NCT03605069

A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene

Terminated Phase 1, PHASE2 Last updated 25 August 2021

What this trial tests

Phase 1, PHASE2 trial testing QR-313 in Epidermolysis Bullosa Dystrophica, Recessive in 2 participants. Terminated before completion.

Timeline

2 July 2018

Primary endpoint
17 December 2018

17 December 2018

Quick facts

Lead sponsor	Phoenicis Therapeutics
Phase	Phase 1, PHASE2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	2
Start date	2 July 2018
Primary completion	17 December 2018
Estimated completion	17 December 2018
Sites	6 locations across France, United States, Spain

Drugs / interventions tested

• QR-313 — full drug profile →
• Placebo

Conditions studied

• Epidermolysis Bullosa Dystrophica, Recessive — all drugs for Epidermolysis Bullosa Dystrophica, Recessive →
• Epidermolysis Bullosa Dystrophica, Dominant — all drugs for Epidermolysis Bullosa Dystrophica, Dominant →

Sponsor

Phoenicis Therapeutics — full company profile →

Who can join

4 and older, any sex, with Epidermolysis Bullosa Dystrophica, Recessive or Epidermolysis Bullosa Dystrophica, Dominant. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

A double-blind, randomized, intra-subject placebo-controlled, multicenter, multiple dose study, evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with confirmed DDEB or RDEB diagnosis with one or more pathogenic mutations in exon 73 in the COL7A1 gene.

Publications & conference data

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

1. Antisense technology: A review.
   Crooke ST, Liang XH, Baker BF, Crooke RM. · · 2021 · cited 237× · PMID 33600796 · DOI 10.1016/j.jbc.2021.100416
2. Epidermolysis Bullosa-Associated Squamous Cell Carcinoma: From Pathogenesis to Therapeutic Perspectives.
   Condorelli AG, Dellambra E, Logli E, Zambruno G, et al · · 2019 · cited 77× · PMID 31739489 · DOI 10.3390/ijms20225707
3. Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines.
   Liu M, Wang Y, Zhang Y, Hu D, et al · · 2025 · cited 62× · PMID 40059188 · DOI 10.1038/s41392-024-02112-8
4. Molecular Therapeutics in Development for Epidermolysis Bullosa: Update 2020.
   Has C, South A, Uitto J. · · 2020 · cited 43× · PMID 32328988 · DOI 10.1007/s40291-020-00466-7
5. Innovations in the Treatment of Dystrophic Epidermolysis Bullosa (DEB): Current Landscape and Prospects.
   Hou PC, Del Agua N, Lwin SM, Hsu CK, et al · · 2023 · cited 39× · PMID 37337559 · DOI 10.2147/tcrm.s386923
6. Gene Delivery to the Skin - How Far Have We Come?
   Ain QU, Campos EVR, Huynh A, Witzigmann D, et al · · 2021 · cited 27× · PMID 32873394 · DOI 10.1016/j.tibtech.2020.07.012
7. Newer Treatment Modalities in Epidermolysis Bullosa.
   Bruckner-Tuderman L. · · 2019 · cited 26× · PMID 31149565 · DOI 10.4103/idoj.idoj_287_18
8. Charge-Conversion Strategies for Nucleic Acid Delivery.
   Dutta K, Das R, Medeiros J, Kanjilal P, et al · · 2021 · cited 25× · PMID 35832306 · DOI 10.1002/adfm.202011103