Intractable PainPhase 1NCT00804154
Resiniferatoxin to Treat Severe Pain Associated With Advanced Cancer
A Phase 1 study testing Intrathecal Resiniferatoxin in people with Intractable Pain and Palliative Care, run by National Institute of Dental and Craniofacial Research (NIDCR) at 1 site in United States.
Trial summary
This study will examine the safety of giving the experimental drug, resiniferatoxin (RTX), to treat severe pain in patients with advanced cancer. RTX is a chemical extracted from a cactus-like plant. It is similar to capsaicin, the active ingredient in hot pepper. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Intractable Pain, Palliative Care
- What is being tested?
- Intrathecal Resiniferatoxin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Aug 2009to 14 Jul 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject inclusion criteria are based on inadequate control of pain despite best efforts, including appropriate use of analgesic medications. To be enrolled in the study, subjects must meet all of the following criteria:
- Age 18 years or older.
- Clinical and histological diagnosis of cancer with disease that has not adequately responded to standard therapies. A pathology report documenting malignancy is required.
- Subject not currently seeking or receiving potentially curative therapies for cancer (e.g., chemotherapy or immunotherapy). Curative cancer therapy may be sought after the Day 15 clinic visit, and palliative anti-tumor therapy is allowed as long as the subject was established on that therapy prior to enrollment (see exclusion criterion 6).
- Mean daily worst pain NRS score of greater than or equal to 6 for pain at or below the T6 dermatome (level of the chest) that is associated with a malignant disease. The mean score must be derived from recordings on at least 4 of 7 consecutive days within 3 weeks preceding treatment.
- Alternative methods of pain control are not sufficiently effective, not indicated, not tolerated, and/or refused by the subject, as determined by the Pain and Palliative Care Service (PPCS). Alternative methods of pain control include, but are not limited to, the following:
Show all 32Show fewer
- -Opioids (all routes of administration including neuraxial infusions)
- -Adjuvant pain medications such as antidepressants, corticosteroids, local anesthetics, and antiseizure medications
- -Procedures such as catheter or implantable pump placement for delivery of analgesic medication
- Prior neurolytic interventions (including intercostal, superior hypogastric, or celiac plexus blocks)
- Complementary medicine approaches
- Transcutaneous electric nerve stimulation
- Radiation therapy
- Reasonable expectation that the subject will be able to complete the study through the 30-day follow-up.
- Medical clearance from referring physician consisting of a statement indicating an adequate recovery period from other previous trials/medication.
- Formal review of the subject s medical records and written approval for his/her inclusion in
- the study by 3 separate persons:
- Principal Investigator (PI) or an Associate Investigator (AI)
- Medical oncologist or oncologic surgeon.
- A member of the PPCS at the NIH or institution s equivalent at other sites.
- 10. International normalized ratio (INR; from prothrombin time [PT]) < 1.5 and partial thromboplastin time (PTT) less than or equal to the upper limit of the reference range. The INR and PTT may be corrected (e.g., by administration of blood products, vitamin K, etc.), provided a repeat blood draw confirms that the values meet this inclusion criterion.
- 10. Platelet count greater than or equal to 50,000/mm^3. Platelets will be transfused as necessary to raise the platelet count to greater than or equal to 100,000/mm^3 prior to dosing.
- 11. Ability to stop any anticoagulant (e.g., Coumadin) and antiplatelet therapies (e.g., aspirin) before and during IT catheter placement according to accepted medical guidelines.1
- 12. Ability and willingness to undergo an eye examination.
- 13. Ability to read, speak, and understand English, and willingness to complete the study tools and forms.
- 14. For women of childbearing potential and men with partners of childbearing potential, the ability and willingness to use an effective method of contraception during the study. Effective methods of birth control include:
- hormonal contraception (birth control pills, injected hormones, or vaginal ring),
- intrauterine device,
- barrier methods (condom or diaphragm) combined with spermicide, or
- surgical sterilization (hysterectomy, tubal ligation, or vasectomy).
- 15. Availability of a responsible adult to assist with activities of daily living as needed for the subject through the Day 15 visit.
- 16. Ability to assign a Durable Power of Attorney (DPA) for research and medical care at NIH
Who cannot join
- Subjects will be excluded from the study if they meet any of the following criteria:
- Primary pain source from anatomical regions at T5 dermatome or above.
- Pain due to causes other than cancer or its treatment that is moderate to severe in intensity.
