Trial Outcomes & Findings for Oral Iron for Erythropoietic Protoporphyrias (NCT NCT02979249)

NCT ID: NCT02979249

Last Updated: 2020-09-11

Results Overview

Relative difference in erythrocyte protoporphyrin levels between baseline and 12 months after treatment

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 16 participants
• Primary outcome timeframe: Baseline and at 12 months
• Results posted on: 2020-09-11

Participant Flow

Participant milestones

Measure	Oral Iron standard dose of oral iron pills for one year
Overall Study STARTED	16
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Measure	Oral Iron standard dose of oral iron pills for one year
Overall Study Lost to Follow-up	2
Overall Study Withdrawal by Subject	4

Baseline Characteristics

Oral Iron for Erythropoietic Protoporphyrias

Baseline characteristics by cohort

Measure	Oral Iron n=16 Participants standard dose of oral iron pills for one year
Age, Continuous	38.8 years STANDARD_DEVIATION 14.5 • n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	14 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	16 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and at 12 months

Relative difference in erythrocyte protoporphyrin levels between baseline and 12 months after treatment

Outcome measures

Measure	Oral Iron n=10 Participants standard dose of oral iron pills for one year
Change in Erythrocyte Protoporphyrin Levels Baseline	2314.6 µg/dL Standard Deviation 1820.3
Change in Erythrocyte Protoporphyrin Levels 12 months	2240.4 µg/dL Standard Deviation 2312.6

SECONDARY outcome

Timeframe: Baseline and 12 months

Quality of life tool designed to capture the specific issues that patients with EPP experience. Score range is 0-100, higher scores represent higher levels or satisfaction, lower scores represent more impacted quality of life. Mean change at 12 months as compared to baseline.

Outcome measures

Measure	Oral Iron n=10 Participants standard dose of oral iron pills for one year
Mean Change in EPP-specific Quality of Life Questionnaire	3.3 mean change in score on a scale Standard Deviation 18.3

Adverse Events

Oral Iron

Serious events: 3 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Measure	Oral Iron n=10 participants at risk standard dose of oral iron pills for one year
Gastrointestinal disorders Diarrhea	10.0% 1/10 • 12 months
Pregnancy, puerperium and perinatal conditions Miscarriage	10.0% 1/10 • 12 months
Eye disorders Conjunctivitis	10.0% 1/10 • 12 months

Other adverse events

Measure	Oral Iron n=10 participants at risk standard dose of oral iron pills for one year
Gastrointestinal disorders Abdominal Pain	20.0% 2/10 • 12 months
Skin and subcutaneous tissue disorders Photosensitivity	20.0% 2/10 • 12 months
Gastrointestinal disorders Nausea	10.0% 1/10 • 12 months
Infections and infestations Sinusitis	10.0% 1/10 • 12 months
Gastrointestinal disorders Dyspepsia	10.0% 1/10 • 12 months
Metabolism and nutrition disorders Elevated Protoporphyrin	10.0% 1/10 • 12 months
Respiratory, thoracic and mediastinal disorders Productive cough	10.0% 1/10 • 12 months
Renal and urinary disorders Urinary Tract Pain	10.0% 1/10 • 12 months
Blood and lymphatic system disorders Elevated Protoporphyrin	10.0% 1/10 • 12 months
Infections and infestations Mucosal Infection	10.0% 1/10 • 12 months
Gastrointestinal disorders Constipation	10.0% 1/10 • 12 months

Additional Information

Dr. Hetanshi Naik

Icahn School of Medicine at Mount Sinai

Phone: 212-659-8512

Email: hetanshi.naik@mssm.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place