Effect of Oral Cimetidine in the Protoporphyrias

• Register: ClinicalTrials.gov
• Last refreshed on: 11 November 2024
• Main ID: NCT05020184
• Date of registration: 26/07/2021
• Prospective Registration: Yes
• Primary sponsor: Amy K. Dickey, M.D.
• Public title: Effect of Oral Cimetidine in the Protoporphyrias
• Scientific title: Effect of Oral Cimetidine in the Protoporphyrias
• Date of first enrolment: June 14, 2022
• Target sample size: 20
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT05020184
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Karl Anderson, MD
• Affiliation: University of Texas

• Name: Amy K Dickey, MD
• Affiliation: Massachusetts General Hospital

Key inclusion & exclusion criteria

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 renal insufficiency


Age minimum: 15 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Erythropoietic Protoporphyria

X-linked Protoporphyria

Intervention(s)

Drug: Cimetidine

Drug: Placebo

Primary Outcome(s)

Erythrocyte total protoporphyrin level [Time Frame: Before and after each 3-month treatment period]

Secondary Outcome(s)

Patient-reported quality of life [Time Frame: Before and after each 3-month treatment period]

Light dose [Time Frame: Last 2 months of each treatment period]

Phototoxic episodes [Time Frame: Last 2 months of each treatment period]

Time to prodrome [Time Frame: Last 2 months of each treatment period]

Secondary ID(s)

PC7211

2021P002095

1R01FD007287-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Wake Forest University Health Sciences

The University of Texas Medical Branch, Galveston