Pan African Clinical Trials Registry
South African Medical Research Council, South African Cochrane Centre
PO Box 19070, Tygerberg, 7505, South Africa
Telephone: +27 21 938 0506 / +27 21 938 0834 Fax: +27 21 938 0836
Email: pactradmin@mrc.ac.za Website: pactr.samrc.ac.za
Trial no.: PACTR202209867711506 Date of Approval: 05/09/2022
Trial Status: Registered in accordance with WHO and ICMJE standards
TRIAL DESCRIPTION
Public title Open-Label Extension of Voxelotor
Official scientific title An Open-Label Extension Study of Voxelotor Administered Orally to Participants with Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials
Brief summary describing the background and objectives of the trial Open-label extension study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials. The objective of this OLE is to assess the safety of, and SCD-related complications of, long-term treatment with voxelotor, in participants who have completed treatment in a Global Blood Therapeutics (GBT)-sponsored voxelotor clinical study.
Type of trial CCT
Acronym (If the trial has an acronym then please provide) Not applicable
Disease(s) or condition(s) being studied Haematological Disorders,Infections and Infestations
Sub-Disease(s) or condition(s) being studied Sickle Cell Disease
Purpose of the trial Treatment: Drugs
Anticipated trial start date 11/12/2022
Actual trial start date 23/03/2023
Anticipated date of last follow up 04/03/2028
Actual Last follow-up date
Anticipated target sample size (number of participants) 600
Actual target sample size (number of participants)
Recruitment status Recruiting
Publication URL
Secondary Ids Issuing authority/Trial register
STUDY DESIGN
Intervention assignment Allocation to intervention If randomised, describe how the allocation sequence was generated Describe how the allocation sequence/code was concealed from the person allocating the participants to the intervention arms Masking If masking / blinding was used
Crossover: all participants receive all interventions in different sequence during study Non-randomised Allocation was determined by the holder of the sequence who is situated off site Open-label(Masking Not Used)
INTERVENTIONS
Intervention type Intervention name Dose Duration Intervention description Group size Nature of control
Experimental Group Voxelotor All participants will receive voxelotor once daily (QD), administered orally as tablets, dispersible tablets, or a stick pack formulation (powder blend formulation packaged as stick packs). Participants aged ≥ 12 years and/or ≥ 40 kgs will receive a voxelotor dose of 1500 mg QD. Participants aged < 12 years and < 40 kgs will receive weight based dosing of voxelotor. The participant's weight at study entry will be used to determine the starting voxelotor dose in this study. Participants may receive study drug as long they continue to receive clinical benefit that outweighs risk as determined by the investigator and/or until the participant has access to voxelotor from an alternative source. Intervention Model: Single Group Assignment Intervention Model Description: Open-Label Extension study available to eligible participants from GBT-sponsored voxelotor pediatric clinical studies. 300
Control Group Not Provided Not Provided Not Provided Not Provided 0 Active-Treatment of Control Group
ELIGIBILITY CRITERIA
List inclusion criteria List exclusion criteria Age Category Minimum age Maximum age Gender
A. Participants who meet all of the following criteria will be eligible for study enrollment: 1. Male or female participant with SCD who participated and received study drug in a GBT-sponsored voxelotor clinical study. Note: Participants who discontinued study drug due to an AE, but who remained on study, may be eligible for treatment in this study provided the AE does not pose a risk for treatment with voxelotor. Note: Participants who discontinued Study GBT440-032 as the result of an abnormal transcranial Doppler (TCD) flow velocity assessment (≥ 200 cm/sec) are eligible for treatment in this study. 2. Female participants of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1. Note: Female participants who become childbearing during the study must be willing to have a negative urine pregnancy test to remain in the study. 3. If sexually active, female participants of childbearing potential must use highly effective methods of contraception until 30 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 30 days after the last dose of study drug. 4. Participant has provided written consent/assent (for pediatric participants, both the consent of the participant’s legal representative or legal guardian and the participant’s assent [where applicable] must be obtained) 1. Female participant who is breastfeeding or pregnant. 2. Participant withdrew consent from a GBT-sponsored voxelotor clinical study. 3. Participant was lost to follow-up from a GBT-sponsored voxelotor clinical study. 4. Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent. 5. Active symptomatic coronavirus disease of 2019 (COVID-19) infection. 6. Known hypersensitivity to voxelotor or any other components of the study drug. 7. Use of St. John’s wort, sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or moderate or strong CYP3A4 inducers within 30 days of Day 1. Adolescent: 13 Year-18 Year,Adult: 19 Year-44 Year,Child: 6 Year-12 Year,Infant: 1 Month-23 Month,Middle Aged: 45 Year(s)-64 Year(s),Preschool Child: 2 Year-5 Year 6 Month(s) 65 Year(s) Both
