Immunotherapy Clinical Trial
Every immunotherapy drug in use today began in a trial, and some patients still gain most from joining one. Most who look do not find a trial they qualify for, and many trials require months near the study site. This guide explains the phases, what modern phase 1 trials achieve, your rights, the warning signs of a fake trial, and how a search is done.
About This Department
Every immunotherapy drug in use today was once an experimental drug in a trial. Only about one cancer patient in twelve ever joins one.
The first trial of an anti-PD-1 antibody, in 296 patients whose cancers had stopped responding to everything else, produced lasting responses in a quarter of those with melanoma or kidney cancer. That drug is now standard. Trials offer patients access to treatments not available elsewhere, and they carry risks and uncertainties that deserve to be understood before anyone signs. Most patients who look for a trial do not find one they qualify for, and many trials require residence near the study site and fluency in the local language. Send us your diagnosis and treatment history, and a medical oncologist will tell you which trials fit, which are realistic from abroad, and what the standard alternative offers.
What an immunotherapy clinical trial is
A clinical trial is a study in which patients receive a treatment under a written protocol so that its effects are measured. Immunotherapy trials test drugs that act through the immune system, such as new checkpoint inhibitors, bispecific antibodies, cell therapies, cancer vaccines, or new combinations and schedules of drugs already approved.
Every trial is registered in a public database with a number, approved by an ethics committee and by the national regulator, and run under the principles of the Declaration of Helsinki. The study drug is supplied by the sponsor. The protocol fixes who can join, what they receive, how often they are seen and when treatment stops.
Should I look for a trial?
The answer depends on three things.
For some patients a trial is the best option. Others do better with a proven standard. The decision rests on what the standard options can do for your cancer at its present stage, on what the trial is testing, and on whether you can meet its practical demands, which for a patient living abroad are frequently the hardest part.
Why so few patients take part
A meta-analysis of 13 studies and 8,883 cancer patients traced what happens when trials are considered. No suitable trial was open at the patient's hospital 55.6 percent of the time. Where one was open, 21.5 percent of patients did not meet its criteria, and 14.8 percent chose not to join. In the end 8.1 percent enrolled, 15.9 percent in academic centers and 7.0 percent in community hospitals.
Availability and eligibility are the obstacles.
The main obstacles lie there, and a patient who wants a trial does best to look widely and early, before several lines of treatment have narrowed the options.
What do the phases mean for a patient?
Four kinds of trial
- Phase 1. The first use of a drug or combination in people. Small groups receive rising doses to find a safe dose. Most patients have exhausted standard options.
- Expansion cohorts. Once a dose is chosen, groups of patients with one cancer type receive it, and the response rate is measured. Many immunotherapy drugs reached patients this way.
- Phase 2. A larger group tests whether the drug works well enough in a given cancer to justify a definitive trial, sometimes against a control group.
- Phase 3. Patients are randomly assigned to the new treatment or to the current standard, and the trial decides whether the new one is better.
Before signing, ask the study team to explain six things.
- What the trial is testing, and what is known about the drug so far.
- What your standard treatment would be, and what it offers.
- Whether you could be assigned to the standard treatment alone.
- How often you must come to the study site and for how long, and whether any visits can happen at home, since this decides whether a patient from abroad can take part at all.
- Who pays for the drug, scans, travel and side-effect care.
- What happens on stopping early. And if the drug works, what happens when the trial ends.
What the numbers say about early-phase trials today
Early-phase immunotherapy has changed what patients can expect. The first trial of an anti-PD-1 antibody, published in 2012, treated 296 patients with advanced cancers. Responses occurred in 18 percent with lung cancer, 28 percent with melanoma and 27 percent with kidney cancer, and two thirds of the responses followed for a year or more lasted that long. Serious drug-related side effects occurred in 14 percent, and three patients died of lung inflammation.
Across 465 phase 1 trials of all kinds sponsored by the United States National Cancer Institute between 2000 and 2019, with 13,847 patients, the response rate rose from 9.6 percent in the early years to 18.0 percent in the most recent period, while treatment-related deaths stayed at 0.7 percent. Combination trials produced responses in 15.8 percent against 3.5 percent for single drugs. Only 3.4 percent of cancer drugs entering trials between 2000 and 2015 went on to approval, which is a reminder that most experimental drugs do not succeed.
- Untreated brain metastases.
- An autoimmune disease needing treatment, or an organ transplant, since releasing the immune system could turn it against the graft or the joints, gut or skin already under attack.
- Poor kidney, liver or bone marrow function on the blood tests.
- Too many or too few earlier lines of treatment, or an earlier drug of the same class.
