NCT03428178: LHON

Efficacy Study of Gene Therapy for The Treatment of Acute LHON Onset Within Three Months

Status unknown NA Last updated 29 July 2020

What this trial tests

NA trial testing rAAV2-ND4 in Acute LHON in 120 participants. Status unknown.

Timeline

8 January 2018

Primary endpoint
6 December 2018

30 December 2020

Quick facts

Lead sponsor	Bin Li
Phase	NA
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	120
Start date	8 January 2018
Primary completion	6 December 2018
Estimated completion	30 December 2020
Sites	1 location across China

Drugs / interventions tested

• rAAV2-ND4 — full drug profile →

Conditions studied

• Acute LHON — all drugs for Acute LHON →
• Onset Within Three Months — all drugs for Onset Within Three Months →
• Onset Between 3 to 6 Months — all drugs for Onset Between 3 to 6 Months →
• Onset Between 6 to 12 Months — all drugs for Onset Between 6 to 12 Months →

Sponsor

Bin Li — full company profile →

Who can join

Adults 8 to 60, any sex, with Acute LHON or Onset Within Three Months. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Efficacy Study of Gene Therapy for The Treatment of Acute Leber's Hereditary Optic Neuropathy (LHON) onset within three months

Publications & conference data

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

1. Clinical trials in mitochondrial disorders, an update.
   Almannai M, El-Hattab AW, Ali M, Soler-Alfonso C, et al · · 2020 · cited 56× · PMID 33129691 · DOI 10.1016/j.ymgme.2020.10.002
2. Therapeutic Options in Hereditary Optic Neuropathies.
   Amore G, Romagnoli M, Carbonelli M, Barboni P, et al · · 2021 · cited 55× · PMID 33159657 · DOI 10.1007/s40265-020-01428-3
3. Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses.
   Ghoraba HH, Akhavanrezayat A, Karaca I, Yavari N, et al · · 2022 · cited 51× · PMID 35685379 · DOI 10.2147/opth.s364200
4. An overview of development in gene therapeutics in China.
   Wang D, Wang K, Cai Y. · · 2020 · cited 20× · PMID 32528163 · DOI 10.1038/s41434-020-0163-7
5. Curative gene therapies for rare diseases.
   Maldonado R, Jalil S, Wartiovaara K. · · 2021 · cited 19× · PMID 32803721 · DOI 10.1007/s12687-020-00480-6
6. Current and Future Landscape in Genetic Therapies for Leber Hereditary Optic Neuropathy.
   Shamsnajafabadi H, MacLaren RE, Cehajic-Kapetanovic J. · · 2023 · cited 13× · PMID 37566092 · DOI 10.3390/cells12152013
7. What are the characteristics and progression of visual field defects in patients with Leber hereditary optic neuropathy: a prospective single-centre study in China.
   Liu HL, Yuan JJ, Tian Z, Li X, et al · · 2019 · cited 8× · PMID 30878986 · DOI 10.1136/bmjopen-2018-025307
8. Three Cases of Leber's Hereditary Optic Neuropathy with Rapid Increase in Visual Acuity After Gene Therapy.
   Zhang Y, Yuan JJ, Liu HL, Tian Z, et al · · 2019 · cited 6× · PMID 31237206 · DOI 10.2174/1566523219666190618094505