The Effects of Tiopronin on 3-Aminopropanal Level & Neurologic Outcome After Aneurysmal Subarachnoid Hemorrhage

NCT ID: NCT01095731

Last Updated: 2025-10-29

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-04-30

Study Completion Date

2013-07-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The purpose of this phase II study is to further assess the safety of tiopronin in aneurysmal subarachnoid hemorrhage(aSAH) patients in order to obtain preliminary data on the efficacy of tiopronin versus placebo in reducing serum and cerebrospinal fluid (CSF) 3AP levels in this patient population.

Funding Source - FDA Office of Orphan Products Development

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

The annual rate of aSAH in United States is approximately 18 to 24 thousand cases each year. Mortality rates following aSAH range from 30-70% with 10-20% of survivors experiencing severe neurological disability. Following aSAH, a major cause of morbidity and mortality is vasospasm, which causes delayed ischemic neurologic deterioration. There is currently no effective treatment for preventing or ameliorating the damage that occurs following cerebral ischemia. A myriad of neuro-toxins are produced in the ischemic brain resulting in a vicious cycle of cellular death and destruction. The polyamines spermine and spermidine are metabolized by polyamine oxidase (PAO) into putrescine and 3-aminopropanal (3AP).

Tiopronin (Thiola) is an FDA approved drug used for the treatment of cystine stones in patients with cystinuria in the U.S. In Europe, it is also used for the treatment of rheumatoid arthritis and bronchial hypersecretion. In previous animal studies, we demonstrated that tiopronin is able to bind and neutralize the toxic effects of 3AP. We have shown in previous studies that aSAH patients have elevated 3AP levels, and higher levels correlate to a poor neurologic outcome.

The goals of this phase II multicenter, randomized, double-blinded safety and efficacy trial are to (1) further evaluate the safety of the drug in our patient population at the dose established in phase I; (2) demonstrate that tiopronin crosses the blood-brain barrier; (3) show that both serum and CSF 3AP levels are reduced by administration of tiopronin; and (4) demonstrate that a reduction in 3AP levels is associated with improved neurologic outcome in aSAH patients.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Aneurysmal Subarachnoid Hemorrhage

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Sugar Pill, Hunt Hess Grade I-V

Good Grade (Hunt Hess I-III) and Poor Grade (Hunt Hess IV-V)

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Dosage will be 1 gram, 3 times daily. Drug dosing will be initiated at time of aSAH confirmation, for a length of 14 days or at hospital discharge.

Tiopronin, Hunt Hess Grade I-V

Good Grade (Hunt Hess I-III) and Poor Grade (Hunt Hess IV-V)

Group Type EXPERIMENTAL

Tiopronin

Intervention Type DRUG

Dosage will be 1 gram, 3 times daily. Drug dosing will be initiated at time of aSAH confirmation, for a length of 14 days or at hospital discharge.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Tiopronin

Dosage will be 1 gram, 3 times daily. Drug dosing will be initiated at time of aSAH confirmation, for a length of 14 days or at hospital discharge.

Intervention Type DRUG

Placebo

Dosage will be 1 gram, 3 times daily. Drug dosing will be initiated at time of aSAH confirmation, for a length of 14 days or at hospital discharge.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

IUPAC Name:2-(2-sulfanylpropanoylamino)acetic acid CAS Number: 1953-02-2 Thiola Thiopronin Thiosol Tioglis Acadione Capen Captimer Epatiol Vincol Mucolysin Sutilan Meprin (detoxicant) Thiolpropionamidoacetic acid N-2-Mercaptopropionyl glycine 2-(2-sulfanylpropanoylamino)ethanoic acid 2-(2-sulfanylpropanoylamino)acetic acid placebo, sugar pill

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Admitted to a recruiting center with aneurysmal subarachnoid hemorrhage
* Ability to initiate study drug treatment within 96 hours of aSAH onset.
* Ability to provide either informed or surrogate consent

