Skip to main menu Skip to main content Skip to footer


What is a Clinical Trial?

A clinical trial is a research study conducted with volunteers to evaluate the safety & effectiveness of a new medical treatment or intervention. It involves carefully designed protocols and methodology to gather scientific evidence and assess the potential benefits and risks of the intervention. These trials play a crucial role in advancing medical knowledge and improving healthcare by testing new drugs, therapies, and medical devices before they can be approved for widespread use.

Woman Smiling

Clinical Trials

MacTel Phase 3 Clinical Trials

United States
United Kingdom

MacTel Studies

A Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2 – Protocol A ID NCT03316300, Information provided by Neurotech Pharmaceuticals (Responsible Party), Last Update Posted 2023-02-08

A Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2 ID NCT03319849, Information provided by Neurotech Pharmaceuticals (Responsible Party), Last Update Posted 2023-02-08

NTMT-03A and NTMT-03B Participant Letter
November 2022

Bilateral Implant and Extension Studies

Safety Study to Evaluate Bilateral CNTF Implants in Subjects With MacTel Type 2 ID NCT04729972, Information provided by Neurotech Pharmaceuticals (Responsible Party), Last Update Posted 2023-02-08

Extension Study of NT-501 Ciliary Neurotrophic Factor (CNTF) Implant for Macular Telangiectasia (MacTel) ID NCT03071965. Information provided by Neurotech Pharmaceuticals (Responsible Party), Last Update Posted 2022-03-17

Clinical Trial Results

Effect of Ciliary Neurotrophic Factor on Retinal Neurodegeneration in Patients with Macular Telangiectasia Type 2: A Randomized Clinical Trial.
Emily Y. Chew, MD, Traci E. Clemons, PhD, Glenn J. Jaffe, MD, Charles A. Johnson, MD, Sina Farsiu, PhD, Eleonora M. Lad, MD, PhD, Robyn Guymer, MD, PhD,
Philip Rosenfeld, MD, PhD, Jean-Pierre Hubschman, MD, Ian Constable, MD, Henry Wiley, MD, Lawrence J. Singerman, MD, Mark Gillies, MD, PhD,
Grant Comer, MD, Barbara Blodi, MD, Dean Eliott, MD, Jiong Yan, MD, Alan Bird, MD, Martin Friedlander, MD, PhD, MacTel Type 2-Ph 2 CNTF Research Group.

Ciliary Neurotrophic Factor Delivered by Encapsulated Cell Intraocular Implants for Treatment of Geographic Atrophy in Age-Related Macular Degeneration
Kang Zhang, Jill J. Hopkins, Jeffrey S. Heier, David G. Birch, Lawrence S. Halperin, Thomas A. Albini, David M. Brown, Glenn J. Jaffe, Weng Tao, and George A. Williams.
Proc Natl Acad Sci U S A. 2011 Apr 12; 108(15): 6241–6245. Published online 2011 Mar 28. doi: 10.1073/pnas.1018987108

Ciliary Neurotrophic Factor (CNTF) for Human Retinal Degeneration: Phase I Trial of CNTF Delivered by Encapsulated Cell Intraocular Implants
Paul A. Sieving, Rafael C. Caruso, Weng Tao,§ Hanna R. Coleman, Darby J. S. Thompson, Keri R. Fullmer, and Ronald A. Bush.
Proc Natl Acad Sci U S A. 2006 Mar 7; 103(10): 3896–3901. Published online 2006 Feb 27. doi: 10.1073/pnas.0600236103

Longitudinal Study of Cone Photoreceptors During Retinal Degeneration and in Response to Ciliary Neurotrophic Factor Treatment
Katherine E Talcott, Kavitha Ratnam, Sanna M Sundquist, Anna S Lucero, Brandon J Lujan, Weng Tao, Travis C Porco, Austin Roorda, Jacque L Duncan.
PMID: 21087953 PMCID: PMC3080173 DOI: 10.1167/iovs.10-6479

Ciliary Neurotrophic Factor for Macular Telangiectasia Type 2: Results from a Phase 1 Safety Trial
Emily Y Chew , Traci E Clemons , Tunde Peto, Ferenc B Sallo, Avner Ingerman, Weng Tao, Lawrence Singerman, Steven D Schwartz, Neal S Peachey,
Alan C Bird, MacTel-CNTF Research Group. PMID: 25528956 PMCID: PMC4361328 DOI: 10.1016/j.ajo.2014.12.013

Ciliary Neurotrophic Factor Treatment Improves Retinal Structure and Function in Macular Telangiectasia Type 2
Jacque L Duncan, PMID: 30910039 DOI: 10.1016/j.ophtha.2018.12.023

Neurotech Pre-Approval Access
And/Or Single Patient Requests

Neurotech required conditions for consideration of Pre-Approval Access and Single Patient Requests

  1. The patient must have a serious or life-threatening disease or condition.
  2. There must be an unmet medical need, or alternative therapies are not available, or the patient must have exhausted all such alternative therapies.
  3. The patient is not eligible or cannot participate in a clinical trial. In assessing the eligibility of a patient for potential pre-approval access, preference will be given to clinical trials, then pre-approval access programs, and then single patient access.
  4. Adequate supply is available to support both clinical development activities and pre-approval access.
  5. Sufficient scientific evidence to demonstrate that the benefits of the investigational medicine outweigh the risks.
  6. Providing pre-approval access will not jeopardize the initiation, conduct, or completion of clinical investigations and the overall development program to support registration of the product.
  7. Pre-approval access must be permitted by, and run in accordance with, applicable laws.
  8. The treating physician making the request is licensed and qualified to administer the investigational medicine and agrees to comply with Neurotech requirements and local regulations governing preapproval access and to adhere to applicable laws and regulations.