CLINICAL TRIALS PROFILE FOR CARNITOR SF

All Clinical Trials for CARNITOR SF

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00481013 ↗	Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy	Completed	Abbott	Phase 2	2007-07-01	The primary objective of this proposal is to determine whether oral VPA is effective in treating SMA in adult patients.
NCT00481013 ↗	Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy	Completed	Families of Spinal Muscular Atrophy	Phase 2	2007-07-01	The primary objective of this proposal is to determine whether oral VPA is effective in treating SMA in adult patients.
NCT00227266 ↗	Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy	Completed	Abbott	Phase 2	2005-09-01	This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8 years of age. Cohort 2 is an open label protocol for SMA "standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes. Outcome measures will include blood chemistries, functional testing, pulmonary function testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign parameters. Six centers will enroll a total of 90 patients.
NCT00227266 ↗	Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy	Completed	Families of Spinal Muscular Atrophy	Phase 2	2005-09-01	This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8 years of age. Cohort 2 is an open label protocol for SMA "standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes. Outcome measures will include blood chemistries, functional testing, pulmonary function testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign parameters. Six centers will enroll a total of 90 patients.
NCT00227266 ↗	Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy	Completed	Leadiant Biosciences, Inc.	Phase 2	2005-09-01	This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8 years of age. Cohort 2 is an open label protocol for SMA "standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes. Outcome measures will include blood chemistries, functional testing, pulmonary function testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign parameters. Six centers will enroll a total of 90 patients.
NCT00227266 ↗	Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy	Completed	Sigma Tau Pharmaceuticals, Inc.	Phase 2	2005-09-01	This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8 years of age. Cohort 2 is an open label protocol for SMA "standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes. Outcome measures will include blood chemistries, functional testing, pulmonary function testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign parameters. Six centers will enroll a total of 90 patients.
NCT00227266 ↗	Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy	Completed	University of Utah	Phase 2	2005-09-01	This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8 years of age. Cohort 2 is an open label protocol for SMA "standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes. Outcome measures will include blood chemistries, functional testing, pulmonary function testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign parameters. Six centers will enroll a total of 90 patients.

Clinical Trial Conditions for CARNITOR SF

Condition Name

Condition Name for CARNITOR SF
Intervention	Trials
Spinal Muscular Atrophy	2
Carnitine Deficiency	2
Ph-Like Acute Lymphoblastic Leukemia	1
Prematurity	1

Condition MeSH

Condition MeSH for CARNITOR SF
Intervention	Trials
Muscular Atrophy	2
Atrophy	2
Precursor Cell Lymphoblastic Leukemia-Lymphoma	2
Leukemia, Lymphoid	2

Clinical Trial Locations for CARNITOR SF

Trials by Country

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−

Trials by Country for CARNITOR SF
Location	Trials
United States	8
Canada	2
Netherlands	1

Trials by US State

+

−

Trials by US State for CARNITOR SF
Location	Trials
Ohio	2
Texas	1
New York	1
Wisconsin	1
Utah	1

Clinical Trial Progress for CARNITOR SF

Clinical Trial Phase

Clinical Trial Phase for CARNITOR SF
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	1
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for CARNITOR SF
Clinical Trial Phase	Trials
Completed	2
Active, not recruiting	1
Terminated	1

Clinical Trial Sponsors for CARNITOR SF

Sponsor Name

Sponsor Name for CARNITOR SF
Sponsor	Trials
Abbott	2
Families of Spinal Muscular Atrophy	2
University of Utah	2

Sponsor Type

Sponsor Type for CARNITOR SF
Sponsor	Trials
Other	11
Industry	4
NIH	1

CARNITOR SF: Clinical Trials, Market Analysis, and Projections

Introduction

CARNITOR SF, containing the active ingredient levocarnitine, is a crucial medication used in the treatment of primary systemic carnitine deficiency and other metabolic disorders. This article will delve into the clinical trials, market analysis, and future projections for CARNITOR SF.

Clinical Trials and Efficacy

Primary Systemic Carnitine Deficiency

Clinical trials have demonstrated that CARNITOR SF effectively increases carnitine levels in patients with primary systemic carnitine deficiency. This condition is characterized by low concentrations of levocarnitine in plasma, red blood cells, and tissues, leading to symptoms such as recurrent episodes of Reye-like encephalopathy, hypoketotic hypoglycemia, and cardiomyopathy[2].

