Effect of Oral Cimetidine in the Protoporphyrias (NCT05020184) | Clinical Trial Compass
CompletedPhase 2
Effect of Oral Cimetidine in the Protoporphyrias
United States26 participantsStarted 2022-06-14
Plain-language summary
Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) result from genetic defects of heme biosynthesis that cause life-long, painful cutaneous sensitivity to light. The objective of this study is to determine the efficacy and safety of oral cimetidine administration for treatment of the protoporphyrias. Efficacy will be based on protoporphyrin levels, photosensitivity, and quality of life questionnaires.
Funding Source- FDA OOPD
Who can participate
Age range
15 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Prior enrollment or co-enrollment in the Longitudinal Study of the Porphyrias (PC Study 7201) with a confirmed diagnosis of EPP or XLP
* Male or female age ≥15 years at screening
* Characteristic history of non-blistering cutaneous photosensitivity
* Willing and capable of giving informed consent and following procedures described in the protocol
Exclusion Criteria:
* Participants not willing to expose themselves to light to the point of prodromal symptoms at least weekly
* History of liver or bone marrow transplant or clinically significant liver dysfunction as determined by the Investigator
* Known or suspected allergy or intolerance to cimetidine
* Use of any other experimental therapy in the past 3 months at screening
* Use of cimetidine within the past 3 months at screening
* Individuals with elevations of porphyrins in plasma or erythrocytes due to other diseases (i.e., secondary porphyrinemia) such as liver and bone marrow diseases
* Patients with any clinically significant comorbid conditions, which in the opinion of the Investigator, precludes participation
* Treatment with any drugs or supplements (Appendix 1) that in the opinion of the Investigator can interfere with subject safety or the objectives of the study
* The participant either does not have a smartphone or is not willing to use his/her smartphone for the study
* Women who are pregnant, breastfeeding, or actively planning to become pregnant
* Individuals with moderate to severe ren…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percent Change in Erythrocyte Total Protoporphyrin Level
Timeframe: Before and after each 3-month treatment period