NCT01978366

An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01

Terminated Phase 2 Last updated 31 August 2020

What this trial tests

Phase 2 trial testing HT-100 in Duchenne Muscular Dystrophy in 17 participants. Terminated before completion.

Timeline

1 October 2013

Primary endpoint
30 April 2016

30 April 2016

Quick facts

Lead sponsor	Processa Pharmaceuticals
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	17
Start date	1 October 2013
Primary completion	30 April 2016
Estimated completion	30 April 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

Processa Pharmaceuticals — full company profile →

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.

• Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD.
  Time frame: Months 2, 4, 6, 7
  * Target Safety profile by review of adverse events (AEs) * Physical examination findings * Clinical laboratory test results * Other diagnostic testing

Sponsor's own description

This study is designed to provide 6-months continuous dosing with the study medication, called HT-100, on participants who successfully completed the predecessor study (HALO-DMD-01). The main purpose of this study is to assess chronic safety, tolerability, pharmacodynamic activity (testing the drug's effect on DMD) and population pharmacokinetics (measuring how much drug is in the bloodstream) in participants with a broad spectrum of Duchenne muscular dystrophy (DMD).

Publications & conference data

3 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. 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
3. 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