Exclusion Criteria

* Hypersensitivity to penicillamine
* Creatinine level greater than 1.5/mm\^3 on admission
* Platelet count of less than 100,000/mm\^3 on admission
* White blood cell count of less than 3.5/mm\^3 on admission
* AST or ALT of greater than 60/L on admission or history of liver failure
* Pregnancy
* History of lupus, Goodpasture's syndrome, myasthenia gravis, pemphigus, nephrotic syndrome, glomerulonephritis, or renal failure
* Patients considered unable to comply with the protocol
Minimum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

University of Florida

OTHER

Sponsor Role collaborator

University of Washington

OTHER

Sponsor Role collaborator

Food and Drug Administration (FDA)

FED

Sponsor Role collaborator

E. Sander Connolly

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

E. Sander Connolly

Professor of Neurological Surgery

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

E Sander Connolly, M.D.

Role: PRINCIPAL_INVESTIGATOR

Columbia University

Brian Hoh, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of Florida

Louis Kim, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of Washington

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

University of Florida

Gainesville, Florida, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

References

Explore related publications, articles, or registry entries linked to this study.

Linn FH, Rinkel GJ, Algra A, van Gijn J. Incidence of subarachnoid hemorrhage: role of region, year, and rate of computed tomography: a meta-analysis. Stroke. 1996 Apr;27(4):625-9. doi: 10.1161/01.str.27.4.625.

Reference Type BACKGROUND
PMID: 8614919 (View on PubMed)

Hop JW, Rinkel GJ, Algra A, van Gijn J. Case-fatality rates and functional outcome after subarachnoid hemorrhage: a systematic review. Stroke. 1997 Mar;28(3):660-4. doi: 10.1161/01.str.28.3.660.

Reference Type BACKGROUND
PMID: 9056628 (View on PubMed)

Mayer S, Kreiter K. Quality of life after subarachnoid hemorrhage. J Neurosurg. 2002 Sep;97(3):741-2; author reply 742. doi: 10.3171/jns.2002.97.3.0741. No abstract available.

Reference Type BACKGROUND
PMID: 12296671 (View on PubMed)

Kassell NF, Sasaki T, Colohan AR, Nazar G. Cerebral vasospasm following aneurysmal subarachnoid hemorrhage. Stroke. 1985 Jul-Aug;16(4):562-72. doi: 10.1161/01.str.16.4.562.

Reference Type BACKGROUND
PMID: 3895589 (View on PubMed)

Solenski NJ, Haley EC Jr, Kassell NF, Kongable G, Germanson T, Truskowski L, Torner JC. Medical complications of aneurysmal subarachnoid hemorrhage: a report of the multicenter, cooperative aneurysm study. Participants of the Multicenter Cooperative Aneurysm Study. Crit Care Med. 1995 Jun;23(6):1007-17. doi: 10.1097/00003246-199506000-00004.

Reference Type BACKGROUND
PMID: 7774210 (View on PubMed)

Shohami E, Nates JL, Glantz L, Trembovler V, Shapira Y, Bachrach U. Changes in brain polyamine levels following head injury. Exp Neurol. 1992 Aug;117(2):189-95. doi: 10.1016/0014-4886(92)90126-b.

Reference Type BACKGROUND
PMID: 1499692 (View on PubMed)

Ivanova S, Botchkina GI, Al-Abed Y, Meistrell M 3rd, Batliwalla F, Dubinsky JM, Iadecola C, Wang H, Gregersen PK, Eaton JW, Tracey KJ. Cerebral ischemia enhances polyamine oxidation: identification of enzymatically formed 3-aminopropanal as an endogenous mediator of neuronal and glial cell death. J Exp Med. 1998 Jul 20;188(2):327-40. doi: 10.1084/jem.188.2.327.

Reference Type BACKGROUND
PMID: 9670045 (View on PubMed)

Dogan A, Rao AM, Hatcher J, Rao VL, Baskaya MK, Dempsey RJ. Effects of MDL 72527, a specific inhibitor of polyamine oxidase, on brain edema, ischemic injury volume, and tissue polyamine levels in rats after temporary middle cerebral artery occlusion. J Neurochem. 1999 Feb;72(2):765-70. doi: 10.1046/j.1471-4159.1999.0720765.x.

