A phase II, double-blind, randomised, placebo-controlled trial to evaluate the efficacy and tolerability of a rectal treatment with budesonide suppositories versus placebo for amelioration of acute radiation proctitis symptoms

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Dr. Falk Pharma GmbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male

Therapeutic area

Diseases [C] - Digestive System Diseases [C06], Diseases [C] - Male Urogenital Diseases [C12]

Trial duration

16 Apr 2026 → ongoing

Decision date (initial)

2026-03-22

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Dr. FALK PHARMA GMBH

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy

To prove the superiority of an 8-week rectal treatment with 4 mg budesonide suppositories once daily versus placebo suppositories in improving CTCAE grading for proctitis in the treatment of
RAP

Secondary objectives 6

1. To further assess the improvement of CTCAE gradings for proctitis of an up to 8-week rectal treatment with 4 mg budesonide suppositories once daily versus placebo suppositories in the treatment of RAP
2. To assess the improvement of CTCAE gradings of the lower GI composite of an up to 8-week rectal treatment with 4 mg budesonide suppositories once daily versus placebo suppositories in the treatment of RAP
3. To assess the participant’s perception of symptom severity and improvement of an 8-week rectal treatment with 4 mg budesonide suppositories once daily versus placebo suppositories in the treatment of RAP
4. To assess bowel-related function, general treatment response, and quality of life of an 8-week rectal treatment with 4 mg budesonide suppositories once daily versus placebo suppositories in the treatment of RAP
5. To assess tolerability life of an 8-week rectal treatment with 4 mg budesonide suppositories once daily versus placebo suppositories in the treatment of RAP
6. To assess participant-reported symptomatic manifestation of an 8-week rectal treatment with 4 mg budesonide suppositories once daily versus placebo suppositories in the treatment of RAP

Conditions and MedDRA coding

Acute radiation proctitis symptoms in male patients aged 18 years or older with prostate carcinoma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Age and Sex: - Male patients (i.e., sex assigned at birth inclusive all gender identities) aged at least 18 years, at the time of signing the informed consent
2. Type of Participant and Disease Characteristics: - Diagnosis of prostate carcinoma (primary or biochemical recurrence); - Patients with Eastern Cooperative Oncology Group (ECOG) performance status ≤2 or Karnofsky performance status 70% (refer to Table 12. Karnofsky performance status); - Local radiotherapy by external beam radiation planned; - Conventional external beam RT with 60-80 Gy complete dose in 20-40 fractions over 4-8 weeks
3. Patients with Eastern Cooperative Oncology Group (ECOG) performance status ≤2 or Karnofsky performance status 70%
4. Local radiotherapy by external beam radiation planned.
5. Conventional external beam RT with 60-80 Gy complete dose in 20-40 fractions over 4-8 weeks.
6. Signed informed consent as described in Section 10.1.3 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
7. CTCAE grading for proctitis and diarrhea ≥ grade 1 for at least 3 days.
8. Sufficient compliance in ePRO as assessed during screening period (defined as at least 3 ePRO entries per week).

Exclusion criteria 24

1. Treatment by brachytherapy
2. Severe renal impairment (GFR ≤ 29 ml /min /1.73 m2 at Baseline) calculated by CKD EPI 2009
3. Patients with morning serum cortisol serum < 4 µg/dL
4. Active infections, clinically apparent uncontrolled hypertension, diabetes mellitus (including familiar predisposition), active peptic ulcer, osteoporosis, glaucoma, cataract, or other conditions where corticoids may have side effects if careful medical monitoring is not ensured in the opinion of the investigator
5. Immunocompromised patients (e.g., due to human immunodeficiency virus infection) (respective diagnosis in medical history).
6. Diagnosis of chickenpox, herpes zoster, or measles within 3 months prior to Baseline
7. Portal hypertension or liver cirrhosis
8. Abnormal hepatic parameters (ALT, AST or AP > 2.5 x upper limit of normal) or known significant functional disorder of the liver
9. Having received live vaccine(s) within the last 14 days prior to Baseline
10. Concomitant medication with systemic and/or topical corticosteroids, other immunosuppressants, or medication that affect peristalsis and/or the intestinal mucosa (e.g., antibiotics, loperamide, opioids, laxatives)
11. Participation in another clinical study and having received an IMP within 30 days prior to Visit 0 (Screening visit) or within 5 half-lives of IMP, whichever is longer
12. Crohn’s disease, indeterminate colitis, ischemic colitis, ulcerative colitis, microscopic colitis (i.e., collagenous colitis or lymphocytic colitis)
13. Grade III internal hemorrhoids
14. Perianal dermatitis
15. Significant bacterial, amoebic, fungal, or viral infections of the gut
16. History of any RT in the area of the pelvis and/or the lower abdomen
17. Active colorectal cancer or a history of colorectal cancer
18. Active malignancy other than prostate cancer or treatment with antineoplastic drugs during the last 5 years. Patients with a history of cancer (other than prostate cancer) and at least 5 years of uneventful follow-up and no signs of recurrence may be eligible
19. Phenylketonuria
20. Known intolerance/hypersensitivity/resistance to the IMP and excipients or drugs of similar chemical structure or pharmacological profile.
21. Known to be or suspected of being unable to comply with the clinical study protocol (e.g., no permanent address, history of drug abuse, known to be non-compliant, or presenting an unstable psychiatric history).
22. Legal incapacity and/or other circumstances rendering the patient unable to understand the nature, scope and possible impact of the clinical study.
23. Patients in custody by juridical or official order or patients who are institutionalized because of legal or regulatory order.
24. Patient, who is a member of the staff of the study center, staff of the sponsor or clinical research organization, the investigator him-/herself or close relatives of the investigator.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Time to first improvement in CTCAE grading for proctitis is defined as the number of days from Baseline to the first occurrence of a lower CTCAE grading compared to Baseline, sustained for at least three consecutive days