- Anatomic abnormality or pathology of the spinal cord and/or IT space on magnetic resonance imaging (MRI) that could increase the risk of adverse effects of catheter placement or interfere with CSF flow.
- Evidence of advanced brain pathology or elevated intracranial pressure as determined by symptoms, history, physical examination (including neurological and eye examination), and/or MRI.
- Presence of an IT shunt device (e.g., ventriculo-peritoneal and ventriculo-atrial shunts).
Show all 20Show fewer
- Anticipating initiation of palliative anti-tumor therapy or significant changes to current palliative anti-tumor therapy before completion of the Day 15 visit.
- Documented allergy to chili peppers or capsaicin (e.g., hives, wheal).
- Contraindication to MRI or MRI contrast.
- Female subjects who are pregnant or lactating.
- Clinically significant disorder or condition that might interfere with study participation or greatly increase safety risk to the subject, as judged by a study investigator.
- Planned use of another investigational agent, therapy, or device within 30 days after dosing.
- Have a history of heart failure or unexplained fainting (syncope).
- Have an EKG abnormality in which the baseline QTc interval exceeds 450 milliseconds.
- Have a known family history of long QT syndrome.
- Have abnormal electrolyte levels (i.e., low potassium) that cannot be corrected.
- Have urinary retention that does not resolve with medical or surgical treatment.
- Have skin ulceration that does not resolve with medical or surgical treatment.
- Have a history of seizure activity in the previous month.
- Have experienced symptoms of opioid toxicity in the past month (i.e. myoclonus, seizures, and/or hallucinations)
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: A Phase I Study of the Intrathecal Administration of Resiniferatoxin for Treating Severe Refractory Pain Associated With Advanced Cancer
This study will examine the safety of giving the experimental drug, resiniferatoxin (RTX), to treat severe pain in patients with advanced cancer. RTX is a chemical extracted from a cactus-like plant. It is similar to capsaicin, the active ingredient in hot pepper. RTX has relieved pain and reduced the need for pain medication in several animal experiments. It works by destroying nerves that transmit pain information.
People at least 18 years of age with severe pain from advanced cancer at or below the level of the chest that cannot be controlled with standard treatments may be eligible for this study. Participants undergo the following procedures:
Read more
Pretreatment Visit
Before beginning treatment with RTX, patients give a medical history and undergo a physical examination that includes:
- Electrocardiogram (EKG)
- Blood draw
- Urinalysis
- Neurological examinations
- Peak expiratory flow rate (PEFR)
- Eye examination
- MRI
- Urology assessment
- Pregnancy test, when appropriate
- Questionnaires to collect information on health, personality, mood, pain levels and symptoms. 2-Day Hospitalization
Patients are hospitalized for 2 days for RTX injection and monitoring, as follows:
- RTX injection: RTX is injected in the operating room under general anesthesia. It is given through a catheter placed in the patient s spine. The catheter is also used to obtain samples of cerebrospinal fluid (CSF) the clear fluid that bathes the spinal cord. The fluid is examined to assess drug effects and side effects, chemical changes in the content of the CSF associated with RTX, and how RTX is handled by the body.
- Post-injection monitoring, including:
- Surveys about symptoms such as pain or weakness
- Neurological examinations
- Blood and CSF sampling
- EKG
- AEs
Outpatient followup
- Vitals
- Blood draw, Urinalysis, neurological and sensory testing, EKG on days 7, 14 and 30 after the injection
- MRI scans of the head and back, Urology assessment and PEFR on day 15 after the injection
- Eye examination
- Follow-up phone calls monthly for 6 months
Read the detailed description
Pain continues to be a major problem in patients with advanced cancer. Resiniferatoxin (RTX), a potent member of the family of drugs that includes capsaicin, selectively and irreversibly destroys the neurons (or their axons) transmitting chronic pain sensation. Intrathecal injection of RTX in several animal species has demonstrated a high level of safety, specificity, and efficacy in treating severe pain. This first-in-human, dose-escalation study is investigating the intrathecal administration of RTX in cancer patients with severe pain.
PRIMARY OBJECTIVE:
To investigate the safety and efficacy of RTX administered intrathecally in subjects with severe refractory pain associated with advanced cancer.
STUDY POPULATION:
Up to 45 subjects will be accrued. Eligible subjects will be greater than or equal to 18 years of age, have a clinical and histological diagnosis of advanced malignancy, and have severe pain due to malignancy that is at or below the level of the chest and not adequately relieved by other pain control therapies.
