N=15, Active, not recruiting, Sponsor: University of Texas Southwestern Medical Center
Recruiting --> Active, not recruiting Enrolling by invitation --> Active, not recruiting | Phase classification: P1 --> P2
- || Dojolvi (triheptanoin) / Ultragenyx
Clinical, P2 data, Journal: Results from a 78-week, single-arm, open-label phase 2 study to evaluate UX007 in pediatric and adult patients with severe long-chain fatty acid oxidation disorders (LC-FAOD). (Pubmed Central) - Apr 2, 2020
Initiation of UX007 eliminated hypoglycemia events leading to hospitalization (from 11 pre-UX007 hospitalizations, 0.30 events/year vs. 0; p = 0.067) and intensive care unit (ICU) care (from 2 pre-UX007 ICU admissions, 0.05 events/year vs. 0; p = 0.161) and reduced cardiomyopathy events (3 events vs. 1 event; 0.07 to 0.02 events/year; 69.7% decrease). The majority of treatment-related adverse events (AEs) were mild to moderate gastrointestinal symptoms, including diarrhea, vomiting, and abdominal or gastrointestinal pain, which can be managed with smaller, frequent doses mixed with food.
- | triheptanoin (UX007) / Ultragenyx, ubidecarenone IV (BPM 31510 IV) / Berg
Clinical, Review, Journal: A Review of the Clinical Evidence for Complementary and Alternative Medicine in Huntington's Disease. (Pubmed Central) - Mar 29, 2020
Currently, there is no evidence supporting disease modification or symptom improvement with any specific dietary or nutraceutical supplement for HD. Supervised exercise and contemporary dance are safe for people with HD, but more robust studies are warranted to guide specific recommendations for these and other mind-body interventions.
- | Dojolvi (triheptanoin) / Ultragenyx
Trial completion: HEMIHEP: Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood (clinicaltrials.gov) - Feb 12, 2020
P2, N=10, Completed, Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
Supervised exercise and contemporary dance are safe for people with HD, but more robust studies are warranted to guide specific recommendations for these and other mind-body interventions. Active, not recruiting --> Completed
- | Dojolvi (triheptanoin) / Ultragenyx
Trial completion date: Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I (clinicaltrials.gov) - Jan 30, 2020
P1, N=5, Not yet recruiting, Sponsor: Areeg El-Gharbawy
Active, not recruiting --> Completed Trial completion date: Mar 2022 --> Nov 2021
- | Dojolvi (triheptanoin) / Ultragenyx
Trial completion date, Trial primary completion date: Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I (clinicaltrials.gov) - Jan 23, 2020
P1, N=5, Not yet recruiting, Sponsor: Areeg El-Gharbawy
Trial completion date: Mar 2022 --> Nov 2021 Trial completion date: Jun 2020 --> Mar 2022 | Trial primary completion date: May 2020 --> Sep 2021
- | Dojolvi (triheptanoin) / Ultragenyx
Trial completion date, Trial primary completion date: Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin (clinicaltrials.gov) - Jan 22, 2020
P2, N=12, Recruiting, Sponsor: Center for Rare Neurological Diseases, Norcross, GA
Trial completion date: Jun 2020 --> Mar 2022 | Trial primary completion date: May 2020 --> Sep 2021 Trial completion date: Feb 2019 --> Feb 2021 | Trial primary completion date: Feb 2019 --> Feb 2020
- | triheptanoin (UX007) / Ultragenyx
Preclinical, Journal: A triheptanoin-supplemented diet rescues hippocampal hyperexcitability and seizure susceptibility in FoxG1 mice. (Pubmed Central) - Dec 29, 2019
This manipulation abated altered neural activity and normalized the enhanced susceptibility to proconvulsant-induced seizures, in addition to rescuing the altered expression of KCC2 and increasing the levels of the GABA transporter vGAT. In conclusion, our data show that FoxG1 haploinsufficiency causes dysfunction of hippocampal circuits and increases the susceptibility to a proconvulsant insult, and that these alterations are rescued by triheptanoin dietary treatment.
- ||| Dojolvi (triheptanoin) / Ultragenyx
Enrollment change: A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS) (clinicaltrials.gov) - Dec 24, 2019
P1/2, N=15, Completed, Sponsor: Richard Bedlack, M.D., Ph.D.