- Inability to attend visits at the study site, or to speak the language in which the trial is run. Registry entries for some Turkish studies list fluency in Turkish among the entry criteria.
Your rights as a participant
The Declaration of Helsinki sets the ethical rules that every legitimate trial follows. Informed consent must be voluntary and in writing, after the aims, methods, sources of funding, risks and expected benefits have been explained. A participant may withdraw at any time without penalty. The interests of the individual take precedence over the interests of science. Placebo is acceptable only where no proven treatment exists or where its use does not add serious risk. Results must be made public, including negative ones.
In practice, that means you should never be asked to pay for the study drug, and you should always be told which hospital, ethics committee and registry number stand behind the study.
This table scrolls sideways on a narrow screen. Swipe or drag to see every column.
| Phase | Main question | Patients | What a participant receives | Chance of benefit |
|---|---|---|---|---|
| Phase 1 | Safety and dose | Tens | The new drug, at rising doses in most | Modest, response around 18 percent in recent trials, higher in combinations |
| Expansion cohort | Activity in one cancer | Tens to hundreds | The chosen dose | Depends on the cancer and the target |
| Phase 2 | Enough promise for a definitive trial | Dozens to hundreds | The new drug, sometimes against a control | Better defined, still uncertain |
| Phase 3 | Superiority over the standard | Hundreds to thousands | New drug or standard, by random assignment | At least the standard, with a chance of more |
Arranging trial access from abroad
Nobody sells trial places. No reputable hospital does. What we offer is a review of your file, a search of the public trial registries for studies you may qualify for, in Turkey and elsewhere, and a frank opinion on which are realistic given the visit schedule, the language and the distance. When a suitable trial exists, we pass your file to its investigators with your consent.
When none does, the review turns to the standard options, which for many cancers include approved immunotherapy drugs that were in trials only a few years ago.
Route decides the length of stay in Istanbul. A file review needs no travel. A consultation with tests takes three to five working days. A patient who joins a trial in Istanbul must expect to live near the study site for months, since visits are frequent and fixed. Flying between visits needs the study team's agreement.
Costs follow the same logic. In a legitimate trial the sponsor pays for the study drug and, in most, for the tests the protocol requires, while travel, accommodation and treatment outside the protocol may fall to the patient, and the information sheet should say which. The consultation and review are separate. Your own figure follows the free review of your file.
One coordinator from the international patients team is assigned from your first message and stays with you through the process. The team works in English, Arabic, French, Russian, Serbian, Romanian and Spanish and arranges interpreting in other languages on request. A trial's own documents may have to be read in the language the protocol specifies, which is one more reason to raise language at the very first contact with a study team and before the screening tests are done. An invitation letter naming the hospital and the treating oncologist is sent ten days before travel. The international patients office arranges the airport transfer and the hotel for you and a companion. Halal, vegetarian and diabetic meals are available, a prayer room is on site, and a request for a female doctor is passed to the department and met wherever the rota allows. Back home, the coordinator stays reachable on the same WhatsApp number, and the team checks new trial openings against your file on request.
Immunotherapy clinical trial FAQ
Is it safe to join an immunotherapy trial?
Do I have to pay to join a clinical trial?
Can a foreign patient join a trial in Turkey?
Could I receive a placebo?
What happens if the trial drug works?
Can my family come with me?
Informed decision making is crucial for patients before participating in phase 1 trials.Analysis of 465 National Cancer Institute phase 1 trials, The Lancet, 2022
References
- Topalian SL, Hodi FS, Brahmer JR, et al. Safety, activity, and immune correlates of anti-PD-1 antibody in cancer. New England Journal of Medicine. 2012;366(26):2443-2454.
- Chihara D, Lin R, Flowers CR, et al. Early drug development in solid tumours: analysis of National Cancer Institute-sponsored phase 1 trials. Lancet. 2022;400(10351):512-521.
- Unger JM, Vaidya R, Hershman DL, Minasian LM, Fleury ME. Systematic Review and Meta-Analysis of the Magnitude of Structural, Clinical, and Physician and Patient Barriers to Cancer Clinical Trial Participation. Journal of the National Cancer Institute. 2019;111(3):245-255.
- Wong CH, Siah KW, Lo AW. Estimation of clinical trial success rates and related parameters. Biostatistics. 2019;20(2):273-286.
- World Medical Association. World Medical Association Declaration of Helsinki: ethical principles for medical research involving human subjects. JAMA. 2013;310(20):2191-2194.
Editor's note
Written by the Biruni Hospital medical editorial team. Reviewed by Prof. Dr. Metin GÜDEN, Radiation Oncology.
Medically reviewed by
Prof. Dr. Metin GÜDEN
Radiation Oncology
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