DESIGN:
This is a single site, non-randomized, open-label, dose-escalation study using a modified Fibonacci scheme. The starting dose of RTX was 13 micrograms given as a 2 mL injection via an intra-spinal catheter over approximately 30 seconds followed by a 1 mL flush. Six subjects were dosed at this level and 3 were dosed at the 26 microgram dose level at the same volume of injectate, flush and injection time. Under the study design, RTX doses were to be increased in progressively smaller percentage increments with each dose-escalation to occur in sequential groups of 3 subjects until 1 escalation above the effective dose in 100% of subjects (ED100), completion of the 100 microgram dose level, or establishment of the maximum tolerated dose (MTD), whichever occurs first. The total duration of study participation for any subject will be up to 7 months.
The amended RTX injection technique reduced the injection volume and increased the injection time to reduce the spread of RTX to above the T6 (sixth thoracic) vertebral level. The present technique is a 1 mL injection over 2 minutes (0.5 mL/minute) given via infusion pump, followed by flushing of the IT catheter with the minimum volume of sterile, preservative-free saline necessary to clear the internal volume of the catheter used for the injection. Three patients were treated at the new starting dose of 13 micrograms with the new injection technique. The next 3-patient cohort will receive a dose of 26 micrograms. The subsequent subject dose tier will receive a dose of 44 micrograms consistent with the protocol s dose-escalation algorithm. Administering this dose will require an infusion volume of 1.76 mL of the present 25 micrograms/mL RTX formulation.
OUTCOME MEASURES:
The primary study outcome is the ED100, the MTD, or the maximum dose administered, whichever is achieved first during dose-escalation. The primary pain variable for determining the ED100 is the daily worst pain intensity score averaged over a 4 to 7-day period during the 3 weeks before RTX dosing and during Days 8 through 14 after dosing. The numerical rating scale (NRS), administered verbally during a daily telephone interview, will be the primary pain assessment instrument. For a given subject, the treatment will be considered effective if the subject experiences a greater than or equal to 50% reduction in the mean daily worst pain score assessed by NRS (evaluated on Study Day 15). We may also consider RTX treatment to be successful if there is greater than or equal to 50% reduction in opiate intake, measured by morphine milligram equivalents (MME) per day, even if pain levels are unchanged after RTX treatment.
Secondary outcome measures will be other surveys of pain, including an assessment of worst daily pain by the visual analog scale, and assessments of function and quality of life.
Safety assessments will include hematology; serum clinical chemistry tests; cerebrospinal fluid examinations; physical, neurological, and eye examinations; reporting of adverse events; electrocardiograms; and findings on magnetic resonance imaging of the spine and brain.
- Conditions
- Intractable Pain, Palliative Care
- Being tested
- Intrathecal Resiniferatoxin
- Keywords
- Vanilloid, Capsaicin, Cancer Pain, Nerve Block
- Medical subject headings
- Pain, Intractable, Cancer Pain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intrathecal ResiniferatoxinDrug
phase I, single-site, non-randomized, open-label, dose-escalation study to determine the safety and efficacy of IT RTX in subjects with severe refractory pain due to advanced malignancy
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | advanced cancer patients with pain Receives: Drug: Intrathecal Resiniferatoxin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Investigate the safety and efficacy of RTX administered intrathecally in subjects with severe refractory pain associated with advanced cancer along with ED100, the MTD, or the maximum dose administered, whichever is achieved first during dose es...
Dose escalation decisions are based on both effectiveness (as assessed by mean worst pain on the NRS) and DLT. The dose escalation population will include all subjects who are dosed, complete the NRS, and have the DLT assessments from baseline through Day 15. Subjects who are not evaluable for dose escalation will be replaced.
Measured: Day 7, Day 15, Day 68, Day 188
Secondary outcomes (1)
Secondary outcome measures will be other surveys of pain, including an assessment of worst daily pain by the visual analog scale, and assessments of function and quality of life.
The secondary outcome variables will be summarized over time by dose cohort as appropriate for the outcome measure. Absolute and percentage change in the NRS and VAS pain scores between the pre- and post-RTX dosing assessments (baseline period and study Days 8 through 14, respectively) will also be summarized.
Measured: Day 7, Day 15, Day 68, Day 188
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2008 (estimated)
- Study start14 Aug 2009
- Primary completion14 Jul 2026
- Study completion14 Jul 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Intractable PainStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- Sponsor type
- US National Institutes of Health
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), Sorrento Therapeutics, Inc.
- Responsible party
- Sponsor
- Investigators
- John D Heiss, M.D., Principal investigator, National Institute of Neurological Disorders and Stroke (NINDS)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00804154
- Sponsor's protocol ID
- 090039
- Other IDs
- 09-N-0039
- Status last verified
- 28 Oct 2026
- First submitted
- 5 Dec 2008
- Last updated
- 30 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: We do plan to share IPD. we will share all IPD that results in a publication on a public repository, as required by most journals. the data will be de-identified and anonymized.
Publications
Daut RL, Cleeland CS. The prevalence and severity of pain in cancer. Cancer. 1982 Nov 1;50(9):1913-8. doi: 10.1002/1097-0142(19821101)50:93.0.co;2-r.
PubMed 7116316Cleeland CS, Gonin R, Hatfield AK, Edmonson JH, Blum RH, Stewart JA, Pandya KJ. Pain and its treatment in outpatients with metastatic cancer. N Engl J Med. 1994 Mar 3;330(9):592-6. doi: 10.1056/NEJM199403033300902.
PubMed 7508092Oster MW, Vizel M, Turgeon LR. Pain of terminal cancer patients. Arch Intern Med. 1978 Dec;138(12):1801-2.
PubMed 718345Mannes AJ, Heiss JD, Berger A, Alewine CC, Butman JA, Hughes MS, Rabbee N, Hayes C, Williams TS, Sapio MR, Iadarola MJ. Treatment of Intractable Cancer Pain with Resiniferatoxin - An Interim Study. NEJM Evid. 2025 Jun;4(6):EVIDoa2400423. doi: 10.1056/EVIDoa2400423. Epub 2025 May 27.
PubMed 40423401
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of the Intrathecal Administration of Resiniferatoxin for Treating Severe Refractory Pain Associated With Advanced Cancer
- ClinicalTrials.gov ID
- NCT00804154
- Acronym / sponsor ID
- 090039
- Conditions
- Intractable Pain, Palliative Care
- Keywords
- Vanilloid, Capsaicin, Cancer Pain, Nerve Block
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)US National Institutes of Health
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), Sorrento Therapeutics, Inc.
- Study start
- 14 Aug 2009 (actual)
- Primary completion
- 14 Jul 2026 (actual)
- Study completion
- 14 Jul 2026 (actual)
- First posted
- 8 Dec 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will examine the safety of giving the experimental drug, resiniferatoxin (RTX), to treat severe pain in patients with advanced cancer. RTX is a chemical extracted from a cactus-like plant. It is similar to capsaicin, the active ingredient in hot pepper. RTX has relieved pain and reduced the need for pain medication in several animal experiments. It works by destroying nerves that transmit pain information.
People at least 18 years of age with severe pain from advanced cancer at or below the level of the chest that cannot be controlled with standard treatments may be eligible for this study. Participants undergo the following procedures:
Pretreatment Visit
Before beginning treatment with RTX, patients give a medical history and undergo a physical examination that includes:
- Electrocardiogram (EKG)
- Blood draw
- Urinalysis
- Neurological examinations
- Peak expiratory flow rate (PEFR)
- Eye examination
- MRI
- Urology assessment
- Pregnancy test, when appropriate
- Questionnaires to collect information on health, personality, mood, pain levels and symptoms. 2-Day Hospitalization
Patients are hospitalized for 2 days for RTX injection and monitoring, as follows:
- RTX injection: RTX is injected in the operating room under general anesthesia. It is given through a catheter placed in the patient s spine. The catheter is also used to obtain samples of cerebrospinal fluid (CSF) the clear fluid that bathes the spinal cord. The fluid is examined to assess drug effects and side effects, chemical changes in the content of the CSF associated with RTX, and how RTX is handled by the body.
- Post-injection monitoring, including:
- Surveys about symptoms such as pain or weakness
- Neurological examinations
- Blood and CSF sampling
- EKG
- AEs
Outpatient followup
- Vitals
- Blood draw, Urinalysis, neurological and sensory testing, EKG on days 7, 14 and 30 after the injection
- MRI scans of the head and back, Urology assessment and PEFR on day 15 after the injection
- Eye examination
- Follow-up phone calls monthly for 6 months
Detailed description
Pain continues to be a major problem in patients with advanced cancer. Resiniferatoxin (RTX), a potent member of the family of drugs that includes capsaicin, selectively and irreversibly destroys the neurons (or their axons) transmitting chronic pain sensation. Intrathecal injection of RTX in several animal species has demonstrated a high level of safety, specificity, and efficacy in treating severe pain. This first-in-human, dose-escalation study is investigating the intrathecal administration of RTX in cancer patients with severe pain.
