Erythropoietic Protoporphyria Clinical Trial
Official title:
(AURORA) A Randomized, Double-blind, Placebo-Controlled Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and Protoporphyrin IX (PPIX) Concentrations in Participants With Erythropoietic Protoporphyria (EPP)
Verified date | April 2024 |
Source | Disc Medicine, Inc |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This is a Phase 2, multi-center, double-blind, placebo-controlled, parallel group study of bitopertin to evaluate the safety, tolerability, efficacy, and PPIX concentration change in participants with EPP. Participants may roll over to an open label extension portion after completing the double-blind treatment period.
Status | Active, not recruiting |
Enrollment | 75 |
Est. completion date | June 2024 |
Est. primary completion date | February 20, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: 1. Aged 18 years or older at the time of signing the informed consent form (ICF). 2. Diagnosis of EPP, based on medical history by ferrochelatase ( FECH) genotyping or by biochemical porphyrin analysis. 3. Body weight =50 kg. 4. Washout of at least 2 months prior to Screening of afamelanotide and dersimelagon, if applicable. 5. Aspartate aminotransferase (AST) and alanine transaminase (ALT) <2× upper limit of normal (ULN) and total bilirubin <ULN (unless documented Gilbert syndrome) at Screening. Albumin >lower limit of normal (LLN). Exclusion Criteria: Medical History: 1. Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery. 2. Other than EPP, an inherited or acquired red cell disease associated with anemia. 3. A history or known allergic reaction to any investigational product excipients or history of anaphylaxis to any food or drug. 4. History of liver transplantation. 5. History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator. 6. Human immunodeficiency virus (HIV), active Hepatitis B, or C. 7. Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude the participant from participating in the study 8. Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months. Treatment History: 9. Concurrent or planned treatment with afamelanotide or dersimelagon during the study period. 10. Treatment with opioids for any period >7 days in the 2 months prior to screening or anticipated to require opioid use for >7 days at any point during the study. 11. New treatment for anemia, including initiation of iron supplementation, in the 2 months prior to Screening. 12. Current or planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of CYP3A4 enzymes for 28 days prior to the first dose and throughout the study. Laboratory Exclusions: 13. Hemoglobin <10 g/dL at Screening. |
Country | Name | City | State |
---|---|---|---|
United States | University of Alabama Hospital | Birmingham | Alabama |
United States | Massachusetts General Hospital | Boston | Massachusetts |
United States | University of Texas | Galveston | Texas |
United States | University of Miami Miller School of Medicine | Miami | Florida |
United States | Mount Sinai Hospital | New York | New York |
United States | Einstein Medical Center | Philadelphia | Pennsylvania |
United States | University of California San Francisco | San Francisco | California |
United States | Fred Hutchinson Cancer Center | Seattle | Washington |
United States | Atrium Health Wake Forest Baptist | Winston-Salem | North Carolina |
Lead Sponsor | Collaborator |
---|---|
Disc Medicine, Inc |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Plasma maximum measured drug concentration (Cmax) | 121 days | ||
Other | Time of maximum concentration (Tmax) | 121 days | ||
Other | Area under the concentration-time curve (AUC) | 121 days | ||
Primary | Percent change from baseline in whole blood metal-free PPIX levels | 121 days | ||
Secondary | Total hours of sunlight exposure to skin on days with no pain from 1000 to 1800 hours (10:00am to 6:00pm) | 121 days | ||
Secondary | Daily sunlight exposure time (minutes) to first prodromal symptom (burning, tingling, itching, or stinging) associated with sunlight exposure between 1 hour post-sunrise and 1 hour pre-sunset | 121 days | ||
Secondary | Pain intensity of phototoxic reactions according to a Likert scale (0-10) | 121 days | ||
Secondary | Incidence of treatment-emergent adverse events | 121 days | ||
Secondary | Erythrocyte total PPIX concentrations | 121 days | ||
Secondary | Plasma total PPIX concentrations | 121 days | ||
Secondary | Whole blood total PPIX concentrations | 121 days | ||
Secondary | Plasma bitopertin concentrations | 121 days |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT00979745 -
Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)
|
Phase 3 | |
Completed |
NCT00004831 -
Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria
|
N/A | |
Completed |
NCT05572125 -
Iron Therapy in Erythropoietic Protoporphyria
|
||
Completed |
NCT01605136 -
Phase III Confirmatory Study in Erythropoietic Protoporphyria
|
Phase 3 | |
Completed |
NCT01422915 -
Sorbent Therapy of the Cutaneous Porphyrias
|
Phase 2/Phase 3 | |
Completed |
NCT04053270 -
Multicentre Phase III Erythropoietic Protoporphyria Study
|
Phase 3 | |
Completed |
NCT00004940 -
Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria
|
Phase 3 | |
Recruiting |
NCT06388642 -
Pharmacokinetics of Afamelanotide in EPP Patients
|
Phase 1/Phase 2 | |
Recruiting |
NCT05020184 -
Effect of Oral Cimetidine in the Protoporphyrias
|
Phase 2 | |
Completed |
NCT01688895 -
Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact
|
||
Completed |
NCT04578496 -
A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
|
Phase 3 | |
Recruiting |
NCT00206869 -
Does Exercise and Heat Increase the Lightsensibility in Patients With Erythropoietic Protoporphyria
|
N/A | |
Recruiting |
NCT05780840 -
Protection Against Visible Light by Dihydroxyacetone in Erythropoietic Protoporphyria
|
N/A | |
Completed |
NCT02979249 -
Oral Iron for Erythropoietic Protoporphyrias
|
N/A | |
Enrolling by invitation |
NCT05883748 -
Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP
|
Phase 2/Phase 3 | |
Completed |
NCT01097044 -
Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)
|
Phase 2 | |
Completed |
NCT03682731 -
Light Exposure Patterns and Symptoms Among Patients With Erythropoietic Protoporphyria
|