A Phase 2/3 Study in Adult and Adolescent Participants With SCD

Официальное название

A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Adult and Adolescent Participants With Sickle Cell Disease

Обзор

The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.

Подробное описание

This is a three-part, multicenter, Phase 2/3 study of orally administered osivelotor in participants with sickle cell disease (SCD).

Part A will evaluate the safety, tolerability, and efficacy of osivelotor in adult participants with SCD to determine an optimal dose.

Part B will evaluate the efficacy of osivelotor versus placebo in adult and adolescent participants with SCD for 48 weeks.

Open Label Extension (OLE) will evaluate the long-term safety and hematologic responses of open-label osivelotor in adult and adolescent participants having completed Part B.

Вмешательства

• Препарат Osivelotor
  Tablets which contain drug substance

Первичные конечные точки

• Part A [Срок оценки: Through week 12]
• Part B [Срок оценки: Through week 48]
• OLE [Срок оценки: Approximately 24 months after last patient enrolled]

Критерии участия

Критерии включения

Part A, Part B, and OLE:

• Male or female with SCD
• Participants with stable Hb value as judged by the Investigator
• For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator.

Part B:

• Participants with SCD ages 12 to 65 years, inclusive
• Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening.

OLE:

\- Participants who have completed the Part B will be eligible.

Критерии исключения

Part A, Part B, and OLE:

• Participants who had more than 10 VOC within 12 months of screening
• Female participant who is breastfeeding or pregnant
• Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1
• Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF or anytime during the screening period.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 24 центра

• Smilow Cancer Hospital — New Haven
• Edward Jenner Research Group Center LLC — Plantation
• Pediatric Hematology / Oncology a division of Kidz Medical services — West Palm Beach
• St. Mary's Medical Center — West Palm Beach
• Alpha Clinical Research Georgia — Dunwoody
• Sonar Clinical Research — Riverdale
• University of Illinois at Chicago Clinical Research Center — Chicago
• University of Illinois Hospital and Health Sciences System - Investigational Drug Services — Chicago
• … и ещё 16 центров

Бразилия · 14 центров

• Hospital Universitario Professor Edgar Santos — Salvador
• Multihemo Servicos Medicos S/A — Recife
• Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP — Ribeirão Preto
• Fundacao Faculdade Regional de Medicina de Sao Jose do Rio Preto — São José do Rio Preto
• BP A Beneficência Portuguesa de São Paulo — São Paulo
• BP - A Beneficência Portuguesa de São Paulo — São Paulo
• Casa de Saude Santa Marcelina — São Paulo
• Casa de Saude Santa Marcelina — São Paulo
• … и ещё 6 центров

Kenya · 4 центра

• KEMRI/CRDR, Siaya, KEMRI Clinical Research Annex — Kisumu
• Gertrude's Children's Hospital — Nairobi
• Kenya Medical Research Institute - Centre for Respiratory Disease Research — Nairobi
• Center for Research In Therapeutic Sciences (CREATES), Strathmore University Medical Centr — Nairobi

Nigeria · 3 центра

• University College Hospital Ibadan — Ibadan
• Aminu Kano Teaching Hospital — Kano
• Lagos University Teaching Hospital — Lagos

Индия · 2 центра

• Nirmal Hospital Pvt Ltd — Surat
• Chopda Medicare & Research Centre Pvt. Ltd: Magnum Heart Institute — Nashik

Великобритания · 2 центра

• University Hospitals Bristol and Weston NHS Foundation Trust — Bristol
• Bristol Royal Infirmary — Bristol

Публикации

• Li Z, Alt C, Dufu K, Hutchaleelaha A, Xu Q, Zhang X, Li CM, Rademacher P, Bosmajian C, Pochron MP, Partridge JR, Oksenberg D, Cathers BE. Discovery of Osivelotor (GBT021601): A Potent, Next-Generation Sickle Hemoglobin Polymerization Inhibitor. ACS Med Chem Lett. 2025 Jul 8;16(8):1526-1532. doi: 10.1021/acsmedchemlett.5c00076. eCollection 2025 Aug 14. PMID 40832524
• Obadina M, Wilson S, Derebail VK, Little J. Emerging Therapies and Advances in Sickle Cell Disease with a Focus on Renal Manifestations. Kidney360. 2023 Jul 1;4(7):997-1005. doi: 10.34067/KID.0000000000000162. Epub 2023 May 31. PMID 37254256

Идентификаторы

NCT: NCT05431088 · GBT021601-021 · C5351004 · 2023-508766-14-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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