VEGF-D gene therapy in severe coronary artery disease. Phase II clinical trial (ReGenHeart).

2024-512255-20-00 Protocol RGH201 Therapeutic exploratory (Phase II) Ended

End 31 May 2024 · Status Ended · 2 EU/EEA countries · 2 sites · Protocol RGH201

Overview

Sponsor-declared trial summary

Phase Therapeutic exploratory (Phase II)
Status Ended
Participants planned 45
Countries 2
Sites 2

Clinical development and proof of principle testing of new regenerative VEGF-D therapy for cost- effective treatment of refractory angina. A phase II randomized, double-blinded, placebo-controlled study.

The purpose of the study is to evaluate the safety and efficacy of catheter mediated AdVEGF-D regenerative gene transfer in patients with refractory angina to whom revascularization cannot be performed.

Key facts

Sponsor
Pohjois-Savon hyvinvointialue
Participant type
Patients
Age range
18-64 years, 65+ years
Gender
Male and Female
Therapeutic area
Diseases [C] - Cardiovascular Diseases [C14]
Trial duration
completed 31 May 2024
Decision date (initial)
2024-05-31
Transition trial
Yes
Low-intervention
No
Rare-disease indication
No
Vulnerable population
No
Funding sources
The study is funded by EU Horizon 2020 program (ReGenHeart; grant no 731532)

External identifiers

EU CT number
2024-512255-20-00
EudraCT number
2017-000789-31
ClinicalTrials.gov
NCT03039751

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

The purpose of the study is to evaluate the safety and efficacy of
catheter mediated AdVEGF-D regenerative gene transfer in patients with refractory angina to whom revascularization cannot be performed.

Secondary objectives 1

  1. The study also investigates the effect of VEGF-D-therapy on myocardial perfusion, patients' symptoms, quality of life and safety of treatment.

Conditions and MedDRA coding

Clinical development and proof of principle testing of new regenerative VEGF-D therapy for cost- effective treatment of refractory angina. A phase II randomized, double-blinded, placebo-controlled study.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

  1. Informed consent signed
  2. Age > 30 but ≤ 85 years
  3. Significant angina pectoris (CCS 2-3) despite of optimal medication
  4. Significant stenosis (≥ 60%) in coronary angiography (≤ 12 months)
  5. Contraindication to CABG or PCI due to diffuse or distal stenosis, chronic total occlusion, vessels with difficult anatomy, stenosis with severe calcifications and stenosis in small vessels (<2.5 mm)
  6. Angina pectoris or equivalent symptoms in the 6-minute walking exercise test
  7. Left ventricle wall ≥ 8 mm detected by transthoracic echocardiography (treatment area)

Exclusion criteria 16

  1. Women of childbearing potential
  2. Diabetes mellitus with severe complications such as proliferative diabetic retinopathy or nephropathy with renal insufficiency (see below)
  3. Clinically significant anemia (hemoglobin count < 120 mg/l in male, < 110 mg/l in female; hematocrit < 0.36)
  4. Leukopenia (b-leukocyte count < 3.0x109/l), leukocytosie count > 12.0x109/l) or thrombocytopenia (b-thrombocyte count < 100x109/l)
  5. Renal insufficiency (P-creatinine > 160 mg/l)
  6. Liver insufficiency (P-alanine aminotransferase or P-alkaline phosphatase over 2 x normal)
  7. Hematuria of unknown origin
  8. Severe hypertension (systolic blood pressure > 200 mmHg or diastolic blood pressure > 110 mmHg) or significant hypotension (systolic blood pressure < 90 mmHg)
  9. Significant obesity (BMI > 35)
  10. Acute infection
  11. Immunosuppressive medication
  12. Significant impairment of left ventricular function (EF < 25%)
  13. Symptomatic congestive heart failure (NYHA 3-4)
  14. Hemodynamically significant (grade 3-4/4) aortic or mitral regurgitation or other heart disease needing surgery
  15. Recent (< 3 months) acute coronary syndrome or myocardial infarction (elevated CK-MB or cardiac troponin), PCI, CABG or TIA/stroke
  16. Current or suspected malignancy

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

  1. Improvement of functional capacity using 6-minute walking test
  2. Improvement of symptoms assessed by Canadian Cardiovascular Society (CCS) class

Secondary endpoints 3

  1. Improvement of functional capacity using 6-minute walking test
  2. Improvement of symptoms assessed by Canadian Cardiovascular Society (CCS) class
  3. Increase in myocardial perfusion

Investigational products

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

Test 1

AdsVEGF-D Delta N Delta C

PRD11250995 · Product

Active substance
Adenovirus Vector Serotype 5 Containing the Vascular Endothelial Growth Factor D Isoform (Preprocessed Short Form)
Substance synonyms
Ad.VEGF-D Delta N Delta C
Pharmaceutical form
INJECTION
Route of administration
INTRACARDIAC USE
Max daily dose
2 Other
Max total dose
2 Other
Max treatment duration
1 Day(s)
Authorisation status
Not Authorised
MA holder
POHJOIS-SAVON HYVINVOINTIALUE
Paediatric formulation
No
Orphan designation
No

Placebo 1

Placebo to AdsVEGF-D Delta N Delta C test product

N/A · Product

Other product name
N/A
Pharmaceutical form
N/A
ATC code
N/A — N/A
Marketing authorisation
N/A
MA holder
N/A
MA country
Iceland
Paediatric formulation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Pohjois-Savon hyvinvointialue

Sponsor organisation
Pohjois-Savon hyvinvointialue
Address
Puijonlaaksontie 2, P. O. Box 1711 P. O. Box 1711
City
Kuopio
Postcode
70211
Country
Finland

Scientific contact point

Organisation
Pohjois-Savon hyvinvointialue
Contact name
Aleksi Leikas

Public contact point

Organisation
Pohjois-Savon hyvinvointialue
Contact name
Aleksi Leikas

Locations

2 EU/EEA countries · 2 investigational sites

By country

CountryMS statusPlanned subjectsSites
Denmark Ended 17 1
Finland Ended 22 1
Rest of world
United Kingdom
6

Investigational sites

Denmark

1 site · Ended
Rigshospitalet
Heart Centre, Blegdamsvej 9, 2100, Copenhagen Oe

Finland

1 site · Ended
Pohjois-Savon hyvinvointialue
Heart Center, Puijonlaaksontie 2, P. O. Box 1711, Kuopio

Application history

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

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2024-04-22 Finland Acceptable
2024-05-29
2024-05-29