ETHICS APPROVAL
Has the study received appropriate ethics committee approval Date the study will be submitted for approval Date of approval Name of the ethics committee
Yes 05/10/2022 National Health Research and Ethics Committee
Ethics Committee Address
Street address City Postal code Country
Federal Ministry of Health, Federal Secretariat Complex Shehu Shagari way, Garki, Abuja, Abuja P.M.B 083 Nigeria
OUTCOMES
Type of outcome Outcome Timepoint(s) at which outcome measured
Primary Outcome 1. TEAEs and SAEs [ Time Frame: Throughout entire study ] Treatment Emergent Adverse Events and Serious Adverse Events 2. Sickle Cell Disease-Related Complications [ Time Frame: Throughout entire study ] Frequency of SCD-related complications Throughout entire study
Secondary Outcome Not Provided Not Provided
RECRUITMENT CENTRES
Name of recruitment centre Street address City Postal code Country
University of Nigeria Teaching Hospital 1 UNTH Road, Ituku/Ozalla, Enugu Enugu Nigeria
Lagos University Teaching Hospital Ishaga Road, Idi-Araba, Lagos, Nigeria Lagos Nigeria
University College Hospital Queen Elizabeth Road, Ibadan, Oyo state, Nigeria Ibadan Nigeria
Aminu Kano Teaching Hospital No 2 Zaria Road, Kano City, Kano State, Nigeria Kano Nigeria
Barau Dikko Teaching Hospital Kaduna State University Lafiya Road, City Centre, Kaduna Kaduna Nigeria
Komfo Anokye Teaching Hospital Bantama High Street, Kumasi, Ghana. Kumasi Ghana
University of Ghana Medical School College of Health Sciences Korle Bu Teaching Hospital Guggisberg Avenue, Accra, Ghana Accra Ghana
Gertrudes Childrens Hospital Muthaiga Road, Off Thika Super Highway, Nairobi Nairobi Kenya
University of Calabar Teaching Hospital Court Road Duke Town Calabar Nigeria
Centre of Excellence for Sickle Cell Research and Training University of Abuja Permanent site, Sultan Maccido road Abuja Nigeria
Center for Research In Therapeutic Sciences CREATES Strathmore University Medical Centre Madaraka Estate, Ole Sangale Road, Nairobi Nairobi Kenya
Kenya Medical Research Institute KEMRI Centre for Respiratory Disease Research CRDR Clinical Research Annex, Kenyatta National Hospital Complex Behind Government Chemist Nairobi. Nairobi Kenya
Kenya Medical Research Institute KEMRI Kondele Childrens Hospital Kisumu, Kenya Kisumu Kenya
Kenya Medical Research Institute KEMRI CRDR Siaya Clinical Research Annex K.N.H Mbagathi Rd,Nairobi Nairobi Kenya
KEMRI Centre for Clinical Research Butere County Hospital Kakamega KE, Butere Butere Kenya
FUNDING SOURCES
Name of source Street address City Postal code Country
Global Blood Therapeutics Inc 181 Oyster Point Blvd South San Francisco CA 94080 United States of America
SPONSORS
Sponsor level Name Street address City Postal code Country Nature of sponsor
Primary Sponsor Global Blood Therapeutics inc 181 Oyster Point Blvd, South San Francisco South San Francisco CA 94080 United States of America Commercial Sector/Industry
COLLABORATORS
Name Street address City Postal code Country
Xcene Research LTD 1, Ogunsiji Close, S and B Tejuosho Estate, Off Allen Avenue, Ikeja, Lagos, Nigeria Ikeja Nigeria
CONTACT PEOPLE
Role Name Email Phone Street address
Principal Investigator Adeseye Akinsete seye.akinsete@gmail.com +2348023535659 Lagos University Teaching Hospital, Ishaga Rd, Idi- Araba, Lagos, Nigeria
City Postal code Country Position/Affiliation
Lagos Nigeria Pediatrician Oncologist and Hematologist
Role Name Email Phone Street address
Public Enquiries Aimee Enriquez aenriquez@gbt.com +16507417777 181 Oyster Point Blvd.
City Postal code Country Position/Affiliation
South San Francisco United States of America Senior Clinical Trial Manager. Global Blood Therapeutics
Role Name Email Phone Street address
Scientific Enquiries Mark Davis mdavis1@gbt.com 9253361055 181 Oyster Point, Blvd
City Postal code Country Position/Affiliation
South San Francisco 94080 United States of America Senior Director. Clinical Science. Global Blood Therapeutics.Inc
REPORTING
Share IPD Description Additional Document Types Sharing Time Frame Key Access Criteria
Yes Plan to Share IPD: No. Progress will be shared at specific intervals defined per the protocol and at the end of the Study. Clinical Study Report,Informed Consent Form,Statistical Analysis Plan,Study Protocol N/A N/A
URL Results Available Results Summary Result Posting Date First Journal Publication Date
No
Result Upload 1: Result Upload 2: Result Upload 3: Result Upload 4: Result Upload 5:
Result URL Hyperlinks Link To Protocol
Result URL Hyperlinks
Changes to trial information