Sickle Cell DiseasePhase 1NCT02380079
Dose-Escalation Study of SCD-101 in Sickle Cell Disease
A Phase 1 study testing SCD-101 in people with Sickle Cell Disease and Sickle-Beta Zero Thalassemia, run by Invenux, LLC at 1 site in United States.
Trial summary
The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Sickle Cell Disease, Sickle-Beta Zero Thalassemia
- What is being tested?
- SCD-101
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2015to Dec 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Male or female, 18-55 years of age
- Homozygous sickle cell disease or S/beta 0 thalassemia
- Hemoglobin F ≤10%
- Hemoglobin ≥ 6.0 g/dL and ≤ 9.5 g/dL
- Female participants of child bearing potential and male participants whose partner is a female of child bearing potential must be willing to use approved contraception during the trial and for 3 months following the end of treatment. Only barrier methods or complete abstinence are acceptable for this study. Participants using hormonal contraception (including morning-after-pill) and IUD are excluded unless willing/able to change to an acceptable form of contraception.
- Ability to adhere to the study visit schedule and other protocol requirements
- Ability to understand and the willingness to sign an informed consent document
Who cannot join
- Red blood cell transfusion within 3 months of enrollment
- Hydroxyurea treatment within 6 months of enrollment
- Painful or other acute sickle cell event that required a hospitalization within 4-weeks of enrollment
- AST and/or ALT >3x upper limit of normal and/or creatinine >2x upper limit of normal or any other significant renal or hepatic impairment
- Estimated creatinine clearance (CrCl) < 60 mL/min (Cockcroft- Gault formula) at screening.
- QTc interval of >470 msec at trial entry and participant with congenital long QT syndrome.
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- No other significant sickle cell or non-sickle cell illness that would confound the results of the trial
- Any condition that, in the view of the investigator, places the participant at risk because of participation in the trial, or may influence the result of the trial or the participant's ability to participate in the trial
- Participant pregnant or nursing an infant or planning pregnancy during the course of the trial
- History of allergic reactions attributed to sorghum or compounds of similar chemical or biologic composition (such as Nicosan, Niprisan, Jobelyn or Xickle).
- Other investigational drug use within 3 months of enrollment
- PROMIS Fatigue Questionnaire 8a T-score ˂ 44.3
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Part A: Phase IB, Single Site, Dose-Escalation of SCD-101 and Part B: Randomized, Double-Blind, Placebo-Controlled Crossover of SCD-101 in Adults With Homozygous Sickle Cell Disease or S/Beta 0 Thalassemia.
The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.
Read the detailed description
This is single site, dose- escalation study of SCD-101 in participants with homozygous sickle cell disease (S/S) or S/beta 0 Thalassemia. All participants will be monitored for safety, tolerability, and dose-limiting toxicities.
The study is divided into two parts. Part A is an open-label, non-randomized, non-placebo-controlled dose escalation study with a 28-day treatment phase and 14-day follow-up phase with five cohorts . Part B is a randomized, placebo-controlled, confirmatory 2x2 crossover cohort with a 28 day washout between periods, and a 28-day follow-up phase.
- Conditions
- Sickle Cell Disease, Sickle-Beta Zero Thalassemia
- Being tested
- SCD-101
- Keywords
- Homozygous Sickle Cell Disease S/Beta 0 Thalassemia
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SCD-101Drug
Administered as gelatin capsules
| Study group | Role | What they receive |
|---|---|---|
| SCD-101 | Experimental | SCD-101 dosed TID for 28-days Receives: Drug: SCD-101 |
| Placebo | Placebo comparator | Placebo dosed TID for 28-days Receives: Drug: SCD-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the safety, tolerability, and dose limiting toxicities of escalating doses of SCD-101, assessed by frequency and severity of adverse events (AEs), and changes in vital signs, 12-lead ECGs and laboratory assessments as compared to baseline
Measured: From the time the participant is administered the first dose through the final follow-up (18 weeks)
Secondary outcomes (6)
Determine the effect of escalating doses of SCD-101 on the mean change in hemoglobin form base line
Measured: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change in percent reticulocytes from baseline
Measured: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in red blood cell hemolysis as measured by lactate dehydrogenase (LDH) and indirect bilirubin.
Measured: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in fatigue as measured by the PROMIS fatigue questionnaire
Measured: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in the percent of venous circulating sickle red blood cells
Measured: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in functional capacity as measured by the 6-Minute Walk Test
Measured: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2015 (estimated)
- Study startFeb 2015
- Primary completionNov 2023 (estimated)
- Study completionDec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- 2x2 crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Invenux, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- State University of New York - Downstate Medical Center
- Responsible party
- Sponsor
- Investigators
- John Muthu, MD, Principal investigator, King's County Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02380079
- Sponsor's protocol ID
- INVX-SCD-101-11
- Status last verified
- Mar 2023
- First submitted
- 23 Feb 2015
- Last updated
- 21 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Wambebe C, Khamofu H, Momoh JA, Ekpeyong M, Audu BS, Njoku OS, Bamgboye EA, Nasipuri RN, Kunle OO, Okogun JI, Enwerem MN, Audam JG, Gamaniel KS, Obodozie OO, Samuel B, Fojule G, Ogunyale O. Double-blind, placebo-controlled, randomised cross-over clinical trial of NIPRISAN in patients with Sickle Cell Disorder. Phytomedicine. 2001 Jul;8(4):252-61. doi: 10.1078/0944-7113-00040.
PubMed 11515714Iyamu EW, Turner EA, Asakura T. In vitro effects of NIPRISAN (Nix-0699): a naturally occurring, potent antisickling agent. Br J Haematol. 2002 Jul;118(1):337-43. doi: 10.1046/j.1365-2141.2002.03593.x.
PubMed 12100171Iyamu EW, Turner EA, Asakura T. Niprisan (Nix-0699) improves the survival rates of transgenic sickle cell mice under acute severe hypoxic conditions. Br J Haematol. 2003 Sep;122(6):1001-8. doi: 10.1046/j.1365-2141.2003.04536.x.
PubMed 12956772Swift, RA, Nathan, S, Tripathi, P, Chen, Q, Asakura,T (2009, September). Research Preview on Improving Nicosan™/Xickle™: A Phyto-Medicine for the Treatment of Sickle Cell Disease. Paper presented at the meeting of the Sickle Cell Disease Association of America, Orlando, FL.
Li Q, Henry ER, Hofrichter J, Smith JF, Cellmer T, Dunkelberger EB, Metaferia BB, Jones-Straehle S, Boutom S, Christoph GW, Wakefield TH, Link ME, Staton D, Vass ER, Miller JL, Hsieh MM, Tisdale JF, Eaton WA. Kinetic assay shows that increasing red cell volume could be a treatment for sickle cell disease. Proc Natl Acad Sci U S A. 2017 Jan 31;114(5):E689-E696. doi: 10.1073/pnas.1619054114. Epub 2017 Jan 17.
PubMed 28096387
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Part A: Phase IB, Single Site, Dose-Escalation of SCD-101 and Part B: Randomized, Double-Blind, Placebo-Controlled Crossover of SCD-101 in Adults With Homozygous Sickle Cell Disease or S/Beta 0 Thalassemia.
- ClinicalTrials.gov ID
- NCT02380079
- Acronym / sponsor ID
- INVX-SCD-101-11
- Conditions
- Sickle Cell Disease, Sickle-Beta Zero Thalassemia
- Keywords
- Homozygous Sickle Cell Disease S/Beta 0 Thalassemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Invenux, LLCIndustry (pharma, biotech, devices)
- Collaborators
- State University of New York - Downstate Medical Center
- Study start
- Feb 2015 (actual)
- Primary completion
- Nov 2023 (estimated)
- Study completion
- Dec 2023 (estimated)
- First posted
- 5 Mar 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.
Detailed description
This is single site, dose- escalation study of SCD-101 in participants with homozygous sickle cell disease (S/S) or S/beta 0 Thalassemia. All participants will be monitored for safety, tolerability, and dose-limiting toxicities.
The study is divided into two parts. Part A is an open-label, non-randomized, non-placebo-controlled dose escalation study with a 28-day treatment phase and 14-day follow-up phase with five cohorts . Part B is a randomized, placebo-controlled, confirmatory 2x2 crossover cohort with a 28 day washout between periods, and a 28-day follow-up phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- 2x2 crossover
| Arm | Type | Receives |
|---|---|---|
| SCD-101 | Experimental | Drug: SCD-101SCD-101 dosed TID for 28-days |
| Placebo | Placebo comparator | Drug: SCD-101Placebo dosed TID for 28-days |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Male or female, 18-55 years of age
- Homozygous sickle cell disease or S/beta 0 thalassemia
- Hemoglobin F ≤10%
- Hemoglobin ≥ 6.0 g/dL and ≤ 9.5 g/dL
- Female participants of child bearing potential and male participants whose partner is a female of child bearing potential must be willing to use approved contraception during the trial and for 3 months following the end of treatment. Only barrier methods or complete abstinence are acceptable for this study. Participants using hormonal contraception (including morning-after-pill) and IUD are excluded unless willing/able to change to an acceptable form of contraception.