In conclusion, our data show that FoxG1 haploinsufficiency causes dysfunction of hippocampal circuits and increases the susceptibility to a proconvulsant insult, and that these alterations are rescued by triheptanoin dietary treatment. N=10 --> 15
- ||| Dojolvi (triheptanoin) / Ultragenyx
Enrollment change: Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies (clinicaltrials.gov) - Dec 5, 2019
P2, N=150, Enrolling by invitation, Sponsor: Ultragenyx Pharmaceutical Inc
N=10 --> 15 N=100 --> 150
- || triheptanoin (UX007) / Ultragenyx
Clinical, Journal: A double-blind, placebo-controlled trial of triheptanoin in adult polyglucosan body disease and open-label, long-term outcome. (Pubmed Central) - Nov 11, 2019
P2
This study also emphasizes the difficulty of conducting trials in very rare diseases presenting with a wide clinical heterogeneity. ClinicalTrials.gov Identifier: NCT00947960.
- | triheptanoin (UX007) / Ultragenyx
Journal: Ketogenic and anaplerotic dietary modifications ameliorate seizure activity in Drosophila models of mitochondrial encephalomyopathy and glycolytic enzymopathy. (Pubmed Central) - Nov 10, 2019
We have found that reduction of glycolysis does not confer seizure protection, but that dietary supplementation with ketone bodies or the anaplerotic lipid triheptanoin, which directly replenishes the citric acid cycle, can mimic the success of the ketogenic diet even in the presence of standard carbohydrate levels...Importantly, one of these mutants is TPI, which models human glycolytic enzymopathy, an incurable metabolic disorder with severe neurological consequences. Overall, these studies reveal widespread success of the KD in Drosophila, further cementing its status as an excellent model for studies of KD treatment and mechanism, and reveal key insights into the therapeutic potential of dietary therapy against neuronal hyperexcitability in epilepsy and metabolic disease.
- ||| Dojolvi (triheptanoin) / Ultragenyx
Trial termination: Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS) (clinicaltrials.gov) - Nov 6, 2019
P2, N=15, Terminated, Sponsor: Ultragenyx Pharmaceutical Inc
Overall, these studies reveal widespread success of the KD in Drosophila, further cementing its status as an excellent model for studies of KD treatment and mechanism, and reveal key insights into the therapeutic potential of dietary therapy against neuronal hyperexcitability in epilepsy and metabolic disease. Active, not recruiting --> Terminated; Study was halted prematurely due to lack of efficacy
- || Dojolvi (triheptanoin) / Ultragenyx
Trial completion date, Trial primary completion date: Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program (clinicaltrials.gov) - Nov 3, 2019
P=N/A, N=22, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Active, not recruiting --> Terminated; Study was halted prematurely due to lack of efficacy Trial completion date: Nov 2019 --> Feb 2020 | Trial primary completion date: Nov 2019 --> Feb 2020
- ||| Dojolvi (triheptanoin) / Ultragenyx
Trial termination: Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) (clinicaltrials.gov) - Oct 29, 2019
P3, N=44, Terminated, Sponsor: Ultragenyx Pharmaceutical Inc
Trial completion date: Nov 2019 --> Feb 2020 | Trial primary completion date: Nov 2019 --> Feb 2020 Active, not recruiting --> Terminated; Study was halted prematurely due to lack of efficacy.
- | Dojolvi (triheptanoin) / Ultragenyx
Trial completion: The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V) (clinicaltrials.gov) - Oct 16, 2019
P2, N=22, Completed, Sponsor: Rigshospitalet, Denmark
Active, not recruiting --> Terminated; Study was halted prematurely due to lack of efficacy. Active, not recruiting --> Completed
- || triheptanoin (UX007) / Ultragenyx
NDA, PDUFA date: $RARE FDA Accepts NDA for UX007 (triheptanoin) for Treatment of Long-chain Fatty Acid Oxidation Disorders. PDUFA 07/31/20 (Twitter) - Oct 14, 2019
- | Dojolvi (triheptanoin) / Ultragenyx
Trial primary completion date: Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) (clinicaltrials.gov) - Aug 28, 2019
P3, N=44, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Active, not recruiting --> Completed Trial primary completion date: Aug 2018 --> Dec 2019
- || triheptanoin (UX007) / Ultragenyx
NDA: $RARE Submits NDA for UX007 (triheptanoin) for Treatment of Long-Chain Fatty Acid Oxidation Disorders (Twitter) - Aug 1, 2019
- | Dojolvi (triheptanoin) / Ultragenyx
Trial completion date, Trial primary completion date: TRIHEP3: A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease (clinicaltrials.gov) - Jul 15, 2019
P2, N=100, Active, not recruiting, Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