Metabolic Abnormalities

CARNITOR SF has been shown to alleviate metabolic abnormalities in patients with inborn errors of metabolism, including glutaric aciduria II, methylmalonic aciduria, propionic acidemia, and medium chain fatty acylCoA dehydrogenase deficiency. It helps clear acylCoA compounds that disrupt intermediary metabolism, thereby preventing life-threatening acidosis[2].

Pharmacokinetics

Studies have indicated that CARNITOR SF is bio-equivalent in its tablet and oral solution forms. The maximum plasma concentration (Cmax) is achieved approximately 3.3 hours after administration, and the drug is primarily excreted in the urine[2].

Side Effects and Safety Profile

Common Side Effects

CARNITOR SF is associated with several common side effects, including gastrointestinal issues such as diarrhea, abdominal pain, vomiting, and nausea. Other frequent side effects include headache, dizziness, and paresthesia[4].

Serious Side Effects

Less common but serious side effects can include seizures, increased platelet aggregation, hypertriglyceridemia, and kidney failure in chronic hemodialysis patients. It is crucial for patients to monitor these potential side effects and consult their healthcare provider if they occur[4].

Market Analysis

Pricing and Availability

The pricing of CARNITOR SF varies depending on the formulation and dosage. For example, the 330 mg tablet has an average pharmacy cost of around $1.17 per unit, while the 100 mg/mL oral solution costs approximately $0.40 per mL[5].

Market Demand

The demand for CARNITOR SF is driven by the need for effective treatments for primary systemic carnitine deficiency and other metabolic disorders. Given the specific and critical nature of these conditions, the market demand is consistent and growing as more patients are diagnosed and treated.

Competitive Landscape

CARNITOR SF is a unique product with a specific indication, which limits direct competition. However, other treatments for related metabolic disorders may indirectly compete for market share. The brand's strong clinical evidence and FDA approval position it favorably in the market.

Projections and Future Outlook

Market Growth

The market for CARNITOR SF is expected to grow as awareness and diagnosis of carnitine deficiency increase. Advances in genetic testing and metabolic disorder diagnosis are likely to expand the patient population, driving demand for the drug.

Regulatory Environment

The FDA approval of CARNITOR SF for primary systemic carnitine deficiency provides a strong regulatory foundation. Continued compliance with FDA guidelines and potential expansion into other indications could further solidify its market position.

Research and Development

Ongoing research into the broader applications of levocarnitine, such as its role in energy metabolism and potential benefits in other conditions, could lead to new indications and expanded market opportunities.

Key Takeaways

• Clinical Efficacy: CARNITOR SF is effective in increasing carnitine levels and alleviating symptoms of primary systemic carnitine deficiency and other metabolic disorders.
• Safety Profile: While generally well-tolerated, CARNITOR SF can have significant side effects that require monitoring.
• Market Demand: The demand for CARNITOR SF is driven by the need for effective treatments for specific metabolic disorders.
• Future Outlook: The market for CARNITOR SF is expected to grow with increased diagnosis and awareness, and potential new indications.

FAQs

What is CARNITOR SF used for?

CARNITOR SF is used in the treatment of primary systemic carnitine deficiency and other metabolic disorders such as glutaric aciduria II, methylmalonic aciduria, propionic acidemia, and medium chain fatty acylCoA dehydrogenase deficiency[2].

What are the common side effects of CARNITOR SF?

Common side effects include diarrhea, abdominal pain, vomiting, nausea, headache, dizziness, and paresthesia[4].

How is CARNITOR SF administered?

CARNITOR SF is available in various forms, including tablets, oral solution, and injectable solution. The choice of formulation depends on the patient's needs and the specific condition being treated[2].

What is the pricing for CARNITOR SF?

The pricing varies by formulation, with the 330 mg tablet costing around $1.17 per unit and the 100 mg/mL oral solution costing approximately $0.40 per mL[5].

Is CARNITOR SF FDA-approved?

Yes, CARNITOR SF is FDA-approved for the treatment of primary systemic carnitine deficiency[2].

What are the potential future indications for CARNITOR SF?

Ongoing research may lead to new indications for CARNITOR SF, potentially expanding its use beyond current approved conditions.

Sources

1. CARNITOR® - Clinical Outcomes. Carnitor.com.
2. Label: CARNITOR SF - levocarnitine solution. DailyMed.
3. Jaguar Health Reports Phase 3 OnTarget Trial Results for its Cancer Supportive Care Drug Crofelemer. Jaguar Health.
4. Carnitor SF Side Effects: Common, Severe, Long Term. Drugs.com.
5. Drug prices and trends for CARNITOR. DrugPatentWatch.