Trial · NCT02015481

Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

NCT02015481, Phase 2, Completed, Bioblast Pharma Ltd.; results first posted 2017-08-23

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients. Trials, record NCT02015481. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02015481

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
NCT number: NCT02015481
Status: Completed
Phases: Phase 2
Enrollment: 25 participants, actual
Lead sponsor: Bioblast Pharma Ltd.
Conditions: Oculopharyngeal Muscular Dystrophy
Interventions: Cabaletta
First posted: 2013-12-19
Last updated: 2017-10-17
Results first posted: 2017-08-23

Source ClinicalTrials.gov

Registered outcomes4

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Safety Lab Evaluations	24 weeks	Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .
Secondary	Drinking Test Score	24 weeks	Change from baseline in ice water drinking time, in seconds, at week 24. Times greater than 8 seconds to complete the drinking test are considered abnormal.
Secondary	Videofluoroscopy (VFS) Score	24 Weeks	Penetration aspiration score results assessed by VFS comparing baseline, prior to treatment, to week 24. This is an 8 point scale. The higher the number the greater the risk of aspiration. The result reported is the difference from the baseline scores.
Secondary	SWAL-QOL, Swallowing Quality of Life Questionnaire	28 weeks	Summary of Quality of Life in Swallowing Disorders total symptom score results over time, change from baseline at weeks 12 and 24 This is a 100 point scale. The higher the number the better the quality of life.

Source ClinicalTrials.gov

Outcomes4

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Safety Lab Evaluations	24 weeks	24 patients were available for analysis at week 24. Not all patients had all labs performed. Missing data accounts for the differing number of patients with specific lab values.	percentage change from baseline	MEAN
SECONDARY	Drinking Test Score	24 weeks	22 patients had data available for analysis at week 24	percentage change from baseline	MEAN
SECONDARY	Videofluoroscopy (VFS) Score	24 Weeks	11 patients had valid baseline data and week 24 data	Points on an 8 point scale	MEAN
SECONDARY	SWAL-QOL, Swallowing Quality of Life Questionnaire	28 weeks		percentage change from baseline	MEAN

Source ClinicalTrials.gov

Results by group17

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Safety Lab Evaluations	Cabaletta 30gr.	White Blood Cell Count	-4.0	Standard Deviation 16.69	percentage change from baseline
Safety Lab Evaluations	Cabaletta 30gr.	Red Blood Cell Count	0.1	Standard Deviation 0.42	percentage change from baseline
Safety Lab Evaluations	Cabaletta 30gr.	Hemoglobin	0.2	Standard Deviation 7.98	percentage change from baseline
Safety Lab Evaluations	Cabaletta 30gr.	Hematocrit	1.1	Standard Deviation 9.28	percentage change from baseline
Safety Lab Evaluations	Cabaletta 30gr.	Platelets	-0.5	Standard Deviation 13.42	percentage change from baseline
Safety Lab Evaluations	Cabaletta 30gr.	Alanine Aminotransferase	-0.8	Standard Deviation 34.59	percentage change from baseline
Safety Lab Evaluations	Cabaletta 30gr.	Aspartate Aminotransferase	-5.7	Standard Deviation 30.93	percentage change from baseline
Safety Lab Evaluations	Cabaletta 30gr.	Creatinine phosphokinase	6.8	Standard Deviation 37.12	percentage change from baseline
Safety Lab Evaluations	Cabaletta 30gr.	Gamma-Glutamyl Transferase	9.3	Standard Deviation 28.73	percentage change from baseline
Safety Lab Evaluations	Cabaletta 30gr.	Total Bilirubin	37.2	Standard Deviation 46.02	percentage change from baseline

1 to 10 of 17Source ClinicalTrials.gov

See all 17 results by group

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Cabaletta 30gr	25
Overall Study	COMPLETED	Cabaletta 30gr	24
Overall Study	NOT COMPLETED	Cabaletta 30gr	1

Source ClinicalTrials.gov

Adverse events27

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Abdominal Pain	Gastrointestinal disorders	Cabaletta 30gr	3	25	3
other	Constipation	Gastrointestinal disorders	Cabaletta 30gr	2	25	2
other	Dysphagia	Gastrointestinal disorders	Cabaletta 30gr	2	25	2
other	Toothache	Gastrointestinal disorders	Cabaletta 30gr	2	25	2
other	Administrative Site Bruise	General disorders	Cabaletta 30gr	4	25	5
other	Asthenia	General disorders	Cabaletta 30gr	2	25	2
other	Fatigue	General disorders	Cabaletta 30gr	4	25	5
other	Influenza Like Illness	General disorders	Cabaletta 30gr	3	25	3
other	Pyrexia	General disorders	Cabaletta 30gr	2	25	2
other	Bacteriuria	Infections and infestations	Cabaletta 30gr	2	25	3

1 to 10 of 27Source ClinicalTrials.gov

See all 27 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Cabaletta 30gr	1	25
Total, other adverse events	Cabaletta 30gr	25	25
Total, serious adverse events	Cabaletta 30gr	2	25

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT02015481

GET https://conductscience.org/api/trials/v1/NCT02015481/events?page=2

API reference For AI assistants