Trial primary completion date: Aug 2018 --> Dec 2019 Trial completion date: Jun 2020 --> Dec 2020 | Trial primary completion date: Jun 2019 --> Dec 2019
- | Dojolvi (triheptanoin) / Ultragenyx
Trial completion date, Trial primary completion date: Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS) (clinicaltrials.gov) - May 9, 2019
P2, N=15, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Trial completion date: Jun 2020 --> Dec 2020 | Trial primary completion date: Jun 2019 --> Dec 2019 Trial completion date: Apr 2019 --> Dec 2019 | Trial primary completion date: Apr 2019 --> Dec 2019
- | Dojolvi (triheptanoin) / Ultragenyx
Trial completion date: Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) (clinicaltrials.gov) - May 9, 2019
P3, N=43, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Trial completion date: Apr 2019 --> Dec 2019 | Trial primary completion date: Apr 2019 --> Dec 2019 Trial completion date: Apr 2019 --> Dec 2019
- | Dojolvi (triheptanoin) / Ultragenyx
Phase classification: Post Study Continuation of C7 for G1D (clinicaltrials.gov) - Apr 20, 2019
P=N/A, N=0, No Longer Available, Sponsor: Juan Pascual
Trial completion date: Apr 2019 --> Dec 2019 Phase classification: P1 --> P=N/A
- || Dojolvi (triheptanoin) / Ultragenyx
Enrollment closed, Trial completion date, Trial primary completion date: TRIHEP3: A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease (clinicaltrials.gov) - Apr 18, 2019
P2, N=100, Active, not recruiting, Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
Phase classification: P1 --> P=N/A Recruiting --> Active, not recruiting | Trial completion date: Jun 2019 --> Jun 2020 | Trial primary completion date: Jun 2017 --> Jun 2019
- Dojolvi (triheptanoin) / Ultragenyx
Trial completion: A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS) (clinicaltrials.gov) - Apr 10, 2019
P1/2, N=10, Completed, Sponsor: Richard Bedlack, M.D., Ph.D.
Recruiting --> Active, not recruiting | Trial completion date: Jun 2019 --> Jun 2020 | Trial primary completion date: Jun 2017 --> Jun 2019 Active, not recruiting --> Completed
- | Lacromid (bezafibrate) / Remedica
Review, Journal: Management and diagnosis of mitochondrial fatty acid oxidation disorders: focus on very-long-chain acyl-CoA dehydrogenase deficiency. (Pubmed Central) - Mar 23, 2019
Additionally, although the clinical efficacy of bezafibrate remains controversial, a recent open-label clinical trial showed efficacy of this drug in improving quality of life. These drugs may be promising for the treatment of FAODs, though further studies are required.
- || Dojolvi (triheptanoin) / Ultragenyx
Enrollment closed: Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program (clinicaltrials.gov) - Feb 18, 2019
P=N/A, N=22, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
These drugs may be promising for the treatment of FAODs, though further studies are required. Not yet recruiting --> Active, not recruiting
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment change, Trial completion date, Trial withdrawal, Trial primary completion date: Triheptanoin (C7 Oil), a Food Supplement, for Glucose Transporter Type I Deficiency (G1D) (clinicaltrials.gov) - Feb 16, 2019
P1/2, N=0, Withdrawn, Sponsor: Juan Pascual
Not yet recruiting --> Active, not recruiting N=50 --> 0 | Trial completion date: Jun 2019 --> Jun 2020 | Not yet recruiting --> Withdrawn | Trial primary completion date: Jun 2019 --> Jun 2020
- | Dojolvi (triheptanoin) / Ultragenyx
Trial completion date, Trial initiation date, Trial primary completion date: Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I (clinicaltrials.gov) - Feb 8, 2019
P1, N=5, Not yet recruiting, Sponsor: Areeg El-Gharbawy
N=50 --> 0 | Trial completion date: Jun 2019 --> Jun 2020 | Not yet recruiting --> Withdrawn | Trial primary completion date: Jun 2019 --> Jun 2020 Trial completion date: Jun 2019 --> Jun 2020 | Initiation date: Dec 2018 --> Apr 2019 | Trial primary completion date: May 2019 --> May 2020
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment closed, Trial primary completion date: The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V) (clinicaltrials.gov) - Jan 10, 2019
P2, N=22, Active, not recruiting, Sponsor: Rigshospitalet, Denmark
Trial completion date: Jun 2019 --> Jun 2020 | Initiation date: Dec 2018 --> Apr 2019 | Trial primary completion date: May 2019 --> May 2020 Recruiting --> Active, not recruiting | Trial primary completion date: Sep 2018 --> Apr 2018
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment change: ACETO-TCA: Study of the Acute Effects of Triheptanoin in Heart Failure (clinicaltrials.gov) - Dec 14, 2018
