Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy (SMA)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02876094|
Recruitment Status : Not yet recruiting
First Posted : August 23, 2016
Last Update Posted : August 2, 2017
|Condition or disease||Intervention/treatment||Phase|
|Spinal Muscular Atrophy (SMA)||Drug: celecoxib||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot, Open-Label, Dose Response Study Investigating the Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy (SMA)|
|Anticipated Study Start Date :||August 2017|
|Estimated Primary Completion Date :||July 2018|
|Estimated Study Completion Date :||July 2018|
U.S. FDA Resources
All patients will be treated at each dose of oral once daily celecoxib (40, 80 and 160 mcg/kg) for a period of two weeks, for a total of 6 weeks (42 days) of treatment.
Other Name: CeleBREX
- low-dose oral celecoxib administered to patients with SMA type II and III is associated with an increase in the levels of peripheral leukocyte SMN protein compared to baseline [ Time Frame: baseline ]1) Investigate change in peripheral leukocyte SMN protein levels from baseline at each dose (40 mcg/kg, 80 mcg/kg, and 160 mcg/kg) of celecoxib.
- Safety Profile Measured by Adverse Event Frequency,Type and Severity [ Time Frame: 4 weeks post ]1) Determine safety profile as measured by number, type and severity of adverse events reported following administration of low dose celecoxib in patients with type II and III SMA
- Recruitment Plan Measured by Number of Potentially Eligible Subjects [ Time Frame: 4 weeks post ]Assess understanding of recruitment barriers measured by the number of potentially eligible subjects and response to study recruitment phase.
- Compliance Measured by Reported Protocol Deviations [ Time Frame: 4 weeks post ]Assess adherence to treatment protocol measured by number of reported protocol deviations.
- Eligibility Measured by Number of Screen Failures [ Time Frame: 4 weeks post ]Assess appropriateness of eligibility criteria based on number of screen failures.
- Delivery Time of Shipped Samples Assessed by Viability [ Time Frame: 4 weeks post ]Assess feasibility of shipping laboratory samples to outside centre for analysis. This will be reported based on the time to deliver and the resulting viability of the received samples by either pre or post testing or both if appropriate.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02876094
|Contact: Hugh McMillan, MD||613-737-7600 ext email@example.com|
|Contact: Angie Tuttle, BioTech,CCRP||613-737-7600 ext firstname.lastname@example.org|
|Children's Hospital of Eastern Ontario||Not yet recruiting|
|Ottawa, Ontario, Canada, K1H8L1|
|Contact: Hugh J McMillan, MD 613-737-7600 ext 1605 HMcMillan@cheo.on.ca|
|Principal Investigator:||Hugh McMillan, MD||Children's Hospital of Eastern Ontario, Research Institute|