Ignite Creation Date:
2025-12-25 @ 2:15 AM
Ignite Modification Date:
2025-12-26 @ 5:28 PM
Study NCT ID:
NCT04069260
Status:
TERMINATED
Last Update Posted:
2020-05-27
First Post:
2019-08-16
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis
Sponsor:
Eloxx Pharmaceuticals, Inc.
Organization:
Study Overview
Official Title:
A Phase 2, Single Center, Open-Label, Multiple Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Daily Subcutaneously Administered ELX-02 in Patients With Nephropathic Cystinosis Bearing One or More CTNS Gene (Cystinosin) Nonsense Mutations
Status:
TERMINATED
Status Verified Date:
2019-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Due to study design limitations, this study has been discontinued and will not proceed with the 2nd cohort as contemplated in the original protocol.
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a Phase 2 open label study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 in patients with cystinosis with nonsense mutation in at least one allele.
Six patients will be enrolled in the trial.
The study will comprise of the following periods for each patient:
* A screening period of up to 6 weeks
* A total treatment period of 4 weeks
* A safety follow-up period of 4 weeks after the last treatment
Each patient will receive three escalating doses as follows:
* Treatment period 1: ELX-02 0.5 mg/kg SC daily for 7 days (total dose not to exceed 3.5 mg/kg for this week; the daily dose will be individualized to achieve the target weekly exposure of about 47.5 µg\*h/mL)
* Treatment period 2: ELX-02 1.0 mg/kg SC daily for 7 days (total dose not to exceed 7.0 mg/kg for this week; the daily dose will be individualized to achieve the target weekly exposure of about 95 µg\*h/mL)
* Treatment period 3: ELX-02 2.0 mg/kg SC daily for 14 days (total dose not to exceed 14 mg/kg for these two weeks; the daily dose will be individualized to achieve the target weekly exposure of about 190 µg\*h/mL)
Six patients will be enrolled in the trial.
The study will comprise of the following periods for each patient:
* A screening period of up to 6 weeks
* A total treatment period of 4 weeks
* A safety follow-up period of 4 weeks after the last treatment
Each patient will receive three escalating doses as follows:
* Treatment period 1: ELX-02 0.5 mg/kg SC daily for 7 days (total dose not to exceed 3.5 mg/kg for this week; the daily dose will be individualized to achieve the target weekly exposure of about 47.5 µg\*h/mL)
* Treatment period 2: ELX-02 1.0 mg/kg SC daily for 7 days (total dose not to exceed 7.0 mg/kg for this week; the daily dose will be individualized to achieve the target weekly exposure of about 95 µg\*h/mL)
* Treatment period 3: ELX-02 2.0 mg/kg SC daily for 14 days (total dose not to exceed 14 mg/kg for these two weeks; the daily dose will be individualized to achieve the target weekly exposure of about 190 µg\*h/mL)
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?: