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Effect of Oral Cimetidine in the Protoporphyrias (IND 153247 submitted 9/2/2020)

Award Number: R01FD007287
ORGANIZATION: FOOD AND DRUG ADMINISTRATION
OPDIV: FDA
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/10/2021
PERIOD OF PERFORMANCE END DATE: 04/30/2026

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Effect of Oral Cimetidine in the Protoporphyrias (IND 153247 submitted 9/2/2020) - PROJECT SUMMARY/ABSTRACT Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) are a genetic defects of heme biosynthesis that cause life-long, painful cutaneous sensitivity to light. EPP and XLP, collectively called the protoporphyrias, result in the accumulation of the light-sensitive molecule protoporphyrin IX (PPIX) in erythrocytes and secondarily in the plasma and the liver. In addition to photosensitivity, protoporphyria can also result in anemia, gallstones, and chronic liver disease, and in 2-5% of cases it results in rapidly progressive cholestatic liver failure that is fatal without liver transplantation. Because patients are sensitive to visible light and not UV light, sunscreen is not effective in EPP. Afamelanotide, which increases cutaneous melanin, was recently approved by the FDA for the prevention of photosensitivity in EPP. Additional therapies besides afamelanotide are necessary because afamelanotide is does not change PPIX level and therefore does not prevent the life-threatening complications of EPP. Cimetidine has gained attention as a possible treatment for human porphyrias because of a potential off-target effect of inhibition of ALAS, the first enzyme of heme biosynthesis. This inhibition was first described in vitro. Later case reports suggested that cimetidine was beneficial for the treatment of acute intermittent porphyria and porphyria cutanea tarda. Subsequently, case reports also described a potential effect in EPP. However, these reports are anecdotal and uncontrolled. Some patients also had pre-existing hepatic damage from EPP, which complicates the observations of drug effects in EPP. Communications in the porphyria community indicate that a large number of patients, including children, have used cimetidine without oversight and without a systematic record of dosage or symptomatic improvement. The considerable interest in the larger porphyria community to assess with care its possible benefit as a treatment approach has therefore neither been addressed nor satisfied. Therefore, the objective of this study is to determine the efficacy and safety of oral cimetidine administration in the protoporphyrias. The study design is a multicenter, prospective, randomized, double-blind, placebo-controlled, crossover trial, and efficacy will be based on protoporphyrin levels, photosensitivity, and quality of life questionnaires. If the results are positive, this would be the first study providing quality evidence for an agent acting as a disease-modifying therapy for protoporphyria.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2025 ( Subtotal = $313,713 )
2025	2025	THE GENERAL HOSPITAL CORPORATION	55 FRUIT ST	BOSTON	MA	02114	SUFFOLK	USA	Food and Drug Administration Research	000	4	5/31/2025	NON-COMPETING CONTINUATION	$313,713
														Subtotal = $313,713

Issue Date FY: 2024 ( Subtotal = $403,457 )
2024	2024	THE GENERAL HOSPITAL CORPORATION	55 FRUIT ST	BOSTON	MA	02114	SUFFOLK	USA	Food and Drug Administration Research	000	3	5/8/2024	NON-COMPETING CONTINUATION	$403,457
														Subtotal = $403,457

Issue Date FY: 2023 ( Subtotal = $403,457 )
2023	2023	THE GENERAL HOSPITAL CORPORATION	55 FRUIT ST	BOSTON	MA	02114	SUFFOLK	USA	Food and Drug Administration Research	000	2	4/27/2023	NON-COMPETING CONTINUATION	$403,457
														Subtotal = $403,457

Issue Date FY: 2022 ( Subtotal = $0 )
2022	2021	MASSACHUSETTS GENERAL HOSPITAL, THE	55 FRUIT ST	BOSTON	MA	02114	SUFFOLK	USA	Food and Drug Administration Research	000	1	5/4/2022	NEW	$0
														Subtotal = $0

Issue Date FY: 2021 ( Subtotal = $498,495 )
2021	2021	MASSACHUSETTS GENERAL HOSPITAL, THE	55 FRUIT ST	BOSTON	MA	02114	SUFFOLK	USA	Food and Drug Administration Research	000	1	9/10/2021	NEW	$498,495
														Subtotal = $498,495

Grand Total All Awards = $1,619,122

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Effect of Oral Cimetidine in the Protoporphyrias (IND 153247 submitted 9/2/2020)

Award Number: R01FD007287

ORGANIZATION: FOOD AND DRUG ADMINISTRATION

OPDIV: FDA

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 09/10/2021

PERIOD OF PERFORMANCE END DATE: 04/30/2026

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