Key exclusion criteria (12 listed)
- Red blood cell transfusion within 3 months of enrollment
- Hydroxyurea treatment within 6 months of enrollment
- Painful or other acute sickle cell event that required a hospitalization within 4-weeks of enrollment
- AST and/or ALT >3x upper limit of normal and/or creatinine >2x upper limit of normal or any other significant renal or hepatic impairment
- Estimated creatinine clearance (CrCl) < 60 mL/min (Cockcroft- Gault formula) at screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SCD-101Administered as gelatin capsules | Drug | Placebo, SCD-101 |
Primary outcome
Determine the safety, tolerability, and dose limiting toxicities of escalating doses of SCD-101, assessed by frequency and severity of adverse events (AEs), and changes in vital signs, 12-lead ECGs and laboratory assessments as compared to baseline
Time frame: From the time the participant is administered the first dose through the final follow-up (18 weeks)
6 secondary outcomes
Determine the effect of escalating doses of SCD-101 on the mean change in hemoglobin form base line
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change in percent reticulocytes from baseline
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in red blood cell hemolysis as measured by lactate dehydrogenase (LDH) and indirect bilirubin.
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in fatigue as measured by the PROMIS fatigue questionnaire
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in the percent of venous circulating sickle red blood cells
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in functional capacity as measured by the 6-Minute Walk Test
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2023.
- Background: PubMed 11515714Wambebe C, Khamofu H, Momoh JA, Ekpeyong M, Audu BS, Njoku OS, Bamgboye EA, Nasipuri RN, Kunle OO, Okogun JI, Enwerem MN, Audam JG, Gamaniel KS, Obodozie OO, Samuel B, Fojule G, Ogunyale O. Double-blind, placebo-controlled, randomised cross-over clinical trial of NIPRISAN in patients with Sickle Cell Disorder. Phytomedicine. 2001 Jul;8(4):252-61. doi: 10.1078/0944-7113-00040.
- Background: PubMed 12100171Iyamu EW, Turner EA, Asakura T. In vitro effects of NIPRISAN (Nix-0699): a naturally occurring, potent antisickling agent. Br J Haematol. 2002 Jul;118(1):337-43. doi: 10.1046/j.1365-2141.2002.03593.x.
- Background: PubMed 12956772Iyamu EW, Turner EA, Asakura T. Niprisan (Nix-0699) improves the survival rates of transgenic sickle cell mice under acute severe hypoxic conditions. Br J Haematol. 2003 Sep;122(6):1001-8. doi: 10.1046/j.1365-2141.2003.04536.x.
- BackgroundSwift, RA, Nathan, S, Tripathi, P, Chen, Q, Asakura,T (2009, September). Research Preview on Improving Nicosan™/Xickle™: A Phyto-Medicine for the Treatment of Sickle Cell Disease. Paper presented at the meeting of the Sickle Cell Disease Association of America, Orlando, FL.
- Derived from this study: PubMed 28096387Li Q, Henry ER, Hofrichter J, Smith JF, Cellmer T, Dunkelberger EB, Metaferia BB, Jones-Straehle S, Boutom S, Christoph GW, Wakefield TH, Link ME, Staton D, Vass ER, Miller JL, Hsieh MM, Tisdale JF, Eaton WA. Kinetic assay shows that increasing red cell volume could be a treatment for sickle cell disease. Proc Natl Acad Sci U S A. 2017 Jan 31;114(5):E689-E696. doi: 10.1073/pnas.1619054114. Epub 2017 Jan 17.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John Muthu, MD principal investigator
King's County Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SCD-101 in people with Sickle Cell Disease and Sickle-Beta Zero Thalassemia, run by Invenux, LLC at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 21 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine the safety, tolerability, and dose limiting toxicities of escalating doses of SCD-101, assessed by frequency and severity of adverse events (AEs), and changes in vital signs, 12-lead ECGs and laboratory assessments as compared to baseline (time frame: From the time the participant is administered the first dose through the final follow-up (18 weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02380079, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Mar 2023.