NCT03558958

Safety and Efficacy of P-188 NF in DMD Patients

Terminated Phase 2 Last updated 11 January 2023

What this trial tests

Phase 2 trial testing P-188 NF in Duchenne Muscular Dystrophy in 2 participants. Terminated before completion.

Timeline

8 August 2018

Primary endpoint
1 September 2021

1 September 2021

Quick facts

Lead sponsor	Phrixus Pharmaceuticals, Inc.
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	2
Start date	8 August 2018
Primary completion	1 September 2021
Estimated completion	1 September 2021
Sites	1 location across United States

Drugs / interventions tested

• P-188 NF — full drug profile →

Conditions studied

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

Sponsor

Phrixus Pharmaceuticals, Inc. — full company profile →

Who can join

Adults 12 to 25, male only, with Duchenne Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is an open-label study to evaluate the safety, tolerability and efficacy of daily, subcutaneous dosing with P-188 NF (Carmeseal-MD™) in non-ambulatory boys with Duchenne Muscular Dystrophy (DMD). This study will determine if continuous treatment with Carmeseal-MD™ can maintain or improve pulmonary function, and skeletal and cardiac muscle function, compared to baseline, in boys 12-25 years of age.

Publications & conference data

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

1. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies.
   Bez Batti Angulski A, Hosny N, Cohen H, Martin AA, et al · · 2023 · cited 109× · PMID 37435300 · DOI 10.3389/fphys.2023.1183101
2. Muscle membrane integrity in Duchenne muscular dystrophy: recent advances in copolymer-based muscle membrane stabilizers.
   Houang EM, Sham YY, Bates FS, Metzger JM. · · 2018 · cited 53× · PMID 30305165 · DOI 10.1186/s13395-018-0177-7
3. Membrane Stabilization by Modified Steroid Offers a Potential Therapy for Muscular Dystrophy Due to Dysferlin Deficit.
   Sreetama SC, Chandra G, Van der Meulen JH, Ahmad MM, et al · · 2018 · cited 46× · PMID 30166241 · DOI 10.1016/j.ymthe.2018.07.021
4. 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
5. Exosome-mediated improvement in membrane integrity and muscle function in dystrophic mice.
   Leng L, Dong X, Gao X, Ran N, et al · · 2021 · cited 25× · PMID 33333294 · DOI 10.1016/j.ymthe.2020.12.018
6. Molecular mechanisms and therapeutic strategies for neuromuscular diseases.
   Zambon AA, Falzone YM, Bolino A, Previtali SC. · · 2024 · cited 7× · PMID 38678519 · DOI 10.1007/s00018-024-05229-9
7. Molecular homing and retention of muscle membrane stabilizing copolymers by non-invasive optical imaging in vivo.
   Batti Angulski AB, Cohen H, Kim M, Hahn D, et al · · 2023 · cited 4× · PMID 36654800 · DOI 10.1016/j.omtm.2022.12.005
8. Leveraging Plasma Membrane Repair Therapeutics for Treating Neurodegenerative Diseases.
   Bulgart HR, Goncalves I, Weisleder N. · · 2023 · cited 3× · PMID 37371130 · DOI 10.3390/cells12121660