P1/2, N=0, Withdrawn, Sponsor: University of Texas Southwestern Medical Center
Recruiting --> Active, not recruiting | Trial primary completion date: Sep 2018 --> Apr 2018 N=24 --> 0
- |||||| Dojolvi (triheptanoin) / Ultragenyx
New trial: Expanded Access to Triheptanoin (clinicaltrials.gov) - Dec 12, 2018
P, N=0, Available, Sponsor: Ultragenyx Pharmaceutical Inc
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment closed, Trial completion date, Trial primary completion date: Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS) (clinicaltrials.gov) - Dec 11, 2018
P2, N=15, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
N=24 --> 0 Enrolling by invitation --> Active, not recruiting | Trial completion date: Dec 2020 --> Apr 2019 | Trial primary completion date: Sep 2020 --> Apr 2019
- | Dojolvi (triheptanoin) / Ultragenyx
Trial completion date: Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) (clinicaltrials.gov) - Dec 11, 2018
P3, N=43, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Enrolling by invitation --> Active, not recruiting | Trial completion date: Dec 2020 --> Apr 2019 | Trial primary completion date: Sep 2020 --> Apr 2019 Trial completion date: Sep 2021 --> Apr 2019
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment open, Trial completion date, Trial primary completion date: Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D (clinicaltrials.gov) - Nov 5, 2018
P1, N=15, Enrolling by invitation, Sponsor: University of Texas Southwestern Medical Center
Trial completion date: Sep 2021 --> Apr 2019 Not yet recruiting --> Enrolling by invitation | Trial completion date: Apr 2023 --> Jul 2023 | Trial primary completion date: Apr 2023 --> Jul 2021
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment closed: A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS) (clinicaltrials.gov) - Oct 17, 2018
P1/2, N=10, Active, not recruiting, Sponsor: Richard Bedlack, M.D., Ph.D.
Not yet recruiting --> Enrolling by invitation | Trial completion date: Apr 2023 --> Jul 2023 | Trial primary completion date: Apr 2023 --> Jul 2021 Recruiting --> Active, not recruiting
- | Dojolvi (triheptanoin) / Ultragenyx
New P1 trial: Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I (clinicaltrials.gov) - Sep 11, 2018
P1, N=5, Recruiting, Sponsor: Areeg El-Gharbawy
- | Dojolvi (triheptanoin) / Ultragenyx
New P2 trial: The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses (clinicaltrials.gov) - Aug 22, 2018
P2, N=20, Recruiting, Sponsor: Rigshospitalet, Denmark
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment open: A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS) (clinicaltrials.gov) - Aug 13, 2018
P1/2, N=10, Recruiting, Sponsor: Richard Bedlack, M.D., Ph.D.
Recruiting --> Active, not recruiting Not yet recruiting --> Recruiting
- | Dojolvi (triheptanoin) / Ultragenyx
Trial completion, Trial completion date, Trial primary completion date: BEAT7: Brain Energy and Aging With Triheptanoin (clinicaltrials.gov) - Aug 10, 2018
P1/2, N=15, Completed, Sponsor: Université de Sherbrooke
Not yet recruiting --> Recruiting Recruiting --> Completed | Trial completion date: Dec 2018 --> Aug 2018 | Trial primary completion date: Dec 2018 --> Aug 2018
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment open: Diet Treatment Glucose Transporter Type 1 Deficiency (G1D) (clinicaltrials.gov) - Jun 25, 2018
P2, N=45, Recruiting, Sponsor: University of Texas Southwestern Medical Center
Recruiting --> Completed | Trial completion date: Dec 2018 --> Aug 2018 | Trial primary completion date: Dec 2018 --> Aug 2018 Not yet recruiting --> Recruiting
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment change, Trial completion date, Trial primary completion date: Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS) (clinicaltrials.gov) - Jun 21, 2018
P2, N=15, Enrolling by invitation, Sponsor: Ultragenyx Pharmaceutical Inc
Not yet recruiting --> Recruiting N=40 --> 15 | Trial completion date: Sep 2018 --> Dec 2020 | Trial primary completion date: Sep 2018 --> Sep 2020
- | Dojolvi (triheptanoin) / Ultragenyx
Enrollment closed: Treatment Development of Triheptanoin (G1D) (clinicaltrials.gov) - Jun 21, 2018
P1, N=36, Active, not recruiting, Sponsor: University of Texas Southwestern Medical Center
N=40 --> 15 | Trial completion date: Sep 2018 --> Dec 2020 | Trial primary completion date: Sep 2018 --> Sep 2020 Recruiting --> Active, not recruiting
- Dojolvi (triheptanoin) / Ultragenyx
New P1/2 trial: A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS) (clinicaltrials.gov) - Apr 24, 2018
P1/2, N=10, Not yet recruiting, Sponsor: Richard Bedlack, M.D., Ph.D.