Secondary endpoints 32

1. Percentage of participants with reduction in CTCAE grading for proctitis by one and by two score point(s) from Baseline to Visit 2 and Baseline to Visit 4
2. Time to first improvement in CTCAE grading for diarrhea after Baseline, where improvement is defined as a lower CTCAE grading compared to Baseline for at least three consecutive days
3. Percentage of participants with reduction in CTCAE grading for diarrhea by one and by two score point(s) from Baseline to Visit 2 and Baseline to Visit 4
4. Time to first improvement in CTCAE grading for rectal pain after Baseline, where improvement is defined as a lower CTCAE grading compared to Baseline for at least three consecutive days
5. Percentage of participants with reduction in CTCAE grading for rectal pain by one and by two score point(s) from Baseline to Visit 2 and Baseline to Visit 4
6. Time to first improvement in each CTCAE grading for further terms of the lower GI composite (fecal incontinence, lower GI hemorrhage, GI pain and other GIdisorder), where improvement is defined as a lower CTCAE grading compared to Baseline for at least three consecutive days
7. Percentage of participants with reduction in each CTCAE gradings for further terms of the lower GI composite (fecal incontinence, lower GI hemorrhage, GI pain and other GI-disorder) by one and by two score point(s) from Baseline to Visit 2 and Baseline to Visit 4
8. Course and change from Baseline in the SHS-GI sum score and sub-scores at Visit 2, 4, and 5
9. Course and change from Baseline in the PGI-S at Visit 2, 4, and 5
10. PGI-C at Visit 2 and 4
11. Course and change from Baseline in the EPIC Bowel Domain Score at Visit 2, 4, and 5
12. Therapeutic success at Visit 4, defined as complete relief or marked improvement of symptoms according to the PGA
13. Therapeutic benefit at Visit 4, defined as at least a slight improvement according to the PGA
14. Assessment of efficacy by investigator and participant at Visit 4
15. Percentage of participants who remained symptom-free for at least ten consecutive days at Visit 4
16. Percentage of participants who used rescue medication at Visit 4.
17. Duration of rescue medication use at Visit 4
18. Assessment of tolerability by investigator and participant at Visit 4
19. Average daily bowel movement frequency within the treatment period
20. Percentage of days within the treatment period with pain during bowel movements
21. Percentage of days within the treatment period with mild pain during bowel movements
22. Percentage of days within the treatment period with moderate pain during bowel movements
23. Percentage of days within the treatment period with severe pain during bowel movements
24. Percentage of days within the treatment period with bloody stool
25. Percentage of days within the treatment period with mucous stool
26. Percentage of days within the treatment period with uncontrolled bowel movements
27. Percentage of days within the treatment period during which no pads were used due to uncontrolled bowel movement
28. Percentage of days within the treatment period during which pads were used sometimes due to uncontrolled bowl movement
29. Percentage of days within the treatment period during which pads were used most of the time due to uncontrolled bowl movement
30. Percentage of days within the treatment period during which symptoms interfered with daily life
31. Percentage of days within the treatment period during which symptoms interfered with daily functioning to the extent that self-care was impossible
32. Percentage of days within the treatment period during which medical intervention was required due to symptoms

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Dr. Falk Pharma GmbH

Sponsor organisation

Dr. Falk Pharma GmbH

Address

Leinenweberstrasse 5, Hochdorf Hochdorf

City

Freiburg Im Breisgau

Postcode

79108

Country

Germany

Scientific contact point

Organisation

Dr. Falk Pharma GmbH

Contact name

Claudia Stosnach

Public contact point

Organisation

Dr. Falk Pharma GmbH

Contact name

Claudia Stosnach

Third parties 4

Locations

2 EU/EEA countries · 6 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 31 files

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

Application history

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