Reference Type BACKGROUND
PMID: 9930751 (View on PubMed)

Seiler N. Polyamine oxidase, properties and functions. Prog Brain Res. 1995;106:333-44. doi: 10.1016/s0079-6123(08)61229-7.

Reference Type BACKGROUND
PMID: 8584670 (View on PubMed)

Ivanova S, Batliwalla F, Mocco J, Kiss S, Huang J, Mack W, Coon A, Eaton JW, Al-Abed Y, Gregersen PK, Shohami E, Connolly ES Jr, Tracey KJ. Neuroprotection in cerebral ischemia by neutralization of 3-aminopropanal. Proc Natl Acad Sci U S A. 2002 Apr 16;99(8):5579-84. doi: 10.1073/pnas.082609299. Epub 2002 Apr 9.

Reference Type BACKGROUND
PMID: 11943872 (View on PubMed)

Cockroft KM, Meistrell M 3rd, Zimmerman GA, Risucci D, Bloom O, Cerami A, Tracey KJ. Cerebroprotective effects of aminoguanidine in a rodent model of stroke. Stroke. 1996 Aug;27(8):1393-8. doi: 10.1161/01.str.27.8.1393.

Reference Type BACKGROUND
PMID: 8711808 (View on PubMed)

Wood PL, Khan MA, Moskal JR. Neurochemical analysis of amino acids, polyamines and carboxylic acids: GC-MS quantitation of tBDMS derivatives using ammonia positive chemical ionization. J Chromatogr B Analyt Technol Biomed Life Sci. 2006 Feb 2;831(1-2):313-9. doi: 10.1016/j.jchromb.2005.12.031. Epub 2006 Jan 10.

Reference Type BACKGROUND
PMID: 16406747 (View on PubMed)

Wood PL, Khan MA, Moskal JR, Todd KG, Tanay VA, Baker G. Aldehyde load in ischemia-reperfusion brain injury: neuroprotection by neutralization of reactive aldehydes with phenelzine. Brain Res. 2006 Nov 29;1122(1):184-90. doi: 10.1016/j.brainres.2006.09.003. Epub 2006 Oct 5.

Reference Type BACKGROUND
PMID: 17026969 (View on PubMed)

Fisher CM, Kistler JP, Davis JM. Relation of cerebral vasospasm to subarachnoid hemorrhage visualized by computerized tomographic scanning. Neurosurgery. 1980 Jan;6(1):1-9. doi: 10.1227/00006123-198001000-00001.

Reference Type BACKGROUND
PMID: 7354892 (View on PubMed)

Lindell A, Denneberg T, Hellgren E, Jeppsson JO, Tiselius HG. Clinical course and cystine stone formation during tiopronin treatment. Urol Res. 1995;23(2):111-7. doi: 10.1007/BF00307941.

Reference Type BACKGROUND
PMID: 7676533 (View on PubMed)

Ironside N, Christophe B, Bruce S, Carpenter AM, Robison T, Yoh N, Cremers S, Landry D, Frey HP, Chen CJ, Hoh BL, Kim LJ, Claassen J, Connolly ES. A phase II randomized controlled trial of tiopronin for aneurysmal subarachnoid hemorrhage. J Neurosurg. 2019 Jul 12;133(2):351-359. doi: 10.3171/2019.4.JNS19478. Print 2020 Aug 1.

Reference Type DERIVED
PMID: 31299655 (View on PubMed)

Related Links

Access external resources that provide additional context or updates about the study.

http://www.columbianeurosurgery.org/

The Columbia University Medical Center Department of Neurosurgery Website

http://www.neurosurgery.ufl.edu/

The University of Florida Department of Neurosurgery Website

http://depts.washington.edu/neurosur/

The University of Washington Department of Neurosurgery Website

http://www.columbianeurosurgery.org/doctors/e-sander-connolly-jr/

Principle Investigator: E. Sander Connolly Jr.

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

1R01FD003728-01

Identifier Type: FDA

Identifier Source: secondary_id

View Link

AAAA8597

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Dexmedetomidine and Subarachnoid Haemorrhage
NCT01664520 COMPLETED PHASE1/PHASE2
The Intra-arterial Vasospasm Trial
NCT01996436 TERMINATED PHASE4