PRIMARY OBJECTIVE:
To investigate the safety and efficacy of RTX administered intrathecally in subjects with severe refractory pain associated with advanced cancer.
STUDY POPULATION:
Up to 45 subjects will be accrued. Eligible subjects will be greater than or equal to 18 years of age, have a clinical and histological diagnosis of advanced malignancy, and have severe pain due to malignancy that is at or below the level of the chest and not adequately relieved by other pain control therapies.
DESIGN:
This is a single site, non-randomized, open-label, dose-escalation study using a modified Fibonacci scheme. The starting dose of RTX was 13 micrograms given as a 2 mL injection via an intra-spinal catheter over approximately 30 seconds followed by a 1 mL flush. Six subjects were dosed at this level and 3 were dosed at the 26 microgram dose level at the same volume of injectate, flush and injection time. Under the study design, RTX doses were to be increased in progressively smaller percentage increments with each dose-escalation to occur in sequential groups of 3 subjects until 1 escalation above the effective dose in 100% of subjects (ED100), completion of the 100 microgram dose level, or establishment of the maximum tolerated dose (MTD), whichever occurs first. The total duration of study participation for any subject will be up to 7 months.
The amended RTX injection technique reduced the injection volume and increased the injection time to reduce the spread of RTX to above the T6 (sixth thoracic) vertebral level. The present technique is a 1 mL injection over 2 minutes (0.5 mL/minute) given via infusion pump, followed by flushing of the IT catheter with the minimum volume of sterile, preservative-free saline necessary to clear the internal volume of the catheter used for the injection. Three patients were treated at the new starting dose of 13 micrograms with the new injection technique. The next 3-patient cohort will receive a dose of 26 micrograms. The subsequent subject dose tier will receive a dose of 44 micrograms consistent with the protocol s dose-escalation algorithm. Administering this dose will require an infusion volume of 1.76 mL of the present 25 micrograms/mL RTX formulation.
OUTCOME MEASURES:
The primary study outcome is the ED100, the MTD, or the maximum dose administered, whichever is achieved first during dose-escalation. The primary pain variable for determining the ED100 is the daily worst pain intensity score averaged over a 4 to 7-day period during the 3 weeks before RTX dosing and during Days 8 through 14 after dosing. The numerical rating scale (NRS), administered verbally during a daily telephone interview, will be the primary pain assessment instrument. For a given subject, the treatment will be considered effective if the subject experiences a greater than or equal to 50% reduction in the mean daily worst pain score assessed by NRS (evaluated on Study Day 15). We may also consider RTX treatment to be successful if there is greater than or equal to 50% reduction in opiate intake, measured by morphine milligram equivalents (MME) per day, even if pain levels are unchanged after RTX treatment.
Secondary outcome measures will be other surveys of pain, including an assessment of worst daily pain by the visual analog scale, and assessments of function and quality of life.
Safety assessments will include hematology; serum clinical chemistry tests; cerebrospinal fluid examinations; physical, neurological, and eye examinations; reporting of adverse events; electrocardiograms; and findings on magnetic resonance imaging of the spine and brain.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Drug: Intrathecal Resiniferatoxinadvanced cancer patients with pain |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (32 listed)
- Subject inclusion criteria are based on inadequate control of pain despite best efforts, including appropriate use of analgesic medications. To be enrolled in the study, subjects must meet all of the following criteria:
- Age 18 years or older.
- Clinical and histological diagnosis of cancer with disease that has not adequately responded to standard therapies. A pathology report documenting malignancy is required.
- Subject not currently seeking or receiving potentially curative therapies for cancer (e.g., chemotherapy or immunotherapy). Curative cancer therapy may be sought after the Day 15 clinic visit, and palliative anti-tumor therapy is allowed as long as the subject was established on that therapy prior to enrollment (see exclusion criterion 6).
- Mean daily worst pain NRS score of greater than or equal to 6 for pain at or below the T6 dermatome (level of the chest) that is associated with a malignant disease. The mean score must be derived from recordings on at least 4 of 7 consecutive days within 3 weeks preceding treatment.
Key exclusion criteria (20 listed)
- Primary pain source from anatomical regions at T5 dermatome or above.
