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Spinal Muscular Atrophy (SMA)Phase 2SMANCT02876094

Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy

A Phase 2 study testing celecoxib in people with Spinal Muscular Atrophy (SMA), run by Hugh McMillan at 1 site in Canada.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Several factors make the use of celecoxib in human SMA patients appealing including: 1) low-dosing required for potential therapeutic effect (the corresponding dose in humans is much lower than that commonly used in adults and children with; 2) favourable side effect profile of this drug (particularly at the dosing… Read more about this study

Who is it for?
Ages 2 to 80 years
Conditions the study is for
Spinal Muscular Atrophy (SMA)
What is being tested?
celecoxib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
1 participants
Where
1 siteCanada
Study dates
29 Jan 2019to 6 Aug 2020
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 80 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Confirmed genetic diagnosis consistent with SMA that can include: SMN1 gene deletions, rearrangements and/or mutations
  • Sufficient clinical information enabling the patient to be classified as either SMA type II or III. (Patients with SMA type II are defined as having achieved the motor milestone of sitting independently for > 30 seconds but not having been able to stand or walk unsupported. Patients with SMA type III are defined as having achieved the motor milestone of standing or walking independently).
  • Confirmed genetic test result indicating number of SMN2 gene copies
  • Age > 2.0 years old at screening
  • Patients weighing at least 12 kg at screening
  • Stable dosing (for at least 3 months) of medications that may affect function of muscle, nerve and/or neuromuscular transmission or gene expression (including but not limited to: coenzyme Q10, creatine monohydrate, nutritional supplements, oral salbutamol, valproic acid, sodium phenylbutyrate, hydroxyurea)
  • Written informed consent obtained from patient and/or parents or legal guardians

Who cannot join

  • Clinical presentation and/or genetic testing that is not consistent with SMA type II or III
  • Inability or unwillingness to swallow celecoxib suspension
  • Major surgery (scoliosis repair, G-tube insertion) within past 3 months
  • Known hypersensitivity or allergy to celecoxib (including asthma, urticaria and/or other allergic symptoms resulting from prior celecoxib ingestion) or its excipients, or other NSAIDs (non-steroidal anti-inflammatory drugs) including ASA (Acetylsalicylic Acid)
  • Known hypersensitivity or allergy to Ora-Blend® or its excipients
  • Demonstrated allergic-type reaction to sulfonamides
Show all 13Show fewer
  • Celecoxib use within 2 weeks prior to screening visit
  • Known cardiac (ie. uncontrolled heart failure, cerebrovascular bleeding, hypertension requiring the use of anti-hypertensive medication), hepatic (i.e. severe liver impairment or active liver disease), gastrointestinal (i.e. inflammatory bowel disease; active gastric/duodenal/peptic ulcer disease; or active gastrointestinal bleeding), hematologic (ie. thrombocytopenia defined as platelets < 50,000 or hemophilia), respiratory or renal disease(i.e. severe renal impairment defined as creatinine clearance < 30 mL/min) wherein the use of NSAIDs is contraindicated as per Product Monograph dated 03 March 2015.
  • Concurrent use of medication contraindicated with Celecoxib use (including but not limited to, warfarin, fluconazole, lithium, hydrochlorothiazide)
  • Female who is pregnant or breast feeding
  • Female of child-bearing potential who is sexually active and unwilling or unable to use at least one form of highly effective and one effective method of birth control.
  • Patients participating in any pharmaceutical clinical trial (with active agent) that could impact with the results of this study
  • Inability or refusal to provide informed consent

Trial locations and contacts

1 site in 1 country.

Canada1 site

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H8L1

Other trials in Canada

About this study

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)

Several factors make the use of celecoxib in human SMA patients appealing including: 1) low-dosing required for potential therapeutic effect (the corresponding dose in humans is much lower than that commonly used in adults and children with; 2) favourable side effect profile of this drug (particularly at the dosing required); 3) the fact that celecoxib crosses the blood brain barrier and 4) demonstration of efficacy in a genetically and pathophysiologically faithful animal mode. The investigators therefore believe that celecoxib is a promising disease modifying therapy for SMA.

Read the detailed description

This is a pilot, open-label, dose-response study in patients with SMA type II or III. All patients will be treated at each dose of 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.

Being tested
celecoxib
Medical subject headings
Muscular Atrophy, Spinal

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • celecoxibDrug

    Also called CeleBREX

    dose-response

Study groups in NCT02876094
Study groupRoleWhat they receive
Open-labelExperimentalAll 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.
Receives: Drug: celecoxib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    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.

    Measured: baseline

Secondary outcomes (5)

  1. Safety Profile Measured by Adverse Event Frequency,Type and Severity

    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

    Measured: 4 weeks post

  2. Recruitment Plan Measured by Number of Potentially Eligible Subjects

    Assess understanding of recruitment barriers measured by the number of potentially eligible subjects and response to study recruitment phase.

    Measured: 4 weeks post

  3. Compliance Measured by Reported Protocol Deviations

    Assess adherence to treatment protocol measured by number of reported protocol deviations.

    Measured: 4 weeks post

  4. Eligibility Measured by Number of Screen Failures

    Assess appropriateness of eligibility criteria based on number of screen failures.

    Measured: 4 weeks post

  5. Delivery Time of Shipped Samples Assessed by Viability

    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.

    Measured: 4 weeks post

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Aug 2016 (estimated)
  2. Study start29 Jan 2019
  3. Primary completion6 Aug 2020
  4. Study completion6 Aug 2020

Why it stopped: Terminated: Study halted prematurely and will not resume; participants are no longer being examined or receiving intervention

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
1 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02876094
Sponsor's protocol ID
15/22E
Acronym
SMA
Status last verified
Oct 2020
First submitted
9 Aug 2016
Last updated
19 Oct 2020
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Results will be submitted for presentation at an international meeting and subsequently submitted for publication in a major international peer-reviewed medical journal.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing celecoxib in people with Spinal Muscular Atrophy (SMA), run by Hugh McMillan at 1 site in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 19 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Terminated: Study halted prematurely and will not resume; participants are no longer being examined or receiving intervention). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02876094.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2020.

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