NCT03167437

An Open-Label, Proof of Consent Study of Vorinostat for the Treatment of Mdoerate-to-Severe Crohn s Disease and Maintenance Therapy With Ustekinumab

Recruiting now Phase 1, PHASE2 Last updated 1 April 2026

What this trial tests

Phase 1, PHASE2 trial testing Vorinostat in Crohn's Disease in 35 participants. Currently enrolling.

Timeline

30 October 2017

Primary endpoint
30 June 2026

30 June 2026

Quick facts

Lead sponsor	National Institute of Allergy and Infectious Diseases (NIAID)
Phase	Phase 1, PHASE2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	35
Start date	30 October 2017
Primary completion	30 June 2026
Estimated completion	30 June 2026
Sites	1 location across United States

Drugs / interventions tested

• Vorinostat (VORINOSTAT) — full drug profile →
• Ustekinumab (ustekinumab) — full drug profile →

Conditions studied

• Crohn's Disease — all drugs for Crohn's Disease →

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Who can join

Adults 18 to 65, any sex, with Crohn's Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Background:

Crohn s disease (CD) is an inflammatory bowel disease. It causes inflammation of the gut. Symptoms may include diarrhea, abdominal pain, fatigue, weight loss and malnutrition. CD has no cure, but symptoms can sometimes be controlled with medicine. Researchers want to see if it is safe to treat CD with the medicine vorinostat. It is thought that vorinostat may reduce the inflammation process of CD. This may then help to relieve symptoms of CD. Participants who respond to Vorinostat will be invited to an extension phase of treatment with Vorinostat and possibly a maintenance treatment using Ustekinumab.

Objectives:

To see if vorinostat is safe for people with moderate-to-severe CD. To see if it is safe for people with moderate-to-sever CD to receive maintenance therapy using Ustekinumab after successful treatment of Vorinostat.

Eligibility:

Adults 18-65 with moderate-to-severe CD that medicine is not controlling.

Design:

Phase I is screening. It may last 120 days. Participants will have:

Physical exam

Medical history

Tests of blood, urine, and stool samples

Heart test

Questionnaires

Tuberculosis skin test

They may have a colonoscopy and lymphapheresis collection. These will be explained in a separate consent.

They will keep a diary of symptoms.

Phase II is treatment using Vorinostat. It will take 12-13 weeks. Participants will take the study drug by mouth twice daily for 12 weeks. They will get a weekly phone call to talk about how the drug makes them feel. They will have blood taken regularly. Every 4 weeks, they will have a check-up that will repeat some screening tests.

Phase III extension treatment of Vorinostat for an additional 6 months for those who respond to vorinostat and it is safe for them to continue treatment. Participants will continue to receive weekly calls to talk about how the drug makes them feel. They will have blood taken regularly. Every 3 months, they will have a check-up that will repeat some screening tests.

Phase IV: is maintenance therapy for 2 years with Ustekinumab. Participants will receive a one time loading dose of ustekinumab, and then will receive the approved maintenance dose once every 8 weeks, at which time they will return to the NIH Clinical Center for evaluation. The participant will get a phone call 3 days after each dose and again 2 weeks later to see how the drug makes them feel. After two years of receiving treatment with ustekinumab the participant will have an end of study visit, where some of the screening tests, including a colonoscopy, will be repeated.

Publications & conference data

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

1. Treatment strategies for glucose-6-phosphate dehydrogenase deficiency: past and future perspectives.
   Garcia AA, Koperniku A, Ferreira JCB, Mochly-Rosen D. · · 2021 · cited 39× · PMID 34389161 · DOI 10.1016/j.tips.2021.07.002
2. The FOXP3<sup>+</sup> Pro-Inflammatory T Cell: A Potential Therapeutic Target in Crohn's Disease.
   Kosinsky RL, Gonzalez MM, Saul D, Barros LL, et al · · 2024 · cited 27× · PMID 38211712 · DOI 10.1053/j.gastro.2024.01.007
3. Unlocking the potential of targeting histone-modifying enzymes for treating IBD and CRC.
   Liang B, Wang Y, Xu J, Shao Y, et al · · 2023 · cited 16× · PMID 37697409 · DOI 10.1186/s13148-023-01562-1
4. Marine-Derived Secondary Metabolites as Promising Epigenetic Bio-Compounds for Anticancer Therapy.
   Conte M, Fontana E, Nebbioso A, Altucci L. · · 2020 · cited 14× · PMID 33396307 · DOI 10.3390/md19010015
5. Advancing therapeutic frontiers: a pipeline of novel drugs for luminal and perianal Crohn's disease management.
   Bertin L, Crepaldi M, Zanconato M, Lorenzon G, et al · · 2024 · cited 10× · PMID 39711916 · DOI 10.1177/17562848241303651
6. Role of Histone Modifications in Kidney Fibrosis.
   Pan S, Yuan T, Xia Y, Yu W, et al · · 2024 · cited 8× · PMID 38929505 · DOI 10.3390/medicina60060888
7. Paradigm Shift in Inflammatory Bowel Disease Management: Precision Medicine, Artificial Intelligence, and Emerging Therapies.
   Caballero Mateos AM, Cañadas de la Fuente GA, Gros B. · · 2025 · cited 7× · PMID 40095460 · DOI 10.3390/jcm14051536
8. Novel candidate drugs in anti-tumor necrosis factor refractory Crohn's diseases: in silico study for drug repositioning.
   Kwak MS, Lee HH, Cha JM, Shin HP, et al · · 2020 · cited 6× · PMID 32612148 · DOI 10.1038/s41598-020-67801-0