NCT02525302

HT-100 Long-term Safety and Pharmacodynamics in Patients With DMD Who Have Completed Protocols HALO-DMD-01 and HALO-DMD-02

Terminated Phase 2 Last updated 8 March 2019

What this trial tests

Phase 2 trial testing HT-100 in Duchenne Muscular Dystrophy in 10 participants. Terminated before completion.

Timeline

1 May 2015

Primary endpoint
30 December 2016

30 December 2016

Quick facts

Lead sponsor	Akashi Therapeutics
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	1 May 2015
Primary completion	30 December 2016
Estimated completion	30 December 2016
Sites	5 locations across United States

Drugs / interventions tested

• HT-100 — full drug profile →

Conditions studied

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

Sponsor

Akashi Therapeutics

Who can join

Adults 6 to 20, male only, with Duchenne 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.

• Number of adverse events by severity and relationship
  Time frame: Every 6 months from enrollment for up to 3 years
• Dose reduction or modification due to upper GI or other adverse events
  Time frame: Every 6 months from enrollment for up to 3 years
• Trial discontinuations due to upper GI or other AEs
  Time frame: Every 6 months from enrollment for up to 3 years
• Vital signs (Number of subjects with clinically significant changes)
  Time frame: Every 6 months from enrollment for up to 3 years
  Number of subjects with clinically significant changes
• Laboratory values (Number of subjects with clinically significant changes)
  Time frame: Every 6 months from enrollment for up to 3 years
  Number of subjects with clinically significant changes.
• Electrocardiograms
  Time frame: Every 6 months from enrollment for up to 3 years
  Number of subjects with clinically significant changes in QT interval

Sponsor's own description

This study, HALO-DMD-03, is a follow-on study to HALO-DMD-01 and HALO-DMD-02, and allows continued open-label access to HT-100 for subjects who have completed these studies. HALO-DMD-03 will provide safety and strength and function data on continuous long-term dosing. Data from this study will be used to inform the safety, tolerability, and dose selection for a future trial of HT-100 in boys with Duchenne Muscular Dystrophy (DMD).

Publications & conference data

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

1. Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials.
   Shimizu-Motohashi Y, Miyatake S, Komaki H, Takeda S, et al · · 2016 · cited 68× · PMID 27398133
2. Current Pharmacological Strategies for Duchenne Muscular Dystrophy.
   Yao S, Chen Z, Yu Y, Zhang N, et al · · 2021 · cited 44× · PMID 34490244 · DOI 10.3389/fcell.2021.689533
3. MYC-family protein overexpression and prominent nucleolar formation represent prognostic indicators and potential therapeutic targets for aggressive high-MKI neuroblastomas: a report from the children's oncology group.
   Niemas-Teshiba R, Matsuno R, Wang LL, Tang XX, et al · · 2018 · cited 32× · PMID 29464082 · DOI 10.18632/oncotarget.23740
4. Anti-Inflammatory and General Glucocorticoid Physiology in Skeletal Muscles Affected by Duchenne Muscular Dystrophy: Exploration of Steroid-Sparing Agents.
   Herbelet S, Rodenbach A, Paepe B, De Bleecker JL. · · 2020 · cited 25× · PMID 32605223 · DOI 10.3390/ijms21134596
5. Control of fibrosis with enhanced safety via asymmetric inhibition of prolyl-tRNA synthetase 1.
   Yoon I, Kim S, Cho M, You KA, et al · · 2023 · cited 19× · PMID 37212275 · DOI 10.15252/emmm.202216940
6. Neo-epitope Peptides as Biomarkers of Disease Progression for Muscular Dystrophies and Other Myopathies.
   Arvanitidis A, Henriksen K, Karsdal MA, Nedergaard A. · · 2016 · cited 7× · PMID 27854226 · DOI 10.3233/jnd-160150
7. Strategy for drug repurposing in fibroadipogenic replacement during muscle wasting: application to duchenne muscular dystrophy.
   Matthews I, Mehra P, Suárez-Calvet X, Piñol-Jurado P, et al · · 2025 · cited 1× · PMID 40206397 · DOI 10.3389/fcell.2025.1505697