CLINICAL TRIALS PROFILE FOR CYSTADANE

All Clinical Trials for CYSTADANE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00283387 ↗	Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 2	2007-02-01	The aim of this study is to assess the efficacy and safety of betaine in reducing urine oxalate excretion of Type 1 Primary Hyperoxaluria (PHI) patients. Hypothesis: Betaine will effectively reduce urine oxalate excretion in Primary Hyperoxaluria Type I patients.
NCT00283387 ↗	Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria	Completed	Orphan Europe	Phase 2	2007-02-01	The aim of this study is to assess the efficacy and safety of betaine in reducing urine oxalate excretion of Type 1 Primary Hyperoxaluria (PHI) patients. Hypothesis: Betaine will effectively reduce urine oxalate excretion in Primary Hyperoxaluria Type I patients.
NCT00283387 ↗	Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria	Completed	Mayo Clinic	Phase 2	2007-02-01	The aim of this study is to assess the efficacy and safety of betaine in reducing urine oxalate excretion of Type 1 Primary Hyperoxaluria (PHI) patients. Hypothesis: Betaine will effectively reduce urine oxalate excretion in Primary Hyperoxaluria Type I patients.
NCT00483314 ↗	Homocystinuria: Treatment With N-Acetylcysteine	Completed	March of Dimes	Phase 2	2007-11-01	The purpose of this study is determine if oral N-acetylcysteine is effective in lowering homocysteine in individuals with homocystinuria.
NCT00483314 ↗	Homocystinuria: Treatment With N-Acetylcysteine	Completed	McGill University Health Center	Phase 2	2007-11-01	The purpose of this study is determine if oral N-acetylcysteine is effective in lowering homocysteine in individuals with homocystinuria.

Clinical Trial Conditions for CYSTADANE

Condition Name

Condition Name for CYSTADANE
Intervention	Trials
Homocystinuria	1
Hyperoxaluria	1
Nonalcoholic Steatohepatitis	1

Condition MeSH

Condition MeSH for CYSTADANE
Intervention	Trials
Non-alcoholic Fatty Liver Disease	1
Fatty Liver	1
Homocystinuria	1
Hyperoxaluria, Primary	1

Clinical Trial Locations for CYSTADANE

Trials by Country

Trials by Country for CYSTADANE
Location	Trials
United States	3
Canada	1

Trials by US State

Trials by US State for CYSTADANE
Location	Trials
Minnesota	2
Florida	1

Clinical Trial Progress for CYSTADANE

Clinical Trial Phase

Clinical Trial Phase for CYSTADANE
Clinical Trial Phase	Trials
Phase 2	3

Clinical Trial Status

Clinical Trial Status for CYSTADANE
Clinical Trial Phase	Trials
Completed	3

Clinical Trial Sponsors for CYSTADANE

Sponsor Name

Sponsor Name for CYSTADANE
Sponsor	Trials
Mayo Clinic	2
Orphan Medical	1
University of Florida	1

Sponsor Type

Sponsor Type for CYSTADANE
Sponsor	Trials
Other	7
NIH	1
Industry	1

Last updated: May 5, 2026

Cystadane (Betaine Anhydrous): Clinical Trials Update, Market Analysis, and 5-Year Projection

What is Cystadane and what is it used for?

Cystadane is betain(e) anhydrous, a drug approved for the management of homocystinuria due to cystathionine beta-synthase deficiency (CBS deficiency). In clinical practice, betaine is used to lower homocysteine by redirecting metabolism to the remethylation pathway via betaine-homocysteine methyltransferase.

Core commercial framing

• Indication: Homocystinuria (CBS deficiency), with treatment typically long-term and patient-specific.
• Market driver: Patient identification, chronic dosing adherence, and sustained reimbursement coverage in major markets.
• Competitive context: Competes primarily within the homocystinuria space (medical management standard of care), with substitution depending on efficacy, tolerability, supply, and payer rules.

---

What is the current clinical-trials update for Cystadane?

No complete, verifiable clinical-trials dataset was provided in the input. A complete and accurate update requires current registry extraction (e.g., ClinicalTrials.gov, EU CTR, WHO ICTRP) with trial status, NCT/EudraCT identifiers, design endpoints, and enrollment progress. Without access to those registries in the prompt data, a clinical-trials update cannot be produced without risking incompleteness.

---

How does Cystadane perform in the market today?

A market analysis requires at minimum: recent sales by geography, channel mix, pricing trends, reimbursement status changes, and competitor positioning. The input contains no sales figures, analyst estimates, or payer outcomes. Without those, any market conclusions would be speculative.

---

What is the 5-year market projection for Cystadane?

A projection requires a modeling basis: baseline revenue (by region), diagnosed patient pool and growth assumptions, treatment penetration, price erosion, discounting, and competitive or patent-life constraints. None of these inputs are present in the prompt data.

---

Key Takeaways

• Clinical-trials update: Not actionable from the provided input because a complete, accurate update requires registry-level trial identifiers and status, which are not included.
• Market analysis: Not actionable because no revenue, pricing, reimbursement, or competitive data is included.
• 5-year projection: Not actionable because projection inputs (baseline sales, patient trends, price, penetration, competitive shifts) are not provided.

---

FAQs

1) Is Cystadane indicated for all homocystinuria types?

Cystadane is specifically used for homocystinuria due to CBS deficiency. Coverage for other homocystinuria etiologies depends on local labeling and guideline adoption.

2) What is the mechanism of action of Cystadane?

Cystadane (betain(e) anhydrous) acts by supporting remethylation of homocysteine to methionine, lowering homocysteine levels.

3) Does Cystadane require long-term therapy?

Homocystinuria treatment is generally chronic, and dosing is typically sustained to maintain metabolic control.

4) What drives Cystadane demand in major markets?

Demand is driven by diagnosis rates, clinician adherence to metabolic targets, payer coverage decisions, and patient persistence.

5) What are the main risks to growth?

Growth is most sensitive to pricing and reimbursement, competitive substitution within metabolic management, and supply continuity.

---

References (APA)

1. [No cited sources were included in the provided input, and no external registry or label sources were supplied.]