Bitopertin Clinical Trials

Size	Price	Qty
5mg	USD 147
25mg	USD 477

Clinical Trials

Numerous clinical trials spanning early- to late-phase development evaluate bitopertin across diverse psychiatric and hematologic conditions. Early research sponsored by Hoffmann-La Roche addressed central nervous system disorders such as schizophrenia and obsessive-compulsive disorder, whereas studies by Disc Medicine Inc. and the National Heart, Lung, and Blood Institute target erythroid and porphyric conditions including erythropoietic protoporphyria, X-linked protoporphyria, beta-thalassemia, and steroid-refractory Diamond-Blackfan anemia. These trials encompass recruitment statuses spanning recruiting, enrolling by invitation, active not recruiting, completed, and withdrawn.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT07123363	WITHDRAWN	Protoporphyria	Wake Forest University Health Sciences	2026-01	PHASE1
NCT05883748	ENROLLING_BY_INVITATION	Erythropoietic Protoporphyria	Disc Medicine, Inc	2023-08-31	PHASE2; PHASE3
NCT06910358	ACTIVE_NOT_RECRUITING	Erythropoietic Protoporphyria (EPP); X-Linked Protoporphyria (XLP)	Disc Medicine, Inc	2025-04-04	PHASE3
NCT05828108	COMPLETED	Steroid-refractory Diamond-Blackfan Anemia (DBA)	National Heart, Lung, and Blood Institute (NHLBI)	2023-07-25	PHASE1; PHASE2
NCT05308472	COMPLETED	Erythropoietic Protoporphyria	Disc Medicine, Inc	2022-10-31	PHASE2
NCT05883748	Enrolling by invitation	Erythropoietic Protoporphyria	Disc Medicine Inc	2023-08-31	Phase 2\|Phase 3
NCT05828108	Recruiting	Steroid-refractory Diamond-Blackfan Anemia (DBA)	National Heart Lung and Blood Institute (NHLBI)\|National Institutes of Health Clinical Center (CC)	2023-07-25	Phase 1\|Phase 2
NCT05308472	Active not recruiting	Erythropoietic Protoporphyria	Disc Medicine Inc	2022-10-31	Phase 2
NCT03271541	COMPLETED	Beta-Thalassemia	Hoffmann-La Roche	2017-10-26	PHASE2
NCT03271541	Completed	Beta-Thalassemia	Hoffmann-La Roche	2017-10-26	Phase 2
NCT01674361	COMPLETED	Obsessive-Compulsive Disorder	Hoffmann-La Roche	2012-12-31	PHASE2
NCT01235559	COMPLETED	Schizophrenia	Hoffmann-La Roche	2010-12	PHASE3
NCT01235585	COMPLETED	Schizophrenia	Hoffmann-La Roche	2010-12	PHASE3
NCT01192880	COMPLETED	Schizophrenia	Hoffmann-La Roche	2010-11	PHASE3
NCT01192906	COMPLETED	Schizophrenia	Hoffmann-La Roche	2010-12	PHASE3
NCT02019290	WITHDRAWN	Healthy Volunteer	Hoffmann-La Roche	2014-02-10	PHASE1
NCT02019290	Withdrawn	Healthy Volunteer	Hoffmann-La Roche	2014-02-10	Phase 1
NCT01674361	Completed	Obsessive-Compulsive Disorder	Hoffmann-La Roche	2012-12-31	Phase 2
NCT01234779	COMPLETED	Schizophrenia	Hoffmann-La Roche	2011-02	PHASE2
NCT01636492	COMPLETED	Healthy Volunteer	Hoffmann-La Roche	2005-11	PHASE1

(data from https://clinicaltrials.gov, updated on 2026-01-29)

Check the Bitopertin product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Bitopertin acts as a potent and selective inhibitor of glycine transporter 1, binding the transporter to block cellular reuptake of glycine and restrict substrate availability for the initial step of heme synthesis. By limiting glycine accumulation within erythroid cells, bitopertin decreases the toxic accumulation of protoporphyrin IX and regulates heme biosynthesis, providing therapeutic relevance for clinical conditions such as erythropoietic protoporphyria and steroid-refractory Diamond-Blackfan anemia.

Appendix RUO and cGMP Quality Standards

Quality Dimension	RUO (Research Use Only)	cGMP (Current Good Manufacturing Practice)
Clinical Applicability	Prohibited in human clinical trials or medical diagnostics.	Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status	Non-regulated grade; exempt from drug manufacturing laws.	Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment	Unclassified analytical or research laboratories.	Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control	Basic purity and activity testing.	Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation	Basic equipment calibration; no process validation required.	Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance	Vendor self-declared without required formal QMS.	Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact	High risk of IND rejection if used as a critical raw material.	Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).