- Pain due to causes other than cancer or its treatment that is moderate to severe in intensity.
- Anatomic abnormality or pathology of the spinal cord and/or IT space on magnetic resonance imaging (MRI) that could increase the risk of adverse effects of catheter placement or interfere with CSF flow.
- Evidence of advanced brain pathology or elevated intracranial pressure as determined by symptoms, history, physical examination (including neurological and eye examination), and/or MRI.
- Presence of an IT shunt device (e.g., ventriculo-peritoneal and ventriculo-atrial shunts).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intrathecal Resiniferatoxinphase I, single-site, non-randomized, open-label, dose-escalation study to determine the safety and efficacy of IT RTX in subjects with severe refractory pain due to advanced malignancy | Drug | Single Arm |
Primary outcome
Investigate the safety and efficacy of RTX administered intrathecally in subjects with severe refractory pain associated with advanced cancer along with ED100, the MTD, or the maximum dose administered, whichever is achieved first during dose es...
Time frame: Day 7, Day 15, Day 68, Day 188
Dose escalation decisions are based on both effectiveness (as assessed by mean worst pain on the NRS) and DLT. The dose escalation population will include all subjects who are dosed, complete the NRS, and have the DLT assessments from baseline through Day 15. Subjects who are not evaluable for dose escalation will be replaced.
1 secondary outcome
Secondary outcome measures will be other surveys of pain, including an assessment of worst daily pain by the visual analog scale, and assessments of function and quality of life.
Time frame: Day 7, Day 15, Day 68, Day 188
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2026.
- Background: PubMed 7116316Daut RL, Cleeland CS. The prevalence and severity of pain in cancer. Cancer. 1982 Nov 1;50(9):1913-8. doi: 10.1002/1097-0142(19821101)50:93.0.co;2-r.
- Background: PubMed 7508092Cleeland CS, Gonin R, Hatfield AK, Edmonson JH, Blum RH, Stewart JA, Pandya KJ. Pain and its treatment in outpatients with metastatic cancer. N Engl J Med. 1994 Mar 3;330(9):592-6. doi: 10.1056/NEJM199403033300902.
- Background: PubMed 718345Oster MW, Vizel M, Turgeon LR. Pain of terminal cancer patients. Arch Intern Med. 1978 Dec;138(12):1801-2.
- Derived from this study: PubMed 40423401Mannes AJ, Heiss JD, Berger A, Alewine CC, Butman JA, Hughes MS, Rabbee N, Hayes C, Williams TS, Sapio MR, Iadarola MJ. Treatment of Intractable Cancer Pain with Resiniferatoxin - An Interim Study. NEJM Evid. 2025 Jun;4(6):EVIDoa2400423. doi: 10.1056/EVIDoa2400423. Epub 2025 May 27.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided. We do plan to share IPD. we will share all IPD that results in a publication on a public repository, as required by most journals. the data will be de-identified and anonymized.
Related trials for Intractable Pain
RecruitingObservational study
Post Market Evaluation of Real World Outcomes and Durability to Support Implantable Inceptiv™ Spinal Cord Stimulation Therapy
Chronic Pain, Intractable Pain
Not yet recruitingPhase 1/2
Periganglionic Resiniferatoxin for the Treatment of Intractable Pain Due to Cancer-induced Bone Pain
Intractable Pain, Palliative Care
Recruiting
Managing Pain Using Optimized Sequences by Adjusting Parameters With Independent Current Control
Chronic Pain, Intractable Pain
Not yet recruitingObservational study
Lidocaine Infusions for Pain in Patients With Malignant Bowel Obstruction
Malignant Bowel Obstruction, Cancer Pain
Active, not recruitingObservational study
Patients Referred to the Chronic Pain Unit for Palliative Treatment With Ozone Therapy Between 2022 and 2025
Radiation Toxicity, Chemotherapeutic Toxicity
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John D Heiss, M.D. principal investigator
National Institute of Neurological Disorders and Stroke (NINDS)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Intrathecal Resiniferatoxin in people with Intractable Pain and Palliative Care, run by National Institute of Dental and Craniofacial Research (NIDCR) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 32 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Investigate the safety and efficacy of RTX administered intrathecally in subjects with severe refractory pain associated with advanced cancer along with ED100, the MTD, or the maximum dose administered, whichever is achieved first during dose es... (time frame: Day 7, Day 15, Day 68, Day 188). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00804154, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2026.