NCT02015481: HOPEMD

Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients

Completed Phase 2 Results posted Last updated 15 September 2017

What this trial tests

Phase 2 trial testing Cabaletta in Oculopharyngeal Muscular Dystrophy in 25 participants. Completed in 1 April 2016.

Timeline

1 February 2014

Primary endpoint
1 April 2016

1 April 2016

Quick facts

Lead sponsor	Bioblast Pharma Ltd.
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	25
Start date	1 February 2014
Primary completion	1 April 2016
Estimated completion	1 April 2016
Sites	4 locations across United States, Canada, Israel

Drugs / interventions tested

• Cabaletta — full drug profile →

Conditions studied

• Oculopharyngeal Muscular Dystrophy — all drugs for Oculopharyngeal Muscular Dystrophy →

Sponsor

Bioblast Pharma Ltd. — full company profile →

Who can join

Adults 18 to 80, any sex, with Oculopharyngeal Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Safety Lab Evaluations
  Time frame: 24 weeks
  Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .

Sponsor's own description

The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Trehalose upregulates progranulin expression in human and mouse models of GRN haploinsufficiency: a novel therapeutic lead to treat frontotemporal dementia.
   Holler CJ, Taylor G, McEachin ZT, Deng Q, et al · · 2016 · cited 88× · PMID 27341800 · DOI 10.1186/s13024-016-0114-3
2. PABPN1 gene therapy for oculopharyngeal muscular dystrophy.
   Malerba A, Klein P, Bachtarzi H, Jarmin SA, et al · · 2017 · cited 53× · PMID 28361972 · DOI 10.1038/ncomms14848
3. Trehalose alleviates the phenotype of Machado-Joseph disease mouse models.
   Santana MM, Paixão S, Cunha-Santos J, Silva TP, et al · · 2020 · cited 28× · PMID 32272938 · DOI 10.1186/s12967-020-02302-2
4. Trehalose restores functional autophagy suppressed by high glucose.
   Xu C, Chen X, Sheng WB, Yang P. · · 2019 · cited 24× · PMID 30769031 · DOI 10.1016/j.reprotox.2019.02.005
5. The increased activity of a transcription factor inhibits autophagy in diabetic embryopathy.
   Xu C, Chen X, Reece EA, Lu W, et al · · 2019 · cited 14× · PMID 30312583 · DOI 10.1016/j.ajog.2018.10.001
6. RNA-Based Therapy Utilizing Oculopharyngeal Muscular Dystrophy Transcript Knockdown and Replacement.
   Abu-Baker A, Kharma N, Perreault J, Grant A, et al · · 2019 · cited 12× · PMID 30831428 · DOI 10.1016/j.omtn.2019.02.003