Treatment of adult Wilson's Disease patients with gene therapy

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Vivet Therapeutics

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trial duration

21 Jun 2022 → 8 Oct 2024

Decision date (initial)

2024-04-18

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2023-509998-23-00

EudraCT number

2020-000963-22

WHO UTN

U1111-1301-8786

ClinicalTrials.gov

NCT04537377

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacodynamic, Efficacy, Dose response

Assessing the safety and tolerability of single ascending doses of VTX-801 administered intravenously (IV) to adult patients with Wilson's Disease prior to and following background WD therapy withdrawal

Secondary objectives 3

1. Exploring VTX-801 pharmacodynamics and efficacy
2. Assessing humoral and cellular immune responses to VTX-801
3. Providing data to support VTX-801 dose level selection

Conditions and MedDRA coding

Wilson's Disease

Regulatory references

Scientific advice from competent authorities

Paul-Ehrlich-Institut, European Medicines Agency, Medicines And Healthcare Products Regulatory Agency

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Male or female aged 18-65 years old (inclusive)
2. Confirmed diagnosis of WD
3. Treated for WD according to international recommendations with no current evidence for inadequate treatment
4. Stable WD for ≥ 1 year, defined as: (i) No significant change in neurologic examination and in status of mood disorder, and (ii) stable laboratory parameters used to assess copper metabolism including 24- hour urinary copper, free serum copper such as NCC or CuEXC (when available), as well as liver enzymes, hemoglobin, and white blood cell count

Exclusion criteria 12

1. ALT level ≥ 2 x ULN that is not readily explained by extrinsic factors (e.g., strenuous exercise, medication use)
2. Total bilirubin > 1.5x ULN in the absence of proven Gilbert’s syndrome; in case of Gilbert’s syndrome, direct bilirubin > ULN.
3. Platelet count < 120,000/µL.
4. Any signs of liver cirrhosis decompensation, including gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrollment visit.
5. Patient has moderate or severe renal impairment or patient has nephritis or nephrotic syndrome.
6. Any history or current evidence of HIV-1, HIV-2, HTLV 1 or HTLV-2 infection.
7. Any history or current evidence of hepatitis B infection
8. Any history of hepatitis C infection, unless previous viral RNA assays in two samples, collected at least 6 months apart, are negative
9. Positive QuantiFERON®-TB Gold tuberculosis test result
10. Any concomitant disorder/condition - including hepatic disorder - or treatment possibly interfering with the conduct or evaluation of the study, according to the Investigator.
11. Pregnancy or breastfeeding.
12. Body Mass Index ≥ 35 kg/m2.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Safety and tolerability profile, including treatment-emergent adverse events (TEAE), clinical examination, changes in laboratory parameters, vital signs, ECG, brain and abdominal MRI

Secondary endpoints 6

1. Free serum Cu
2. Total serum Cu
3. 24-hour urinary Cu
4. Serum ceruloplasmin activity (enzymatic assay)
5. VTX-801 Responder status
6. Immune response to VTX-801

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Vivet Therapeutics

Sponsor organisation

Vivet Therapeutics

Address

80 Boulevard Haussmann

City

Paris

Postcode

75008

Country

France

Scientific contact point

Organisation

Vivet Therapeutics

Contact name

Info Vivet

Public contact point

Organisation

Vivet Therapeutics

Contact name

Info Vivet

Third parties 12

Locations

2 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Layperson summary Annex V

Documents 1 file

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications