NCT01847573

A Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-100 in Patients With Duchenne Muscular Dystrophy

Terminated Phase 1/Phase 2 Last updated 31 August 2020

What this trial tests

Phase 1/Phase 2 trial testing HT-100 in Duchenne Muscular Dystrophy in 17 participants. Terminated before completion.

Timeline

1 May 2013

Primary endpoint
30 March 2016

30 March 2016

Quick facts

Lead sponsor	Processa Pharmaceuticals
Phase	Phase 1/Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	17
Start date	1 May 2013
Primary completion	30 March 2016
Estimated completion	30 March 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 administering single and multiple ascending doses of HT-100 in DMD boys
  Time frame: 1 week
  Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing

Sponsor's own description

The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD). The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with DMD. In this study, pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream will also be taken.

Publications & conference data

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

1. Understanding the process of fibrosis in Duchenne muscular dystrophy.
   Kharraz Y, Guerra J, Pessina P, Serrano AL, et al · · 2014 · cited 192× · PMID 24877152 · DOI 10.1155/2014/965631
2. 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
3. Therapeutic advances in muscular dystrophy.
   Leung DG, Wagner KR. · · 2013 · cited 54× · PMID 23939629 · DOI 10.1002/ana.23989
4. Progress and prospects of gene therapy clinical trials for the muscular dystrophies.
   Bengtsson NE, Seto JT, Hall JK, Chamberlain JS, et al · · 2016 · cited 48× · PMID 26450518 · DOI 10.1093/hmg/ddv420
5. Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy.
   Markati T, De Waele L, Schara-Schmidt U, Servais L. · · 2021 · cited 30× · PMID 34790118 · DOI 10.3389/fphar.2021.735912
6. 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
7. Fibrogenesis in <i>LAMA2</i>-Related Muscular Dystrophy Is a Central Tenet of Disease Etiology.
   Accorsi A, Cramer ML, Girgenrath M. · · 2020 · cited 24× · PMID 32116541 · DOI 10.3389/fnmol.2020.00003