Ignite Creation Date:
2024-05-06 @ 3:44 PM
Last Modification Date:
2024-10-26 @ 1:55 PM
Study NCT ID:
NCT04732429
Status:
TERMINATED
Last Update Posted:
2024-01-05
First Post:
2021-01-21
Brief Title:
Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia
Sponsor:
HemoShear Therapeutics
Organization:
HemoShear Therapeutics
Study Overview
Official Title:
A Phase 2 Open-label Dose Escalation Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Followed by a Randomized Double-blind Placebo-controlled 2-period Crossover Study and an Open-label Long-term Extension Study
Status:
TERMINATED
Status Verified Date:
2024-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
The decision to terminate the study was based on business considerations by the Sponsor
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
HERO
Brief Summary:
This is an interventional study to assess the safety PK and efficacy of HST5040 in 12 subjects - 6 with Methylmalonic Acidemia MMA and 6 with Propionic Acidemia PA The study consists of 3 parts
Part A Open-label within-subject dose escalation study in PA and MMA subjects 2 years old to identify a safe and pharmacologically active optimal dose of HST5040 for use in Part B Subjects will continue in a Part A open-label extension until all subjects complete Part A and the optimal dose of HST5040 is identified for use in Part B
Part B 6-month randomized double-blind placebo-controlled 2-period crossover in the same subjects from Part A to evaluate safety and efficacy of the optimal dose of HST5040 in addition to standard of care SoC
Part C open-label long-term extension study in PA and MMA subjects 2 years old N approximately 12 6 each to evaluate the long-term safety and efficacy of the optimal dose of HST5040
This study will determine whether HST5040 can improve levels of disease-associated toxins that accumulate in patients with PA and MMA
Part A Open-label within-subject dose escalation study in PA and MMA subjects 2 years old to identify a safe and pharmacologically active optimal dose of HST5040 for use in Part B Subjects will continue in a Part A open-label extension until all subjects complete Part A and the optimal dose of HST5040 is identified for use in Part B
Part B 6-month randomized double-blind placebo-controlled 2-period crossover in the same subjects from Part A to evaluate safety and efficacy of the optimal dose of HST5040 in addition to standard of care SoC
Part C open-label long-term extension study in PA and MMA subjects 2 years old N approximately 12 6 each to evaluate the long-term safety and efficacy of the optimal dose of HST5040
This study will determine whether HST5040 can improve levels of disease-associated toxins that accumulate in patients with PA and MMA
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None