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Also known as sepiapterin
CNSA-001 · 2 trials · 2 indications
Analysis for participants ≥30 months to \<6 years of age.
Analysis for participants ≥6 years to 16 years of age.
Participants consumed 150 milliliters (mL) of Ensure™ every 5 minutes for the nutrient satiety test. At 5-minute intervals, participants scored their fullness using a rating scale that combines verbal descriptors on a scale graded 0 to 5 (0 = no symptoms, 1 = first sensation of fullness \[threshold\], 2 = mild, 3 = moderate, 4 = severe and 5 = maximum or unbearable fullness). Participants were told to stop when a score of 5 was obtained. The actual volume of Ensure™ consumed at this point was the maximum tolerated volume.
Participants consumed 150 mL of Ensure™ every 5 minutes for the nutrient satiety test. At 5-minute intervals, participants scored their fullness using a rating scale that combines verbal descriptors on a scale graded 0 to 5 (0: no symptoms, 1 = first sensation of fullness \[threshold\], 2 = mild, 3 = moderate, 4 = severe and 5 = maximum or unbearable fullness). Participants were told to stop when a score of 5 was obtained. The actual volume of Ensure™ consumed at this point was the maximum tolerated volume.
| Arm | Type | Description |
|---|---|---|
| Sepiapterin | EXPERIMENTAL | Participants will receive age- and weight-adjusted doses of sepiapterin orally once daily for up to 6 years. |
| CNSA-001 | EXPERIMENTAL | Participants will receive CNSA-001 20 mg/kg/day (10 mg/kg twice daily \[BID\]) as an oral suspension for 14 days. |
| Placebo | PLACEBO_COMPARATOR | Participants will receive placebo matching to CNSA-001 BID for 14 days. |
| Name | Type | Description |
|---|---|---|
| Sepiapterin | DRUG | Sepiapterin powder for oral use will be mixed in water or apple juice prior to administration. |
| CNSA-001 | DRUG | CNSA-001 Powder for Suspension |
| Placebo | DRUG | Placebo Suspension |
Key Inclusion Criteria: For all participants: * Women of childbearing potential must have a negative pregnancy test at Screening and agree to abstinence or the use of at least one highly effective form of contraception for the duration of the study, and for at least 90 days after the last dose of ...
Top 1 of 2 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vanda Pharmaceuticals Inc. | VNDA | 1 | PHASE4 | Tradipitant |
CNSA-001 is an investigational small molecule being developed for the treatment of gastroparesis, a condition where the stomach cannot empty properly. It is being studied specifically in women with diabetic gastroparesis. The drug is not yet approved and remains in clinical development.
CNSA-001 is being developed by PTC Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker PTCT. The company is conducting clinical research to evaluate the drug's safety and efficacy in patients with gastroparesis.
CNSA-001 is in Phase 2 clinical development. A Phase 2 study of the drug has been completed, but it remains investigational and has not received regulatory approval. Further clinical development would be needed before it could be considered for approval.
CNSA-001 has one completed clinical trial, registered as NCT03712124, titled "A Study of CNSA-001 in Women With Diabetic Gastroparesis." This Phase 2 study enrolled 21 female participants aged 18 years and older in the United States. The trial was randomized, double-blind, and placebo-controlled.
CNSA-001 is the primary name used for this investigational drug in clinical trials. No alternative names have been reported for this compound. It is distinct from other drugs in development and is being studied specifically for gastroparesis.