A pilot study of the safety and tolerability of oral administration of PD L 506 and the subsequent diagnostic value of porphyrin concentration (coproporphyrin I / III, CP I / III) in urine in patients with the first occurrence of colorectal cancer and in healthy subjects.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Photonamic GmbH & Co. KG

Participant type

Patients, Healthy volunteers

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Diagnosis [E01]

Trial duration

12 Nov 2024 → ongoing

Decision date (initial)

2024-08-20

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

photonamic GmbH & Co. KG

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Diagnosis, Safety

To investigate the safety and tolerability of repeated oral administration of 300 mg PD L 506 in subjects with the first occurrence of CRC (Cohort 1) and of single oral administration of 300 mg PD L 506 in subjects without CRC (Healthy Subjects, Cohort 2).

Secondary objectives 1

1. To investigate the feasibility and diagnostic value of a CRC screening in urine using spectrofluorometry for analyses of CP I / III after oral administration of 300 mg PD L 506.

Conditions and MedDRA coding

Colorectal Carcinoma

Study design 1 period

Regulatory references

Plan to share IPD

No

IPD plan description

N/A

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Signed and dated informed consent after comprehensive information.
2. Male or female ≥18 years of age.
3. CRC patients (cohort 1 only) with newly diagnosed, non-metastatic, resectable colorectal cancer (< UICC IV) must meet the following criteria: Confirmed histopathological diagnosis and anti-cancer therapy for CRC not started.
4. Healthy subject (cohort 2 only) meeting the following criteria: Has undergone colonoscopy that extended to the ileocecal valve and was negative for malignancy within 5 years before screening visit
5. Willingness of women of childbearing/ reproductive potential and male subjects to use highly effective contraception (Pearl index <1) consistently and correctly for the duration of the clinical trial period through the last follow-up visit required by the protocol.
6. Willingness of pre-menopausal female subjects who are of childbearing potential to obtain a negative serum pregnancy test max. 72 h prior to first application of PD L 506 and during the conduct of the clinical trial as appropriate.

Exclusion criteria 13

1. Subject is not willing to give or sign the informed consent and/or not able to comply with scheduled visits, treatment plan, laboratory tests, and other clinical trial procedures.
2. Clinically significant renal or hepatic impairment including severe chronic conditions/co-morbidities.
3. Any current diseases with poor prognosis, e.g., severe coronary heart disease, heart failure (NYHA III/IV), severe and poorly controlled diabetes, immune deficiency, residual deficits after stroke, severe mental retardation or other serious concomitant systemic disorders incompatible with the clinical trial (at the discretion of the investigator).
4. Simultaneous participation in another clinical trial or participation in another clinical trial in the 30 days directly preceding treatment or within 5 plasma half-life’s of the preceding trial drug, whatever is longer.
5. Pregnancy or breastfeeding.
6. Metastatic colorectal cancer (UICC IV).
7. Current diagnosis of any other tumour (malignant or benign).
8. Suffers from Ulcerative Colitis or Crohn’s Disease.
9. Intercurrent severe infection.
10. Previous allogenic bone marrow transplantation or insufficient bone marrow function.
11. Any psychological, cognitive, familial, sociological, or geographical condition that, in the investigator’s opinion, compromises the patient’s ability to understand the patient information, to give informed consent or to comply with the clinical trial protocol.
12. Known hypersensitivity to porphyrins or the active substance 5-ALA HCl.
13. Known diagnosis of acute or chronic types of porphyria.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Number of Adverse Drug Reactions (ADRs) graded according to CTCAE v5.0.
2. Number of discontinuations due to ADRs.

Secondary endpoints 3

1. Change of CP I / III levels in CRC patient urine samples taken before PD L 506 administration to CP I / III levels in CRC patient urine samples collected 5 ± 1.0 hours after PD L 506 administration at the same day, at Visit 1 (before NAT, for NAT patients only) and Visit 2 (before tumour resection) and Visits 3, 4 and 6 (after NAT / tumour removal).
2. Change of CP I / III levels in CRC patient urine samples collected 5 ± 1.0 hours after PD L 506 administration before NAT / tumour removal (Visit 1 for NAT patients and Visit 2 for other patients) to after NAT / tumour removal (Visits 3, 4 and 6).
3. Change of CP I / III levels in healthy subject urine samples taken before PD L 506 administration to CP I / III levels in healthy subject urine samples collected 5 ± 1.0 hours after PD L 506 administration at the same day (Visit 2).

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Photonamic GmbH & Co. KG

Sponsor organisation

Photonamic GmbH & Co. KG

Address

Eggerstedter Weg 12

City

Pinneberg

Postcode

25421

Country

Germany

Scientific contact point

Organisation

Photonamic GmbH & Co. KG

Contact name

Clinical Project Management

Public contact point

Organisation

Photonamic GmbH & Co. KG

Contact name

Clinical Project Management

Third parties 2

Locations

1 EU/EEA country · 2 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

Documents 11 files

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

Application history

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